Clinical Trials

Multiple clinical trials evaluate penicillamine across diverse therapeutic areas, including Wilson's disease, lead poisoning, scleroderma, and solid tumors such as glioblastoma and recurrent head and neck cancer. Spanning Early Phase 1 through Phase 4, these studies are supported by academic medical centers, hospital networks, government organizations, and pharmaceutical sponsors. The overall landscape encompasses a range of recruitment statuses, including upcoming, actively recruiting, completed, and withdrawn protocols.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT07465718 NOT_YET_RECRUITING
Wilson's Disease
Orphalan
 2026-08 PHASE3
NCT06945081 RECRUITING
Wilson Disease; D-Penicillamine; Effect of D-penicilline on Cutaneous Elastity of Wilson's Patient
Centre Hospitalier Universitaire de Saint Etienne
 2025-11-28
NCT03957720 UNKNOWN
Wilson's Disease
Second Affiliated Hospital, Zhejiang University, School of Medicine
 2019-03-15 EARLY_PHASE1
NCT06103617 UNKNOWN
Head and Neck Cancer; Recurrent Cancer
Nanfang Hospital, Southern Medical University
 2023-11-15 PHASE2
NCT03539952 COMPLETED
Wilson Disease
Orphalan
 2018-09-03 PHASE3
NCT02426905 UNKNOWN
Wilson Disease
Univar BV
 2016-01 PHASE4
NCT00552630 Withdrawn
Lead Poisoning|Vitamin D Deficiency
FDA Office of Orphan Products Development|Bezoloven Inc.
 2007-09 Phase 2|Phase 3
NCT00003751 COMPLETED
Brain and Central Nervous System Tumors
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
 1999-03 PHASE2
NCT00552630 WITHDRAWN
Lead Poisoning; Vitamin D Deficiency
FDA Office of Orphan Products Development
 2007-09 PHASE2; PHASE3

(data from https://clinicaltrials.gov, updated on 2026-08-05)

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

Compliance for Clinical Use

Mechanism and Biochemical Profile

Penicillamine functions as a thiol-containing chelating agent that directly binds free copper and cystine to form stable, soluble coordination complexes, thereby promoting renal clearance and selectively suppressing T-lymphocyte function. By depleting systemic heavy metal ions and dampening autoimmune responses, this chelation mechanism reduces pathological metal accumulation and tissue damage, establishing direct clinical relevance in trials for Wilson's disease, lead poisoning, and copper-depletion cancer therapies.

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.