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Chemical Structure| 873054-44-5 Chemical Structure| 873054-44-5

Structure of Ivacaftor
CAS No.: 873054-44-5

Chemical Structure| 873054-44-5

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Ivacaftor is a potentiator of CFTR targeting G551D-CFTR and F508del-CFTR with EC50 of 100 nM and 25 nM, respectively.

Synonyms: VX-770

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Product Details of Ivacaftor

CAS No. :873054-44-5
Formula : C24H28N2O3
M.W : 392.49
SMILES Code : O=C(C1=CNC2=C(C=CC=C2)C1=O)NC3=CC(O)=C(C(C)(C)C)C=C3C(C)(C)C
Synonyms :
VX-770
MDL No. :MFCD17171361
InChI Key :PURKAOJPTOLRMP-UHFFFAOYSA-N
Pubchem ID :16220172

Safety of Ivacaftor

GHS Pictogram:
Signal Word:Warning
Hazard Statements:H302-H315-H319-H335
Precautionary Statements:P261-P305+P351+P338

Isoform Comparison

Biological Activity

In Vitro:

Cell Line
Concentration Treated Time Description References
human corneal epithelial cells (pHCECs) 10 µM 72 h To evaluate the effect of Ivacaftor on EXOA-induced P38 phosphorylation and cell death in CF cells, results showed that Ivacaftor significantly reduced P38 phosphorylation and decreased EXOA-induced cell death in CF cells. PMC10465324
human nasal epithelial cells (pHNECs) 10 µM 72 h To evaluate the effect of Ivacaftor on EXOA-induced P38 phosphorylation and cell death in CF cells, results showed that Ivacaftor significantly reduced P38 phosphorylation and decreased EXOA-induced cell death in CF cells. PMC10465324
FRT cells 10 µM 48 h To evaluate the effect of SRI-37240 on CFTR-dependent chloride conductance, results showed that SRI-37240 significantly increased CFTR-dependent chloride conductance and had synergistic effects with G418 PMC8285393
16HBEge cells 5 µM 72 h To evaluate the effect of SRI-41315 on CFTR function, results showed that SRI-41315 significantly increased CFTR function and had synergistic effects with G418 PMC8285393
Calu-3 2B4 cells 5 µM 48 h To evaluate the effect of Ivacaftor on SARS-CoV-2 replication, results showed that Ivacaftor treatment significantly reduced virus titers. PMC9973274

In Vivo:

Species
Animal Model
Administration Dosage Frequency Description References
Mice K18-hACE2 mice Intraperitoneal injection 6 mg/kg Once daily for 8 days To evaluate the protective effect of Ivacaftor in SARS-CoV-2 infected mice, results showed that Ivacaftor treatment significantly improved survival and reduced lung edema. PMC9973274
Mice BALB/CJ mice Subcutaneous injection 50 mg/kg Single injection, lasting 28 days To study the pharmacokinetics of Ivacaftor-loaded PLGA microparticles in mice, the results showed that the microparticle formulation maintained stable plasma drug concentrations for 28 days, with a 6-fold increase in AUC compared to intravenous administration of solubilized Ivacaftor. PMC10843602

Clinical Trial:

NCT Number Conditions Phases Recruitment Completion Date Locations
NCT03525548 Cystic Fibrosis Phase 3 Active, not recruiting March 2019 -
NCT03447249 Cystic Fibrosis Phase 3 Active, not recruiting April 16, 2019 -
NCT03525574 Cystic Fibrosis Phase 3 Enrolling by invitation June 2021 -
NCT03506061 Cystic Fibrosis Phase 2 Not yet recruiting May 2023 United States, Alabama ... More >> University of Alabama Cystic Fibrosis Research Center Not yet recruiting Birmingham, Alabama, United States, 35233 Contact: George Soloman, MD    205-975-9776    msolomon@uab.edu    United States, Georgia Emory Children's Center Not yet recruiting Atlanta, Georgia, United States, 30322 Contact: Eric Sorscher, MD    205-612-1327    esorscher@emory.edu Less <<
NCT03447262 Cystic Fibrosis Phase 3 Enrolling by invitation April 24, 2021 -
NCT03587961 Cystic Fibrosis Early Phase 1 Not yet recruiting January 1, 2021 United States, Alabama ... More >> University of Alabama at Birmingham Birmingham, Alabama, United States, 35233 Less <<
NCT02951182 Cystic Fibrosis Phase 2 Completed - -
NCT02392702 Healthy Phase 1 Completed - Australia, South Australia ... More >> CMAX Adelaide, South Australia, Australia, 5000 Less <<
NCT02599792 Healthy Phase 1 Completed - Australia, South Australia ... More >> Adelaide, South Australia, Australia, 5000 Less <<
NCT03445793 - Recruiting December 31, 2019 United States, Colorado ... More >> National Jewish Heatlh Recruiting Denver, Colorado, United States, 80206 Contact: Terri Herrud, BS    303-398-1453    herrudt@njhealth.org Less <<
NCT02997020 - Recruiting May 2022 United States, Alabama ... More >> University of Alabama at Birmingham Recruiting Birmingham, Alabama, United States, 35294 Contact: Lisa Clemons, RN, MSN    205-934-9714    lkclemons@uabmc.edu Less <<
NCT02589236 Cystic Fibrosis Phase 2 Completed - -
NCT02311140 - Unknown January 2018 Germany ... More >> Jena University Hospital, CF Center Recruiting Jena, Germany, 07743 Contact: Jochen G Mainz, MD, PD    +493641938425    jochen.mainz@med.uni-jena.de Less <<
NCT02722057 - Active, not recruiting December 2019 -
NCT01549314 - Active, not recruiting December 2018 United States, Massachusetts ... More >> Massachusetts General Hospital Boston, Massachusetts, United States, 02114 Less <<
NCT02871778 Primary Ciliary Dyskinesia Phase 2 Active, not recruiting April 2019 -
NCT03045523 Cystic Fibrosis Phase 2 Completed - Australia ... More >> The Prince Charles Hospital Chermside, Australia The Alfred Melbourne, Australia Sir Charles Gairdner Hospital Nedlands, Australia Westmead Hospital Westmead, Australia Belgium UZ Brussel Brussels, Belgium UZ Gent Ghent, Belgium UZ Leuven Leuven, Belgium Czechia Fakultni nemocnice v Motole Praha, Czechia Germany Universitaetsklinikum Carl Gustav Carus TU Dresden Dresden, Germany Universitätsklinikum Erlangen Erlangen, Germany University Children´s Hospital Tübingen, Germany Ireland Cork University Hospital Cork, Ireland Beaumont Hospital Dublin, Ireland St Vincents University Hospital Dublin, Ireland United Kingdom Birmingham Heartlands Birmingham, United Kingdom Royal Devon and Exeter Exeter, United Kingdom St James's University Leeds, United Kingdom Liverpool Heart and Chest Hospital Liverpool, United Kingdom Royal Brompton Hospital London, United Kingdom University Hospital of South Manchester Manchester, United Kingdom Royal Victoria Infirmary Newcastle, United Kingdom Southampton General Hospital Southampton, United Kingdom Less <<
NCT03537651 Cystic Fibrosis Phase 3 Enrolling by invitation December 2020 -
NCT02445053 - Active, not recruiting December 2020 Italy ... More >> Bari, Italy Messina, Italy Milano, Italy Milan, Italy Naples, Italy Potenza, Italy Verona, Italy Netherlands Den Haag, South Holland, Netherlands Amsterdam, Netherlands Gronningen, Netherlands Heidelberglaan, Netherlands Nijmegen, Netherlands Rotterdam, Netherlands United Kingdom London, Greater London, United Kingdom Birmingham, West Midlands, United Kingdom Less <<
NCT02347657 Cystic Fibrosis Phase 3 Completed - -
NCT03559062 Cystic Fibrosis Phase 3 Recruiting February 2019 Australia ... More >> Hunter Medical Research Institute (HMRI) Recruiting New Lambton Heights, Australia Princess Margaret Hospital for Children Recruiting Perth, Australia Lady Cilento Children's Hospital Recruiting South Brisbane, Australia The Children's Hospital at Westmead Recruiting Westmead, Australia Belgium Universitair Ziekenhuis Brussel - Campus Jette Recruiting Brussels, Belgium Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg Recruiting Leuven, Belgium Denmark University of Copenhagen Rigshospitalet Recruiting Copenhagen, Denmark France Groupe Hospitalier Pellegrin - Hôpital des Enfants Recruiting Bordeaux Cedex, France Hôpital Necker - Enfants Malades Recruiting Paris, France Germany Universitaetsklinikum Essen Recruiting Essen, Germany Klinikum der Johann Wolfgang Goethe-Universitaet Recruiting Frankfurt, Germany Universitaetsklinikum Giessen und Marburg GmbH Standort Giessen Recruiting Giessen, Germany Medizinische Hochschule Hannover Recruiting Hannover, Germany Universitaetsklinikum Heidelberg Recruiting Heidelberg, Germany Universitaetsklinikum Jena Recruiting Jena, Germany Universitaetsklinikum Koeln Not yet recruiting Koeln, Germany Universitaetsklinikum Tuebingen Recruiting Tuebingen, Germany Ireland Our Lady's Children's Hospital Recruiting Dublin, Ireland University Hospital Limerick Recruiting Limerick, Ireland Poland Klinika Mukowiscydozy, Oddział Chorób Płuc SZP ZOZ Recruiting Dziekanow Lesny, Poland Switzerland Inselspital - Universitaetsspital Bern Recruiting Bern, Switzerland Kinderspital Zuerich Recruiting Zuerich, Switzerland United Kingdom Royal Hospital for Sick Children Recruiting Edinburgh, United Kingdom Leeds General Infirmary Recruiting Leeds, United Kingdom Royal Brompton Hospital Recruiting London, United Kingdom Nottingham University Hospital City Campus Recruiting Nottingham, United Kingdom Southampton General Hospital Recruiting Southampton, United Kingdom Less <<
NCT01381289 Cystic Fibrosis APPROVED_FOR_MARKETING - -
NCT03256968 Cystic Fibrosis Phase 4 Active, not recruiting December 31, 2018 United States, Alabama ... More >> University of Alabama at Birmingham Birmingham, Alabama, United States, 35233 Less <<
NCT02724527 Cystic Fibrosis Phase 2 Unknown April 2017 United States, Colorado ... More >> National Jewish Health Denver, Colorado, United States, 80206 United States, Maryland Johns Hopkins Hospital Baltimore, Maryland, United States, 21287 United States, Massachusetts Boston Children's Hospital Boston, Massachusetts, United States, 02115 United States, Missouri Washington University St. Louis, Missouri, United States, 63110 United States, New York Columbia University New York, New York, United States, 10032 United States, Ohio Cincinnati Children's Hospital Cincinnati, Ohio, United States, 45229 Rainbow Babies and Children's Hospital - Case Medical Center Cleveland, Ohio, United States, 44106 Nationwide Children's Hospital Columbus, Ohio, United States, 43205 United States, Oregon Oregon Health and Science University Portland, Oregon, United States, 97239 United States, Pennsylvania Children's Hospital Pittsburgh Pittsburgh, Pennsylvania, United States, 15224 United States, Utah University of Utah Salt Lake City, Utah, United States, 84132 United States, Wisconsin Medical Center of Wisconsin Madison, Wisconsin, United States, 53792 Less <<
NCT03624101 Cystic Fibrosis Early Phase 1 Recruiting November 1, 2021 United States, Alabama ... More >> University of Alabama at Birmingham Recruiting Birmingham, Alabama, United States, 35233 Contact: Heather R Hathorne, MAE, RRT    205-939-9568    hhathorne@peds.uab.edu    Principal Investigator: Steven M Rowe, MD, MpH Less <<
NCT02875366 Cystic Fibrosis Phase 4 Completed - Australia, Victoria ... More >> Melbourne, Victoria, Australia Parkville, Victoria, Australia Australia Adelaide, Australia Camperdown, Australia Clayton, Australia Nedlands, Australia New Lambton Heights, Australia Randwick, Australia South Brisbane, Australia Subiaco, Australia Westmead, Australia United Kingdom Edinburgh, United Kingdom Less <<
NCT02347657 - Completed - -
NCT03061331 - Completed - -
NCT03061331 Cystic Fibrosis Phase 2 Completed - Netherlands ... More >> HagaZiekenhuis Den Haag, Netherlands University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis Heidelberglaan, Netherlands Less <<
NCT02141464 - Completed - United States, Pennsylvania ... More >> Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States, 19104 Less <<
NCT02070744 Cystic Fibrosis Phase 2 Completed - -
NCT00966602 Cystic Fibrosis Phase 1 Completed - Netherlands ... More >> Zuidlaren, Netherlands Less <<
NCT03390985 - Completed - -
NCT02070744 - Completed - -
NCT03278314 - - - -
NCT01216046 Cystic Fibrosis Phase 1 Completed - United States, Kansas ... More >> PRA Lenexa, Kansas, United States, 66219 Less <<
NCT01225211 Cystic Fibrosis Phase 2 Completed - -
NCT01208285 In Development for Cystic Fibr... More >>osis Less << PHASE1 COMPLETED 2025-12-10 Hradec Králové, Czech Republic... More >>|Praha, Czech Republic|Bratislava, Slovakia Less <<
NCT01910415 Cystic Fibrosis Phase 1 Completed - Netherlands ... More >> Zuidlaren, Netherlands Less <<
NCT01060566 Cystic Fibrosis Phase 1 Completed - United States, Florida ... More >> Covan CRU, Inc. Daytona Beach, Florida, United States, 32117 Less <<
NCT02039986 - Completed - United States, Pennsylvania ... More >> The Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States, 19104 Less <<
NCT01531673 Cystic Fibrosis Phase 2 Completed - -
NCT03601637 Cystic Fibrosis Phase 3 Recruiting September 2020 United States, Alabama ... More >> University of Alabama at Birmingham Recruiting Birmingham, Alabama, United States, 35294 United States, Arkansas Arkansas Children's Hospital Recruiting Little Rock, Arkansas, United States, 72202 United States, Illinois Ann & Robert Lurie Children's Hospital of Chicago Recruiting Chicago, Illinois, United States, 60611 United States, Indiana Riley Hospital for Children at Indiana University Health Recruiting Indianapolis, Indiana, United States, 46202 United States, Massachusetts Boston Children's Hospital Recruiting Boston, Massachusetts, United States, 02115 United States, Minnesota Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota Recruiting Minneapolis, Minnesota, United States, 55404 United States, Missouri The Children's Mercy Hospital Recruiting Kansas City, Missouri, United States, 64108 United States, New York Albany Medical College Recruiting Albany, New York, United States, 12208 United States, Pennsylvania Children's Hospital of Philadelphia Recruiting Philadelphia, Pennsylvania, United States, 19104 United States, Texas Cook Children's Medical Center Recruiting Fort Worth, Texas, United States, 76104 United States, Utah University of Utah / Primary Children's Medical Center Recruiting Salt Lake City, Utah, United States, 84132 United States, Washington Seattle Children's Hospital Recruiting Seattle, Washington, United States, 98105 United States, Wisconsin University of Wisconsin Hospital and Clinics Recruiting Madison, Wisconsin, United States, 53792 Canada British Columbia's Children's Hospital Recruiting Vancouver, Canada Less <<
NCT01262352 Cystic Fibrosis Phase 2 Completed - United States, California ... More >> Stanford, California, United States United States, Iowa Iowa City, Iowa, United States United States, North Carolina Durham, North Carolina, United States United States, Pennsylvania Pittsburgh, Pennsylvania, United States Canada, Ontario Toronto, Ontario, Canada United Kingdom Belfast, United Kingdom Edinburgh, United Kingdom London, United Kingdom Less <<
NCT01153542 In Development for Cystic Fibr... More >>osis Less << Phase 1 Completed - United States, Florida ... More >> Covance CRU, Inc. Daytona Beach, Florida, United States, 32117 Less <<
NCT01262352 - Completed - -
NCT01018368 Cystic Fibrosis Phase 1 Completed - United States, Florida ... More >> Daytona Beach, Florida, United States Less <<
NCT03625466 Cystic Fibrosis Phase 2 Recruiting May 2021 Germany ... More >> Charite Paediatric Pulmonology Department Recruiting Berlin, Germany Justus-Leibig-Universitat Zentrum fur Kinderheilkunde und Jugendmedizin Recruiting Giessen, Germany Hannover Medical School Recruiting Hannover, Germany Heidelberg Cystic Fibrosis Center Recruiting Heidelberg, Germany Universitatsklinikum Schleswig-Holstein, Klinik für Kinder- und Jugendmedizin Recruiting Lubeck, Germany Less <<
NCT01225211 - Completed - -
NCT03486236 Cystic Fibrosis Phase 1 Completed - United Kingdom ... More >> The Medicines Evaluation Unit Manchester, United Kingdom Less <<
NCT03125395 Cystic Fibrosis Phase 3 Active, not recruiting July 26, 2019 -
NCT03150719 Cystic Fibrosis Phase 3 Completed - -
NCT02797132 - Completed - -
NCT03565692 - Recruiting March 1, 2020 France ... More >> CHU de Bordeaux - CRCM Recruiting Bordeaux, France, 33000 Contact: Mickael FAYON, MD,PhD       mickael.fayon@chu-bordeaux.fr    Principal Investigator: Mickael FAYON, MD,PhD          Sub-Investigator: Patrick BERGER, MD,PhD          Sub-Investigator: Raphael ENAUD, MD          Centre Hospitalier Universitaire Grenoble Alpes Recruiting Grenoble, France, 38000 Contact: Catherine Llerena, MD       CLlerena@chu-grenoble.fr    Principal Investigator: Catherine Llerena, MD          Sub-Investigator: Boubou Camara, MD          CHRU de Lille Not yet recruiting Lille, France, 69000 Contact: Nathalie Wizla, MD       nathalie.wizla@chru-lille.fr    Principal Investigator: Nathalie Wizla, MD          Sub-Investigator: Anne Prévotat, MD          Hospices Civils de Lyon Not yet recruiting Lyon, France, 69000 Contact: Philippe Reix, MD       philippe.reix@chu-lyon.fr    Principal Investigator: Philippe Reix, MD          Assistance publique Hôpitaux Marseille Recruiting Marseille, France, 13000 Contact: Jean Christophe Dubus, MD,PhD       JeanChristophe.DUBUS@ap-hm.fr    Principal Investigator: Jean Christophe Dubus, MD,PhD          Assistance Publique Hôpitaux de paris Recruiting Paris, France, 75000 Contact: Harriet Corvol, MD,PhD       harriet.corvol@aphp.fr    Principal Investigator: Harriet Corvol, MD,PhD          Sub-Investigator: Pierre-Regis BURGEL, MD          Sub-Investigator: Isabelle Sermet-Gaudelus, MD,PhD          Sub-Investigator: Michèle Gérardin Gérardin, MD          Hôpital FOCH Recruiting Suresnes, France, 92150 Contact: Dominique Grenet, MD       d.grenet@hopital-foch.org    Principal Investigator: Dominique Grenet, MD          CHU de Toulouse Recruiting Toulouse, France, 31000 Contact: Marlène Murris-Espin, MD       murris.m@chu-toulouse.fr    Contact: Murris-Espin          Principal Investigator: Marlène Murris-Espin, MD Less <<
NCT02516410 Cystic Fibrosis Phase 3 Completed - -
NCT01531673 - Completed - -
NCT02516410 - Completed - -
NCT02730208 Cystic Fibrosis Phase 2 Completed - Australia ... More >> Chermside, Australia Melbourne, Australia Nedlands, Australia New Lambton Heights, Australia Parkville SIC, Australia Randwick, Australia South Brisbane, Australia Subiaco, Australia Westmead, Australia Less <<
NCT01931839 Cystic Fibrosis, Homozygous or... More >> Heterozygous for the F508del-CFTR Mutation Less << Phase 3 Completed - -
NCT00457821 - Completed - -
NCT02508207 Cystic Fibrosis Phase 2 Completed - United States, Alabama ... More >> Birmingham, Alabama, United States United States, Indiana Indianapolis, Indiana, United States United States, Maryland Baltimore, Maryland, United States United States, North Carolina Chapel Hill, North Carolina, United States United States, Ohio Cincinnati, Ohio, United States Toledo, Ohio, United States United States, Pennsylvania Pittsburgh, Pennsylvania, United States Less <<
NCT00457821 Cystic Fibrosis Phase 2 Completed - United States, Alabama ... More >> University of Alabama Hospital Birmingham, Alabama, United States, 35211 United States, California Stanford University Medical Center Palo Alto, California, United States, 34304 United States, Colorado The Children's Hospital Aurora, Colorado, United States, 80045 United States, Iowa Roy J. and Lucille A. Carver College of Medicine, The University of Iowa Iowa City, Iowa, United States, 52242-1083 United States, Maryland Johns Hopkins Hospital Baltimore, Maryland, United States, 21287 United States, Massachusetts Pulmonary and Critical Care Medicine, Massachusetts General Hospital Boston, Massachusetts, United States, 02114 Children's Hospital of Boston Boston, Massachusetts, United States, 02215 United States, Minnesota Division of Pulmonary, Allergy and Critical Care Medicine, University of Minnesota Minneapolis, Minnesota, United States, 55455 United States, North Carolina Cystic Fibrosis Pulmonary Research and Treatment Center, University of North Carolina at Chapel Hill Chapel Hill, North Carolina, United States, 27599 United States, Ohio Rainbow Babies and Children's Hospital Cleveland, Ohio, United States, 44106 United States, Pennsylvania The Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States, 19104-4399 University of Pittsburgh Pittsburgh, Pennsylvania, United States, 15213 United States, Washington Pulmonary Critical Care, University of Washington Seattle, Washington, United States, 98195 Canada, Ontario Division of Gastroenterology, Hepatology and Nutrition, The Hospital for Sick Children Toronto, Ontario, Canada, M5G 1X8 Germany CF Clinic, Pediatric Pulmonology and Neonatology, Medical School of Hannover Strasse, Hannover, Germany, D-30625 Less <<
NCT02508207 - Completed - -
NCT02797132 Cystic Fibrosis Phase 3 Completed - -
NCT01931839 - Completed - -
NCT01807949 Cystic Fibrosis, Homozygous fo... More >>r the F508del CFTR Mutation Less << Phase 3 Completed - -
NCT01807923 Cystic Fibrosis, Homozygous fo... More >>r the F508del CFTR Mutation Less << Phase 3 Completed - -
NCT02953314 Cystic Fibrosis Phase 3 Completed - -
NCT02392234 Cystic Fibrosis Phase 3 Completed - -
NCT01888393 Hepatic Impairment ... More >> Healthy Less << Phase 1 Completed - Czech Republic ... More >> Praha, Czech Republic Slovakia Bratislava, Slovakia Less <<
NCT01807949 - Completed - -
NCT00909727 Cystic Fibrosis Phase 3 Completed - -
NCT02514473 - Completed - -
NCT00909532 Cystic Fibrosis Phase 3 Completed - -
NCT02392234 - Completed - -
NCT00909532 - Completed - -
NCT01807923 - Completed - -
NCT01768663 Cystic Fibrosis Phase 1 Completed - United States, Indiana ... More >> Evansville, Indiana, United States Less <<
NCT02514473 Cystic Fibrosis Phase 3 Completed - -
NCT01161537 Cystic Fibrosis Phase 2 Completed - United States, Virginia ... More >> Charlottesville, Virginia, United States Less <<
NCT01117012 Cystic Fibrosis PHASE3 COMPLETED 2025-05-14 Birmingham, Alabama, United St... More >>ates|Oakland, California, United States|Palo Alto, California, United States|San Diego, California, United States|Denver, Colorado, United States|Atlanta, Georgia, United States|Boise, Idaho, United States|Chicago, Illinois, United States|Indianapolis, Indiana, United States|Iowa City, Iowa, United States|Baltimore, Maryland, United States|Boston, Massachusetts, United States|Ann Arbor, Michigan, United States|Detroit, Michigan, United States|Minneapolis, Minnesota, United States|Kansas City, Missouri, United States|St. Louis, Missouri, United States|Omaha, Nebraska, United States|Long Branch, New Jersey, United States|Buffalo, New York, United States|New Hyde Park, New York, United States|Syracuse, New York, United States|Cincinnati, Ohio, United States|Cleveland, Ohio, United States|Columbus, Ohio, United States|Toledo, Ohio, United States|Portland, Oregon, United States|Hershey, Pennsylvania, United States|Philadelphia, Pennsylvania, United States|Pittsburgh, Pennsylvania, United States|Knoxville, Tennessee, United States|Nashville, Tennessee, United States|Salt Lake City, Utah, United States|Chrlottesville, Virginia, United States|Seattle, Washington, United States|Morgantown, West Virginia, United States|Westmead, New South Wales, Australia|Chermside, Queensland, Australia|Herston, Queensland, Australia|South Brisbane, Queensland, Australia|Parkville, Victoria, Australia|Nedlands, Western Australia, Australia|Subiaco, Western Australia, Australia|Vancouver, British Columbia, Canada|Halifax, Nova Scotia, Canada|Toronto, Ontario, Canada|Montreal, Quebec, Canada|Prague, Czech Republic|Paris, France|Roscoff, France|Erlangen, Germany|Jena, Germany|Munich, Germany|Wurzburg, Germany|Cork, Ireland|Dublin, Ireland|Belfast, Northern Ireland, United Kingdom|London, United Kingdom Less <<
NCT01161537 - Completed - -
NCT00909727 - Completed - -
NCT01117012 Cystic Fibrosis PHASE3 COMPLETED 2025-05-14 Birmingham, Alabama, United St... More >>ates|Oakland, California, United States|Palo Alto, California, United States|San Diego, California, United States|Denver, Colorado, United States|Atlanta, Georgia, United States|Boise, Idaho, United States|Chicago, Illinois, United States|Indianapolis, Indiana, United States|Iowa City, Iowa, United States|Baltimore, Maryland, United States|Boston, Massachusetts, United States|Ann Arbor, Michigan, United States|Detroit, Michigan, United States|Minneapolis, Minnesota, United States|Kansas City, Missouri, United States|St. Louis, Missouri, United States|Omaha, Nebraska, United States|Long Branch, New Jersey, United States|Buffalo, New York, United States|New Hyde Park, New York, United States|Syracuse, New York, United States|Cincinnati, Ohio, United States|Cleveland, Ohio, United States|Columbus, Ohio, United States|Toledo, Ohio, United States|Portland, Oregon, United States|Hershey, Pennsylvania, United States|Philadelphia, Pennsylvania, United States|Pittsburgh, Pennsylvania, United States|Knoxville, Tennessee, United States|Nashville, Tennessee, United States|Salt Lake City, Utah, United States|Chrlottesville, Virginia, United States|Seattle, Washington, United States|Morgantown, West Virginia, United States|Westmead, New South Wales, Australia|Chermside, Queensland, Australia|Herston, Queensland, Australia|South Brisbane, Queensland, Australia|Parkville, Victoria, Australia|Nedlands, Western Australia, Australia|Subiaco, Western Australia, Australia|Vancouver, British Columbia, Canada|Halifax, Nova Scotia, Canada|Toronto, Ontario, Canada|Montreal, Quebec, Canada|Prague, Czech Republic|Paris, France|Roscoff, France|Erlangen, Germany|Jena, Germany|Munich, Germany|Wurzburg, Germany|Cork, Ireland|Dublin, Ireland|Belfast, Northern Ireland, United Kingdom|London, United Kingdom Less <<
NCT01937325 Cystic Fibrosis Phase 4 Unknown December 2016 Australia, Victoria ... More >> The Alfred Melbourne, Victoria, Australia, 3004 Less <<
NCT00953706 Cystic Fibrosis Phase 2 Terminated(Following review of... More >> results obtained from a pre-specified 6-month analysis of Part B data the study was terminated on the basis of futility.) Less << - -
NCT02390219 Cystic Fibrosis ... More >> Advanced Lung Disease Less << Phase 3 Completed - United States, Colorado ... More >> Denver, Colorado, United States United States, Florida Tampa, Florida, United States United States, Illinois Chicago, Illinois, United States United States, Missouri Saint Louis, Missouri, United States United States, Pennsylvania Pittsburgh, Pennsylvania, United States United States, Texas Houston, Texas, United States Less <<
NCT00953706 - Terminated(Following review of... More >> results obtained from a pre-specified 6-month analysis of Part B data the study was terminated on the basis of futility.) Less << - -
NCT02390219 - Completed - -
NCT01863238 - Completed - -
NCT03256799 Cystic Fibrosis Phase 4 Completed - United States, Alabama ... More >> University of Alabama at Birmingham Birmingham, Alabama, United States, 35233 Less <<
NCT01685801 Cystic Fibrosis Phase 2 Completed - United States, Colorado ... More >> Denver, Colorado, United States Less <<
NCT01685801 - Completed - -
NCT02823470 Cystic Fibrosis Phase 4 Terminated(Study terminated ea... More >>rly due to slow recruitment of patients.) Less << - United States, Connecticut ... More >> Hartford, Connecticut, United States United States, District of Columbia Washington, District of Columbia, United States United States, Florida Orlando, Florida, United States United States, Georgia Atlanta, Georgia, United States United States, Mississippi Jackson, Mississippi, United States United States, New Mexico Albuquerque, New Mexico, United States United States, South Carolina Columbia, South Carolina, United States Canada, Alberta Edmonton, Alberta, Canada Canada, British Columbia Victoria, British Columbia, Canada Canada, New Brunswick Saint John, New Brunswick, Canada Canada Quebec, Canada Less <<
NCT02823470 - Terminated(Study terminated ea... More >>rly due to slow recruitment of patients.) Less << - -
NCT02015507 Cystic Fibrosis Phase 1 Completed - -
NCT02544451 Cystic Fibrosis Phase 3 Active, not recruiting November 2019 -
NCT01897233 Cystic Fibrosis Phase 3 Completed - -
NCT01897233 - Completed - -
NCT01899105 Cystic Fibrosis Phase 1 Completed - United States, Florida ... More >> Daytona, Florida, United States Less <<
NCT02742519 - Terminated(Low enrollment.) - -
NCT01614470 Cystic Fibrosis Phase 3 Completed - United States, Florida ... More >> Tampa, Florida, United States United States, Georgia Atlanta, Georgia, United States United States, Illinois Chicago, Illinois, United States United States, Massachusetts Boston, Massachusetts, United States United States, Michigan Ann Arbor, Michigan, United States United States, Minnesota Minneapolis, Minnesota, United States United States, Missouri St. Louis, Missouri, United States United States, Texas Houston, Texas, United States Belgium Leuven, Belgium France Lyon, France Montpellier, France Paris, France Less <<
NCT02653027 Diabetes Cyst... More >>ic Fibrosis Less << Not Applicable Withdrawn(We couldn't find any... More >> eligible subjects for the study because most of the study population had already started on the combination therapy.) Less << - -
NCT01614470 - Completed - -
NCT02742519 Cystic Fibrosis Phase 3 Terminated(Low enrollment.) - Australia, Victoria ... More >> Parkville, Victoria, Australia Australia South Brisbane, Australia Subiaco, Australia Westmead, Australia Canada, Ontario Toronto, Ontario, Canada United Kingdom London, United Kingdom Less <<
NCT02412111 Cystic Fibrosis Phase 3 Completed - -
NCT02565914 Cystic Fibrosis Phase 3 Enrolling by invitation September 2019 -
NCT02858843 Cystic Fibrosis ... More >> Diabetes Less << Not Applicable Terminated(We were unable to e... More >>nroll eligible subjects for the study.) Less << - -
NCT03068312 Cystic Fibrosis Phase 3 Active, not recruiting December 2018 Israel ... More >> Hadassah Medical Organization Jerusalem, Israel Less <<
NCT03512119 - Recruiting October 10, 2018 France ... More >> Hôpitaux Universitaires de Strasbourg Recruiting Strasbourg, Alsace, France, 67000 Contact: Laurence KESSLER          Principal Investigator: Laurence KESSLER, MD          Centre Hospitalier Universitaire d'Angers Recruiting Angers, France, 49033 Contact: Françoise TROUSSIER          Principal Investigator: Françoise TROUSSIER          Hôpital Renée Sabran Recruiting Giens, France, 83406 Contact: Laurent MELY          Principal Investigator: Laurent MELY          Centre Hospitalier Lyon Sud Recruiting Lyon, France, 69000 Contact: Isabelle DURIEU          Principal Investigator: Isabelle DURIEU          Hôpital NORD - Assistance Publique Hôpitaux de Marseille Recruiting Marseille, France, 13385 Contact: Bérengère COLTEY          Principal Investigator: Bérengère COLTEY          Hôpital Arnaud de Villeneuve Recruiting Montpellier, France, 34295 Contact: Raphael CHIRON          Principal Investigator: Raphael CHIRON          Hôpital Robert Debré Recruiting Paris, France, 75019 Contact: Michèle GERARDIN          Principal Investigator: Michèle GERARDIN          American Memorial Hospital Recruiting Reims, France, 51092 Contact: Michel ABLEY          Principal Investigator: Michel ABELY          Clinique "Mucoviscidose" Presqu'île de Perharidy Recruiting Roscoff, France, 29684 Contact: Gilles RAULT          Principal Investigator: Gilles RAULT          Hôpital Charles Nicolle Recruiting Rouen, France, 76031 Contact: Stéphane DOMINIQUE          Principal Investigator: Stéphane DOMINIQUE          Hôpital FOCH Recruiting Suresnes, France, 92151 Contact: Florence BOUILLOUD          Principal Investigator: Florence BOUILLOUD          Hôpital Bretonneau - CHRU de Tours Recruiting Tours, France, 37044 Contact: Julie MANKIKIAN          Principal Investigator: Julie MANKIKIAN          Hôpital de Clocheville - CHRU de Tours Recruiting Tours, France, 37044 Contact: Laure COSSON          Principal Investigator: Laure COSSON Less <<
NCT02310789 - Completed - -
NCT03251911 Chronic Bronchitis Phase 4 Not yet recruiting February 1, 2021 United States, Alabama ... More >> University of Alabama at Birmingham Not yet recruiting Birmingham, Alabama, United States, 35233 Contact: Heather Hathorne, PhD    205-638-9568    hhathorne@peds.uab.edu    Contact: Ginger Reeves, BS    205-638-5970    greeves@peds.uab.edu Less <<
NCT02807415 - Recruiting June 2019 Germany ... More >> Justus-Liebig-University Recruiting Gießen, Germany, 35385 Contact: Lutz Nährlich, MD    004964198557620    lutz.naehrlich@paediat.med.uni-giessen.de    Contact: Claudia Rückes-Nilges, Dipl-Biol    004964198556945    claudia.rueckes-nilges@paediat.med.uni-giessen.de    Principal Investigator: Lutz Nährlich, MD          Sub-Investigator: Stefan Kuhnert, MD          Hannover Medical School Recruiting Hannover, Germany, 30625 Contact: Burkhard Tümmler, MD PhD    00495115322920    tuemmler.burkhard@mh-hannover.de    Contact: Christian Dopfer, MD    00495115326111    dopfer.christian@mh-hannover.de    Principal Investigator: Burkhard Tümmler, MD PhD          Sub-Investigator: Christian Dopfer, MD          University of Heidelberg Recruiting Heidelberg, Germany, 69120 Contact: Marcus Mall, MD    00496221564502    Marcus.Mall@med.uni-heidelberg.de    Contact: Simon Gräber, MD    004962215638926    Simon.Graeber@med.uni-heidelberg.de    Principal Investigator: Marcus Mall, MD          Sub-Investigator: Simon Gräber, MD          Sub-Investigator: Susanne Dittrich, MD Less <<
NCT01614457 Cystic Fibrosis Phase 3 Completed - -
NCT01946412 Cystic Fibrosis PHASE3 COMPLETED 2025-12-15 Birmingham, Alabama, United St... More >>ates|Denver, Colorado, United States|Atlanta, Georgia, United States|Indianapolis, Indiana, United States|Boston, Massachusetts, United States|Grand Rapids, Michigan, United States|Minneapolis, Minnesota, United States|Kansas city, Missouri, United States|Omaha, Nebraska, United States|Salt Lake City, Utah, United States|Richmond, Virginia, United States|Seattle, Washington, United States|Vancouver, British Columbia, Canada|Edinburgh, United Kingdom|Liverpool, United Kingdom|London, United Kingdom Less <<
NCT01946412 Cystic Fibrosis PHASE3 COMPLETED 2025-12-15 Birmingham, Alabama, United St... More >>ates|Denver, Colorado, United States|Atlanta, Georgia, United States|Indianapolis, Indiana, United States|Boston, Massachusetts, United States|Grand Rapids, Michigan, United States|Minneapolis, Minnesota, United States|Kansas city, Missouri, United States|Omaha, Nebraska, United States|Salt Lake City, Utah, United States|Richmond, Virginia, United States|Seattle, Washington, United States|Vancouver, British Columbia, Canada|Edinburgh, United Kingdom|Liverpool, United Kingdom|London, United Kingdom Less <<
NCT02725567 Cystic Fibrosis Phase 3 Recruiting June 2020 -
NCT02310789 Cystic Fibrosis Not Applicable Completed - United States, California ... More >> Stanford Hospital and Clinics Stanford, California, United States, 94305 Less <<
NCT02934698 Cystic Fibrosis PHASE3 COMPLETED 2025-05-18 -
NCT02934698 Cystic Fibrosis PHASE3 COMPLETED 2025-05-18 -
NCT02135432 Chronic Obstructive Pulmonary ... More >>Disease Less << Early Phase 1 Completed - United States, Alabama ... More >> University of Alabama at Birmingham Birmingham, Alabama, United States, 35233 Less <<
NCT03475381 - Active, not recruiting June 30, 2018 France ... More >> Adult CF center, Service de Pneumologie, Cochin Hospital Paris, France, 75006 Less <<
NCT01614457 - Completed - -
NCT02135432 - Completed - -
NCT01707290 Cystic Fibrosis PHASE3 COMPLETED 2025-04-16 Birmingham, Alabama, United St... More >>ates|Palo Alto, California, United States|Denver, Colorado, United States|Hartford, Connecticut, United States|Tampa, Florida, United States|Atlanta, Georgia, United States|Chicago, Illinois, United States|Iowa City, Iowa, United States|Lexington, Kentucky, United States|Baltimore, Maryland, United States|Boston, Massachusetts, United States|Ann Arbor, Michigan, United States|Detroit, Michigan, United States|Grand Rapids, Michigan, United States|Minneapolis, Minnesota, United States|St. Louis, Missouri, United States|Omaha, Nebraska, United States|New York, New York, United States|Syracuse, New York, United States|Valhalla, New York, United States|Chapel Hill, North Carolina, United States|Philadelphia, Pennsylvania, United States|Pittsburgh, Pennsylvania, United States|Charleston, South Carolina, United States|Nashville, Tennessee, United States|Dallas, Texas, United States|Fort Worth, Texas, United States|Houston, Texas, United States|Salt Lake City, Utah, United States|Richmond, Virginia, United States|Seattle, Washington, United States|Morgantown, West Virginia, United States|Madison, Wisconsin, United States|Leuven, Belgium|Montpellier, France|Paris, France|Belfast, United Kingdom|Edinburgh, United Kingdom Less <<
NCT01705145 Cystic Fibrosis PHASE3 COMPLETED 2025-03-14 Birmingham, Alabama, United St... More >>ates|Aurora, Colorado, United States|Atlanta, Georgia, United States|Indianapolis, Indiana, United States|Lexington, Kentucky, United States|Boston, Massachusetts, United States|Detroit, Michigan, United States|Grand Rapids, Michigan, United States|Minneapolis, Minnesota, United States|Kansas City, Missouri, United States|Omaha, Nebraska, United States|Pittsburgh, Pennsylvania, United States|Salt Lake City, Utah, United States|Charlottesville, Virginia, United States|Richmond, Virginia, United States|Seattle, Washington, United States|Vancouver, Canada|Edinburgh, United Kingdom|Liverpool, United Kingdom|London, United Kingdom Less <<
NCT03439865 Chronic Rhinosinusitis (Diagno... More >>sis) Less << Early Phase 1 Not yet recruiting July 2022 United States, Alabama ... More >> University of Alabama at Birmingham Not yet recruiting Birmingham, Alabama, United States, 35233 Contact: Lisa Clemons, MSN, RN    205-934-9714    lkclemons@uabmc.edu    Principal Investigator: Brad Woodworth, MD Less <<
NCT01784419 Cystic Fibrosis Not Applicable Unknown December 2015 United States, California ... More >> University of California, San Francisco Recruiting San Francisco, California, United States, 94143 Contact: Dennis Nielson, MD, PhD    415-476-2072    nielsond@peds.ucsf.edu    Contact: Dennis W Nielson, MD. PhD    415-476-2072    nielsond@peds.ucsf.edu    Principal Investigator: Dennis W Nielson, MD, PhD          Sub-Investigator: Meghan McGarry, MD          Sub-Investigator: Ngoc Ly, MD, MPH          Sub-Investigator: Mary Ellen Kleinhenz, MD Less <<
NCT02194881 - Completed - France ... More >> Hôpital Cochin Paris, France, 75014 Less <<
NCT01707290 Cystic Fibrosis PHASE3 COMPLETED 2025-04-16 Birmingham, Alabama, United St... More >>ates|Palo Alto, California, United States|Denver, Colorado, United States|Hartford, Connecticut, United States|Tampa, Florida, United States|Atlanta, Georgia, United States|Chicago, Illinois, United States|Iowa City, Iowa, United States|Lexington, Kentucky, United States|Baltimore, Maryland, United States|Boston, Massachusetts, United States|Ann Arbor, Michigan, United States|Detroit, Michigan, United States|Grand Rapids, Michigan, United States|Minneapolis, Minnesota, United States|St. Louis, Missouri, United States|Omaha, Nebraska, United States|New York, New York, United States|Syracuse, New York, United States|Valhalla, New York, United States|Chapel Hill, North Carolina, United States|Philadelphia, Pennsylvania, United States|Pittsburgh, Pennsylvania, United States|Charleston, South Carolina, United States|Nashville, Tennessee, United States|Dallas, Texas, United States|Fort Worth, Texas, United States|Houston, Texas, United States|Salt Lake City, Utah, United States|Richmond, Virginia, United States|Seattle, Washington, United States|Morgantown, West Virginia, United States|Madison, Wisconsin, United States|Leuven, Belgium|Montpellier, France|Paris, France|Belfast, United Kingdom|Edinburgh, United Kingdom Less <<
NCT03085485 Chronic Obstructive Pulmonary ... More >>Disease Chronic Bronchitis Less << Phase 2 Recruiting April 2019 United States, Alabama ... More >> UAB Lung Health Center Recruiting Birmingham, Alabama, United States, 35294 Contact: Mark T Dransfield, MD    205-934-5555       Principal Investigator: Mark T Dransfield, MD Less <<
NCT01705145 Cystic Fibrosis PHASE3 COMPLETED 2025-03-14 Birmingham, Alabama, United St... More >>ates|Aurora, Colorado, United States|Atlanta, Georgia, United States|Indianapolis, Indiana, United States|Lexington, Kentucky, United States|Boston, Massachusetts, United States|Detroit, Michigan, United States|Grand Rapids, Michigan, United States|Minneapolis, Minnesota, United States|Kansas City, Missouri, United States|Omaha, Nebraska, United States|Pittsburgh, Pennsylvania, United States|Salt Lake City, Utah, United States|Charlottesville, Virginia, United States|Richmond, Virginia, United States|Seattle, Washington, United States|Vancouver, Canada|Edinburgh, United Kingdom|Liverpool, United Kingdom|London, United Kingdom Less <<
NCT03277196 Cystic Fibrosis Phase 3 Recruiting June 7, 2021 -
NCT03783286 Cystic Fibrosis COMPLETED 2021-12-30 Children's Hospital of Philade... More >>lphia, Philadelphia, Pennsylvania, 19146, United States Less <<
NCT04066751 Chronic Obstructive Pulmonary ... More >>Disease Less << PHASE2 WITHDRAWN 2024-10-31 -

Protocol

Bio Calculators
Preparing Stock Solutions 1mg 5mg 10mg

1 mM

5 mM

10 mM

2.55mL

0.51mL

0.25mL

12.74mL

2.55mL

1.27mL

25.48mL

5.10mL

2.55mL

Dissolving Methods
Please choose the appropriate dissolution scheme according to your animal administration guide.For the following dissolution schemes, clear stock solution should be prepared according to in vitro experiments, and then cosolvent should be added in turn:

in order to ensure the reliability of the experimental results, the clarified stock solution can be properly preserved according to the storage conditions; The working fluid for in vivo experiment is recommended to be prepared now and used on the same day;

The percentage shown in front of the following solvent refers to the volume ratio of the solvent in the final solution; If precipitation or precipitation occurs in the preparation process, it can be assisted by heating and/or ultrasound.
Protocol 1
Protocol 2

References

 

Historical Records

Categories