Ivacaftor (VX-770) Clinical Trials

Size	Price	Qty
5mg	USD 170
50mg	USD 970

Clinical Trials

Ivacaftor has been evaluated across numerous clinical trials primarily investigating cystic fibrosis—including F508del-CFTR genetic subpopulations—as well as related conditions such as chronic obstructive pulmonary disease, chronic pancreatitis, and cystic fibrosis-related diabetes. Spanning Phase 1 through Phase 4 across active, recruiting, and completed statuses, research is sponsored by pharmaceutical developers like Vertex Pharmaceuticals alongside academic medical centers such as the University of Alabama at Birmingham and Sheffield Teaching Hospitals. These Phase 2 and Phase 3 protocols assess the efficacy, safety, and pharmacokinetics of ivacaftor as monotherapy and combination therapy in pediatric and adult populations.

NCT Number	Recruitment	Conditions	Sponsor/Collaborators	Start Date	Phases
NCT07148739	RECRUITING	Cystic Fibrosis (CF)	Arkansas Children's Hospital Research Institute	2025-06-10	PHASE4
NCT06457451	RECRUITING	Cystic Fibrosis	University Hospital, Tours	2024-11-25
NCT06460506	ACTIVE_NOT_RECRUITING	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2024-11-21	PHASE3
NCT05331183	ACTIVE_NOT_RECRUITING	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2022-11-23	PHASE3
NCT03439865	RECRUITING	Chronic Rhinosinusitis (Diagnosis)	University of Alabama at Birmingham	2019-05-02	EARLY_PHASE1
NCT07314229	RECRUITING	Cystic Fibrosis (CF); Mucoviscidosis	University Hospital, Lille	2025-12-17
NCT06237335	ACTIVE_NOT_RECRUITING	Cystic Fibrosis	ReCode Therapeutics	2024-02-01	PHASE2
NCT05937815	RECRUITING	Cystic Fibrosis	University Hospital, Bordeaux	2021-09-13
NCT05153317	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2022-01-17	PHASE3
NCT05818319	RECRUITING	Cystic Fibrosis (CF); CFTR Gene Mutation	University of Aarhus	2023-06-01
NCT05882357	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2023-06-27	PHASE3
NCT04581408	COMPLETED	Long QT Syndrome	Istituto Auxologico Italiano	2021-06-15	PHASE2
NCT04066751	WITHDRAWN	Chronic Obstructive Pulmonary Disease	University of Alabama at Birmingham	2021-12-01	PHASE2
NCT04840862	COMPLETED	Drug Drug Interaction	University of Southern California	2022-05-09	PHASE4
NCT06331000	Not yet recruiting	Cystic Fibrosis	University Hospital Strasbourg France	2024-03	--
NCT05968612	COMPLETED	Healthy Volunteer	Qanatpharma Canada LTD	2023-11-17	PHASE1
NCT03277196	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2017-08-16	PHASE3
NCT04235140	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2020-02-24	PHASE3
NCT04545515	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2021-01-11	PHASE3
NCT04362761	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2020-05-04	PHASE3
NCT05111145	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2022-01-14	PHASE3
NCT04135495	COMPLETED	Cystic Fibrosis	Eloxx Pharmaceuticals, Inc.	2019-11-25	PHASE2
NCT03085485	COMPLETED	Chronic Obstructive Pulmonary Disease; Chronic Bronchitis	University of Alabama at Birmingham	2017-03-16	PHASE2
NCT04969224	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2021-10-12	PHASE3
NCT04599465	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2021-01-15	PHASE3
NCT05519020	Recruiting	Cystic Fibrosis	Sheffield Teaching Hospitals NHS Foundation Trust	2022-07-27	--
NCT02725567	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2016-06-02	PHASE3
NCT04537793	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2020-11-19	PHASE3
NCT04126473	COMPLETED	Cystic Fibrosis	Eloxx Pharmaceuticals, Inc.	2019-11-05	PHASE2
NCT03251911	WITHDRAWN	Chronic Bronchitis	University of Alabama at Birmingham	2020-01-01	PHASE4
NCT04684823	COMPLETED	Cystic Fibrosis; Adherence, Medication	The University of Texas Health Science Center at San Antonio	2021-01-15
NCT03601637	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2018-09-07	PHASE3
NCT04353817	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2020-06-19	PHASE3
NCT04006873	COMPLETED	Cystic Fibrosis	Nottingham University Hospitals NHS Trust	2019-09-03	PHASE2
NCT03537651	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2018-04-25	PHASE3
NCT03625466	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2018-08-10	PHASE2
NCT04105972	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2019-10-03	PHASE3
NCT03956589	TERMINATED	Cystic Fibrosis	University Hospital, Antwerp	2018-10-01	PHASE4
NCT04254705	Withdrawn	Cystic Fibrosis	Universitaire Ziekenhuizen KU Leuven\|Vertex Pharmaceuticals Incorporated\|KU Leuven\|University of Lisbon	2020-03-01	Not Applicable
NCT03624101	TERMINATED	Cystic Fibrosis	University of Alabama at Birmingham	2018-12-01	PHASE4
NCT03125395	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2017-05-12	PHASE3
NCT02565914	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2015-08	PHASE3
NCT03559062	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2018-05-17	PHASE3
NCT03068312	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2017-07-18	PHASE3
NCT03256968	COMPLETED	Cystic Fibrosis	University of Alabama at Birmingham	2017-01-27	PHASE4
NCT02871778	COMPLETED	Primary Ciliary Dyskinesia	Parion Sciences	2016-08	PHASE2
NCT02953314	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2016-11	PHASE3
NCT03150719	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2017-05-24	PHASE3
NCT02544451	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2015-08	PHASE3
NCT02730208	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2016-09	PHASE2
NCT02858843	TERMINATED	Cystic Fibrosis; Diabetes	Massachusetts General Hospital	2016-08-01
NCT02934698	COMPLETED	Cystic Fibrosis	Medical University of South Carolina	2016-11-01	PHASE3
NCT02653027	WITHDRAWN	Diabetes; Cystic Fibrosis	Massachusetts General Hospital	2018-01-01
NCT03061331	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2017-01-31	PHASE2
NCT02797132	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2016-05	PHASE3
NCT02875366	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2016-09	PHASE4
NCT02412111	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2015-06	PHASE3
NCT03045523	COMPLETED	Cystic Fibrosis	Lakefront Biotherapeutics NV	2017-01	PHASE2
NCT03029455	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2016-11	PHASE1
NCT02823470	TERMINATED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2016-06	PHASE4
NCT02742519	TERMINATED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2016-05	PHASE3
NCT03256799	COMPLETED	Cystic Fibrosis	University of Alabama at Birmingham	2017-03-17	PHASE4
NCT02508207	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2016-02	PHASE2
NCT02724527	UNKNOWN	Cystic Fibrosis	Nivalis Therapeutics, Inc.	2016-04	PHASE2
NCT03085485	Completed	Chronic Obstructive Pulmonary Disease\|Chronic Bronchitis	University of Alabama at Birmingham\|National Heart Lung and Blood Institute (NHLBI)\|Vertex Pharmaceuticals Incorporated	2017-03-16	Phase 2
NCT02392234	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2015-03	PHASE3
NCT02347657	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2015-01	PHASE3
NCT02589236	COMPLETED	Cystic Fibrosis	Nivalis Therapeutics, Inc.	2015-11	PHASE2
NCT02390219	COMPLETED	Cystic Fibrosis; Advanced Lung Disease	Vertex Pharmaceuticals Incorporated	2015-03	PHASE3
NCT03486236	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2016-07-20	PHASE1
NCT02514473	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2015-07	PHASE3
NCT02310789	COMPLETED	Cystic Fibrosis	Richard Barry Moss	2015-07-31
NCT02516410	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2015-08	PHASE3
NCT02070744	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2014-03	PHASE2
NCT01707290	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2013-02	PHASE3
NCT01931839	COMPLETED	Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation	Vertex Pharmaceuticals Incorporated	2013-10	PHASE3
NCT01946412	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2013-12	PHASE3
NCT01784419	COMPLETED	Cystic Fibrosis	University of California, San Francisco	2013-10
NCT02135432	COMPLETED	Chronic Obstructive Pulmonary Disease	University of Alabama at Birmingham	2015-05	EARLY_PHASE1
NCT01897233	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2013-07	PHASE3
NCT01937325	UNKNOWN	Cystic Fibrosis	The Alfred	2014-02	PHASE4
NCT01117012	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2010-07	PHASE3
NCT01807949	COMPLETED	Cystic Fibrosis, Homozygous for the F508del CFTR Mutation	Vertex Pharmaceuticals Incorporated	2013-04	PHASE3
NCT01225211	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2010-10	PHASE2
NCT01685801	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2012-09	PHASE2
NCT01807923	COMPLETED	Cystic Fibrosis, Homozygous for the F508del CFTR Mutation	Vertex Pharmaceuticals Incorporated	2013-05	PHASE3
NCT01531673	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2012-02	PHASE2
NCT01910415	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2013-06	PHASE1
NCT01705145	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2013-01	PHASE3
NCT02015507	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2014-01	PHASE1
NCT01888393	COMPLETED	Hepatic Impairment; Healthy	Vertex Pharmaceuticals Incorporated	2013-06	PHASE1
NCT01614457	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2012-07	PHASE3
NCT01614470	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2012-07	PHASE3
NCT01768663	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2013-01	PHASE1
NCT01899105	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2013-07	PHASE1
NCT01161537	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2010-10	PHASE2
NCT01262352	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2011-01	PHASE2
NCT01216046	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2010-10	PHASE1
NCT01208285	COMPLETED	In Development for Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2010-09	PHASE1
NCT00909727	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2009-08	PHASE3
NCT01153542	COMPLETED	In Development for Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2010-06	PHASE1
NCT00953706	TERMINATED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2009-09	PHASE2
NCT00909532	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2009-06	PHASE3
NCT01060566	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2010-02	PHASE1
NCT01018368	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2009-11	PHASE1
NCT00966602	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2009-09	PHASE1
NCT00457821	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2007-05	PHASE2

(data from https://clinicaltrials.gov, updated on 2026-06-02)

Check the Ivacaftor (VX-770) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Ivacaftor selectively binds to and potentiates the cystic fibrosis transmembrane conductance regulator (CFTR) channel, increasing its open-state probability to enhance transmembrane chloride ion transport while reducing excessive sodium and fluid absorption to preserve apical surface hydration and improve ciliary beating. This restoration of epithelial fluid homeostasis directly addresses the underlying channelopathy, facilitating airway clearance and demonstrating clinical relevance in the treatment of cystic fibrosis and chronic obstructive pulmonary disease.

Appendix RUO and cGMP Quality Standards

Quality Dimension	RUO (Research Use Only)	cGMP (Current Good Manufacturing Practice)
Clinical Applicability	Prohibited in human clinical trials or medical diagnostics.	Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status	Non-regulated grade; exempt from drug manufacturing laws.	Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment	Unclassified analytical or research laboratories.	Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control	Basic purity and activity testing.	Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation	Basic equipment calibration; no process validation required.	Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance	Vendor self-declared without required formal QMS.	Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact	High risk of IND rejection if used as a critical raw material.	Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).