Clinical Trials

Multiple clinical trials spanning Phase I, Phase II, and Phase III evaluate the small-molecule agent deutivacaftor (VX-561) primarily for cystic fibrosis, alongside pharmacokinetic evaluations in healthy volunteers. Sponsored by commercial industry partners such as Vertex Pharmaceuticals and Concert Pharmaceuticals as well as academic institutions like Charité University, these studies assess safety, pharmacokinetics, and therapeutic efficacy across monotherapy and combination regimens. Their recruitment statuses encompass completed, terminated, recruiting, active not recruiting, and enrolling by invitation.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
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
NCT06154447 COMPLETED
Cystic Fibrosis
Vertex Pharmaceuticals Incorporated
 2023-12-12 PHASE1
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
NCT05437120 COMPLETED
Cystic Fibrosis
Vertex Pharmaceuticals Incorporated
 2022-07-22 PHASE1
NCT05535959 COMPLETED
Cystic Fibrosis
Vertex Pharmaceuticals Incorporated
 2022-09-15 PHASE1
NCT03911713 COMPLETED
Cystic Fibrosis
Vertex Pharmaceuticals Incorporated
 2019-04-17 PHASE2
NCT03912233 COMPLETED
Cystic Fibrosis
Vertex Pharmaceuticals Incorporated
 2019-04-30 PHASE2
NCT03911713 Completed
Cystic Fibrosis
Vertex Pharmaceuticals Incorporated
 2019-04-17 Phase 2
NCT03227471 COMPLETED
Cystic Fibrosis
Vertex Pharmaceuticals Incorporated
 2017-01-23 PHASE1; PHASE2
NCT03224351 COMPLETED
Cystic Fibrosis
Vertex Pharmaceuticals Incorporated
 2017-08-08 PHASE2
NCT02971839 TERMINATED
Cystic Fibrosis
Vertex Pharmaceuticals Incorporated
 2016-12 PHASE2
NCT02680249 COMPLETED
Healthy
Concert Pharmaceuticals
 2016-02 PHASE1
NCT02599792 COMPLETED
Healthy
Concert Pharmaceuticals
 2015-11 PHASE1
NCT02680249 Completed
Healthy
Concert Pharmaceuticals
 2016-02 Phase 1

(data from https://clinicaltrials.gov, updated on 2026-05-28)

Check the Deutivacaftor (VX-561, Ivacaftor-D9) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Deutivacaftor functions as a potent cystic fibrosis transmembrane conductance regulator (CFTR) modulator that directly binds to CFTR ion channels, facilitating channel gating and enhancing transepithelial chloride ion transport. By restoring functional ion secretion and surface liquid hydration across cell membranes, this targeted potentiation directly addresses the cellular defects causing cystic fibrosis, providing the biological foundation for its clinical study 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>).

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Note: Technical data last updated: Sep 1, 2026.