Tezacaftor (VX-661) Clinical Trials

Size	Price	Qty
5mg	USD 270
50mg	USD 970

Clinical Trials

Numerous clinical trials spanning Phase 1 through Phase 4 evaluate tezacaftor (VX-661) as a monotherapy and in combination regimens, such as elexacaftor/tezacaftor/ivacaftor, primarily for cystic fibrosis, CFTR gene mutations, and related respiratory complications. These studies, which range in status from completed to active not recruiting and currently recruiting, assess safety and therapeutic efficacy across diverse patient cohorts. Major sponsors include Vertex Pharmaceuticals Incorporated alongside academic institutions such as Ospedale San Raffaele and Charité University.

NCT Number	Recruitment	Conditions	Sponsor/Collaborators	Start Date	Phases
NCT07148739	RECRUITING	Cystic Fibrosis (CF)	Arkansas Children's Hospital Research Institute	2025-06-10	PHASE4
NCT05422222	ACTIVE_NOT_RECRUITING	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2022-06-21	PHASE3
NCT05844449	ENROLLING_BY_INVITATION	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2023-08-11	PHASE3
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
NCT07314229	RECRUITING	Cystic Fibrosis (CF); Mucoviscidosis	University Hospital, Lille	2025-12-17
NCT05937815	RECRUITING	Cystic Fibrosis	University Hospital, Bordeaux	2021-09-13
NCT06154447	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2023-12-12	PHASE1
NCT07349394	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2026-01-12	PHASE1
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
NCT04840862	COMPLETED	Drug Drug Interaction	University of Southern California	2022-05-09	PHASE4
NCT06299709	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2024-03-13	PHASE1
NCT06299696	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2024-03-26	PHASE1
NCT04545515	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2021-01-11	PHASE3
NCT05437120	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2022-07-22	PHASE1
NCT04362761	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2020-05-04	PHASE3
NCT05111145	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2022-01-14	PHASE3
NCT05535959	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2022-09-15	PHASE1
NCT04969224	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2021-10-12	PHASE3
NCT04599465	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2021-01-15	PHASE3
NCT04537793	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2020-11-19	PHASE3
NCT04684823	COMPLETED	Cystic Fibrosis; Adherence, Medication	The University of Texas Health Science Center at San Antonio	2021-01-15
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
NCT04105972	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2019-10-03	PHASE3
NCT02565914	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2015-08	PHASE3
NCT03559062	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2018-05-17	PHASE3
NCT02953314	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2016-11	PHASE3
NCT03150719	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2017-05-24	PHASE3
NCT02730208	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2016-09	PHASE2
NCT02412111	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2015-06	PHASE3
NCT03029455	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2016-11	PHASE1
NCT02508207	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2016-02	PHASE2
NCT02392234	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2015-03	PHASE3
NCT02347657	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2015-01	PHASE3
NCT03486236	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2016-07-20	PHASE1
NCT02516410	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2015-08	PHASE3
NCT02070744	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2014-03	PHASE2
NCT02412111	Completed	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2015-06	Phase 3
NCT01531673	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2012-02	PHASE2
NCT02015507	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2014-01	PHASE1
NCT02015507	Completed	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2014-01	Phase 1

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

Check the Tezacaftor (VX-661) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Tezacaftor selectively binds to the misfolded F508del cystic fibrosis transmembrane conductance regulator (CFTR) protein, facilitating proper intracellular processing and trafficking of the channel to the cell surface. By increasing plasma membrane expression of functional CFTR proteins, it restores transepithelial chloride transport, thereby alleviating airway obstruction and pulmonary exacerbations in cystic fibrosis patients.

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>).