Vanzacaftor (VX-121) Clinical Trials

Size	Price	Qty
1mg	USD 197
5mg	USD 347

Clinical Trials

Several clinical trials evaluate vanzacaftor (VX-121) exclusively for the management of cystic fibrosis, encompassing Phase 1 pharmacokinetic and safety studies, Phase 3 evaluations, and observational research. Industry sponsor Vertex Pharmaceuticals Incorporated leads interventional trials assessing vanzacaftor monotherapy and combination regimens, while academic sponsors like Charite University, Berlin, Germany contribute biomarker studies. Recruitment statuses across this portfolio include completed studies, actively recruiting protocols, and trials enrolling by invitation.

NCT Number	Recruitment	Conditions	Sponsor/Collaborators	Start Date	Phases
NCT05844449	ENROLLING_BY_INVITATION	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2023-08-11	PHASE3
NCT07349394	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2026-01-12	PHASE1
NCT06299709	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2024-03-13	PHASE1
NCT06299696	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2024-03-26	PHASE1
NCT05867147	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2023-04-26	PHASE1

(data from https://clinicaltrials.gov, updated on 2026-04-29)

Check the Vanzacaftor (VX-121) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Vanzacaftor (VX-121) functions as an orally active cystic fibrosis transmembrane conductance regulator (CFTR) corrector that binds the CFTR protein to facilitate its cellular processing and trafficking to the plasma membrane. By promoting proper protein folding and surface localization, vanzacaftor elevates epithelial chloride ion transport, thereby mitigating mucosal hyperviscosity and restoration of airway clearance in cystic fibrosis.

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>).