Nesolicaftor (PTI-428) Clinical Trials

Size	Price	Qty
1mg	USD 207

Clinical Trials

Several clinical trials sponsored by Proteostasis Therapeutics Inc. have evaluated the CFTR modulator nesolicaftor (PTI-428) across Phase I and Phase II development stages. These studies assessed the safety, tolerability, pharmacokinetics, and therapeutic profile of the compound in healthy adult volunteers and patients with cystic fibrosis. All of these clinical trials have reached completed status, providing foundational data for the treatment of cystic fibrosis.

NCT Number	Recruitment	Conditions	Sponsor/Collaborators	Start Date	Phases
NCT03500263	Completed	Cystic Fibrosis	Proteostasis Therapeutics Inc.	2018-01-30	Phase 1\|Phase 2
NCT03251092	Completed	Healthy Volunteer - Complete\|Cystic Fibrosis - Complete	Proteostasis Therapeutics Inc.	2017-07-17	Phase 1\|Phase 2

(data from https://clinicaltrials.gov, updated on 2024-05-22)

Check the Nesolicaftor (PTI-428) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Nesolicaftor selectively functions as a cystic fibrosis transmembrane conductance regulator (CFTR) amplifier, increasing CFTR protein expression to restore cellular chloride ion transport across epithelial cell membranes. By enhancing functional ion channel abundance at the cell surface, this compound addresses the fundamental transport defect in cystic fibrosis, directly supporting its clinical investigation for this therapeutic indication.

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>).