Icenticaftor (QBW251) Clinical Trials

Size	Price	Qty
1mg	USD 459
5mg	USD 1,379

Clinical Trials

Multiple Phase 1 and Phase 2 clinical trials, sponsored primarily by Novartis Pharmaceuticals in collaboration with the Innovative Medicines Initiative, have evaluated the efficacy, safety, and pharmacokinetics of icenticaftor. These studies targeted respiratory and metabolic conditions, specifically chronic obstructive pulmonary disease, bronchiectasis, cystic fibrosis, and hepatic impairment. Although several trials evaluating systemic pharmacokinetics and therapeutic efficacy were successfully completed, other studies in bronchiectasis and cystic fibrosis were terminated.

NCT Number	Recruitment	Conditions	Sponsor/Collaborators	Start Date	Phases
NCT04396366	TERMINATED	Bronchiectasis	Novartis Pharmaceuticals	2021-02-02	PHASE2
NCT04587622	COMPLETED	Hepatic Failure	Novartis Pharmaceuticals	2020-10-30	PHASE1
NCT04268823	TERMINATED	Chronic Obstructive Pulmonary Disease	Novartis Pharmaceuticals	2020-09-10	PHASE2
NCT04072887	COMPLETED	Pulmonary Disease, Chronic Obstructive	Novartis Pharmaceuticals	2019-09-12	PHASE2
NCT04396366	Terminated	Bronchiectasis	Novartis Pharmaceuticals\|Innovative Medicines Initiative\|Novartis	2021-02-02	Phase 2
NCT04587622	Completed	Hepatic Failure	Novartis Pharmaceuticals\|Novartis	2020-10-30	Phase 1
NCT04268823	Terminated	Chronic Obstructive Pulmonary Disease	Novartis Pharmaceuticals\|Novartis	2020-09-10	Phase 2
NCT02449018	COMPLETED	Chronic Obstructive Pulmonary Disease, COPD	Novartis Pharmaceuticals	2015-04-30	PHASE2
NCT02190604	TERMINATED	Cystic Fibrosis	Novartis Pharmaceuticals	2012-07-31	PHASE1; PHASE2
NCT02449018	Completed	Chronic Obstructive Pulmonary Disease COPD	Novartis Pharmaceuticals\|Novartis	2015-04-30	Phase 2
NCT02190604	Terminated	Cystic Fibrosis	Novartis Pharmaceuticals\|Novartis	2012-07-31	Phase 1\|Phase 2

(data from https://clinicaltrials.gov, updated on 2025-09-26)

Check the Icenticaftor (QBW251) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Icenticaftor acts as a selective oral potentiator of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, binding to the channel to enhance gating kinetics and restore transepithelial chloride ion secretion across mucosal membranes. By increasing anion conductance and improving mucosal hydration, this compound promotes mucociliary clearance, mitigating airway dysfunction in clinical conditions such as chronic obstructive pulmonary disease, bronchiectasis, and 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>).