Clinical Trials

Multiple clinical trials spanning Phase 1 through Phase 3, early exploratory research, and expanded access protocols evaluate verdiperstat in healthy volunteers and patients with neurodegenerative conditions, including Multiple System Atrophy (cerebellar and parkinsonian variants), Amyotrophic Lateral Sclerosis (ALS), and Semantic Dementia. Featuring both active and completed recruitment statuses, these studies encompass Phase 3 trials and platform regimens for ALS. Sponsorship includes pharmaceutical companies such as Biohaven Pharmaceuticals and AstraZeneca, alongside academic and federal institutions including Brigham and Women's Hospital and the National Institutes of Health.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT04297683 ACTIVE_NOT_RECRUITING
Amyotrophic Lateral Sclerosis
Merit E. Cudkowicz, MD
2020-06-14 PHASE2; PHASE3
NCT05184569 ACTIVE_NOT_RECRUITING
Semantic Dementia
Peter Ljubenkov, MD
2022-04-14 PHASE1
NCT04436510 COMPLETED
Amyotrophic Lateral Sclerosis
Merit E. Cudkowicz, MD
2020-07-28 PHASE2; PHASE3
NCT05184569 Recruiting
Semantic Dementia
Peter Ljubenkov MD|National Institutes of Health (NIH)|Alzheimer''s Association|National Institute on Aging (NIA)|University of California San Francisco
2022-04-14 Phase 1
NCT04616456 COMPLETED
Multiple System Atrophy; Multiple System Atrophy, Cerebellar Variant; Multiple System Atrophy, Parkinson Variant (Disorder); Multiple System Atrophy (MSA) With Orthostatic Hypotension
Brigham and Women's Hospital
2020-12-30 EARLY_PHASE1
NCT03952806 COMPLETED
Multiple System Atrophy
Biohaven Pharmaceuticals, Inc.
2019-07-29 PHASE3
NCT00914303 Completed
Healthy
AstraZeneca
2009-06 Phase 1

(data from https://clinicaltrials.gov, updated on 2026-07-21)

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

Compliance for Clinical Use

Mechanism and Biochemical Profile

Verdiperstat is a selective, orally active, and irreversible inhibitor of myeloperoxidase (MPO) with an IC50 of 630 nM that targets the active site of the enzyme to block the generation of hypochlorous acid and reactive oxygen species. By suppressing toxic microglial activation and reducing oxidative stress within neural tissue, this inhibition protects neurons from chronic neuroinflammation and slows disease progression in neurodegenerative conditions such as Multiple System Atrophy and Amyotrophic Lateral Sclerosis.

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>).

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