VX-809 (Lumacaftor) Clinical Trials

Size	Price	Qty
5mg	USD 170
50mg	USD 970

Clinical Trials

Numerous clinical trials spanning Phase 1 through Phase 4 evaluate VX-809 (lumacaftor), focusing primarily on cystic fibrosis in F508del-CFTR populations—including pediatric patients and individuals with related glucose intolerance or diabetes—as well as non-respiratory indications such as Long QT syndrome and heart failure with reduced ejection fraction. Supported by both commercial entities and academic medical centers, these studies encompass completed, recruiting, terminated, and withdrawn recruitment statuses to comprehensively assess lumacaftor's therapeutic potential across metabolic, cardiac, and respiratory conditions.

NCT Number	Recruitment	Conditions	Sponsor/Collaborators	Start Date	Phases
NCT07695090	RECRUITING	Heart Failure With Reduced Ejection Fraction	Qanatpharma AG	2026-07	PHASE2
NCT04581408	COMPLETED	Long QT Syndrome	Istituto Auxologico Italiano	2021-06-15	PHASE2
NCT05968612	COMPLETED	Healthy Volunteer	Qanatpharma Canada LTD	2023-11-17	PHASE1
NCT04235140	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2020-02-24	PHASE3
NCT03601637	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2018-09-07	PHASE3
NCT03625466	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2018-08-10	PHASE2
NCT03956589	TERMINATED	Cystic Fibrosis	University Hospital, Antwerp	2018-10-01	PHASE4
NCT03125395	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2017-05-12	PHASE3
NCT02544451	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2015-08	PHASE3
NCT02858843	TERMINATED	Cystic Fibrosis; Diabetes	Massachusetts General Hospital	2016-08-01
NCT02653027	WITHDRAWN	Diabetes; Cystic Fibrosis	Massachusetts General Hospital	2018-01-01
NCT03061331	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2017-01-31	PHASE2
NCT02875366	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2016-09	PHASE4
NCT02797132	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2016-05	PHASE3
NCT02823470	TERMINATED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2016-06	PHASE4
NCT02589236	COMPLETED	Cystic Fibrosis	Nivalis Therapeutics, Inc.	2015-11	PHASE2
NCT02390219	COMPLETED	Cystic Fibrosis; Advanced Lung Disease	Vertex Pharmaceuticals Incorporated	2015-03	PHASE3
NCT02514473	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2015-07	PHASE3
NCT01931839	COMPLETED	Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation	Vertex Pharmaceuticals Incorporated	2013-10	PHASE3
NCT03512119	Completed	Cystic Fibrosis Homozygous for Phe 508 Del CFTR\|Glucose Intolerance or Newly Diagnosis Diabetes	University Hospital Strasbourg France	2016-02-11	--
NCT02589236	Completed	Cystic Fibrosis	Nivalis Therapeutics Inc.\|Medidata Solutions	2015-11	Phase 2
NCT01897233	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2013-07	PHASE3
NCT02514473	Completed	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2015-07	Phase 3
NCT01807923	COMPLETED	Cystic Fibrosis, Homozygous for the F508del CFTR Mutation	Vertex Pharmaceuticals Incorporated	2013-05	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
NCT01910415	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2013-06	PHASE1
NCT01888393	COMPLETED	Hepatic Impairment; Healthy	Vertex Pharmaceuticals Incorporated	2013-06	PHASE1
NCT01768663	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2013-01	PHASE1
NCT01899105	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2013-07	PHASE1
NCT01899105	Completed	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2013-07	Phase 1
NCT01216046	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2010-10	PHASE1
NCT00865904	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2009-03	PHASE2
NCT00966602	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2009-09	PHASE1

(data from https://clinicaltrials.gov, updated on 2026-07-17)

Check the VX-809 (Lumacaftor) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

VX-809 (lumacaftor) directly targets and binds to mutant F508del-CFTR protein, promoting its conformational stability and enhancing cellular maturation and trafficking to the plasma membrane. The presence of functional CFTR channels at the cell surface restores transmembrane chloride transport, mitigating hyper-viscous mucus accumulation to alleviate airway obstruction in cystic fibrosis patients evaluated in clinical trials.

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>).