Clinical Trials

Multiple completed clinical trials sponsored by Vertex Pharmaceuticals Incorporated have evaluated inaxaplin across various manifestations of renal dysfunction. These include a Phase IIb open-label study assessing its safety and efficacy in proteinuric APOL1-mediated kidney disease, as well as Phase I trials evaluating single-dose safety and pharmacokinetics in severe renal impairment, focal segmental glomerulosclerosis, and hepatic impairment. Together, these studies establish key safety, pharmacokinetic, and therapeutic parameters for inaxaplin.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT06794996 COMPLETED
Proteinuric Kidney Disease
Vertex Pharmaceuticals Incorporated
 2025-02-04 PHASE2
NCT06529796 COMPLETED
Focal Segmental Glomerulosclerosis (FSGS)
Vertex Pharmaceuticals Incorporated
 2024-08-02 PHASE1
NCT05865171 COMPLETED
Renal Impairment
Vertex Pharmaceuticals Incorporated
 2023-06-12 PHASE1

(data from https://clinicaltrials.gov, updated on 2026-08-20)

Check the Inaxaplin product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Inaxaplin is an orally active small molecule that directly binds to apolipoprotein L1 (APOL1), thereby inhibiting downstream cytotoxic APOL1 channel activity and cellular toxicity. By preventing APOL1-mediated podocyte injury and preserving glomerular barrier function, inaxaplin reduces proteinuria and mitigates disease progression in patients with APOL1-associated proteinuric kidney disease and focal segmental glomerulosclerosis.

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.