Clinical Trials

Numerous clinical trials spanning Phase 1, Phase 2, and Phase 3 have evaluated cysteamine across diverse indications, including cystinosis, nephropathic cystinosis, melasma, skin aging, HIV infection, and nonalcoholic fatty liver disease. Sponsored by pharmaceutical companies, academic institutions, and research centers, these studies address both systemic metabolic dysfunction and localized dermatological conditions. Across this research portfolio, trial recruitment statuses include completed, recruiting, active, terminated, and withdrawn studies.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT06990984 NOT_YET_RECRUITING
Leigh Syndrome
Thiogenesis Therapeutics, Inc.
 2026-10 PHASE2
NCT05994534 ACTIVE_NOT_RECRUITING
Cystinosis
Nacuity Pharmaceuticals, Inc.
 2023-10-29 PHASE1; PHASE2
NCT02735707 RECRUITING
Community-acquired Pneumonia, Influenza, COVID-19
UMC Utrecht
 2016-04-11 PHASE3
NCT07477288 COMPLETED
Skin Aging
Hasanuddin University
 2025-03-12 PHASE2
NCT06278948 UNKNOWN
Melasma
Cosmetique Active International
 2023-10-06
NCT05656833 UNKNOWN
Melasma
UnionDerm
 2022-10-28
NCT05206318 UNKNOWN
Hyperpigmentation; Postinflammatory
Chang Gung Memorial Hospital
 2021-12-10
NCT04262895 WITHDRAWN
Mild to Moderate TBI; Posttraumatic Stress Disorder
VA Office of Research and Development
 2021-10-01 PHASE1; PHASE2
NCT03883984 COMPLETED
Asthma
Children's Hospital Medical Center, Cincinnati
 2019-06-18 PHASE1
NCT05969587 COMPLETED
Melasma
China Medical University Hospital
 2019-11-28 PHASE3
NCT03000348 COMPLETED
Cystic Fibrosis
NovaBiotics Ltd.
 2016-12 PHASE2
NCT01197378 COMPLETED
Cystinosis
Amgen
 2010-08-27 PHASE3
NCT02473445 TERMINATED
Mitochondrial Diseases
Amgen
 2015-05-19 PHASE2
NCT01744782 COMPLETED
Cystinosis
Amgen
 2012-12-20 PHASE3
NCT02012114 COMPLETED
Cystinosis
Hospices Civils de Lyon
 2011-12
NCT02023866 COMPLETED
Inherited Mitochondrial Disease, Including Leigh Syndrome
Amgen
 2014-05 PHASE2
NCT02101957 UNKNOWN
Huntington's Disease
University Hospital, Angers
 2010-10 PHASE2; PHASE3
NCT02212431 COMPLETED
Cystic Fibrosis
University of Aberdeen
 2014-08 PHASE1; PHASE2
NCT01529268 COMPLETED
Nonalcoholic Fatty Liver Disease (NAFLD)
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
 2012-06 PHASE2; PHASE3
NCT01733316 COMPLETED
Cystinosis
Amgen
 2013-01-31 PHASE3
NCT00001213 COMPLETED
Cystinosis
National Eye Institute (NEI)
 1986-04 PHASE2
NCT02766855 COMPLETED
Nephropathic Cyctinosis; Corneal Cystine Crystals
The Eye Center and The Eye Foundation for Research in Ophthalmology
 2004-01
NCT01139125 TERMINATED
Schizophrenia; Schizoaffective
Augusta University
 2009-09
NCT01432561 COMPLETED
Cystinosis; Nephropathic Cystinosis
University of California, San Diego
 2011-09
NCT01000961 COMPLETED
Cystinosis
Amgen
 2010-06 PHASE3
NCT00872729 COMPLETED
Cystinosis
Amgen
 2009-05 PHASE1; PHASE2
NCT00799578 COMPLETED
Fatty Liver
Joel Lavine
 2008-10 PHASE1; PHASE2
NCT00715559 TERMINATED
Major Depressive Disorder
Icahn School of Medicine at Mount Sinai
 2008-07
NCT00002110 COMPLETED
HIV Infections
Mylan Laboratories
 PHASE2
NCT00001736 COMPLETED
Cystinosis
National Eye Institute (NEI)
 1998-05 PHASE1
NCT00010426 COMPLETED
Cystinosis
FDA Office of Orphan Products Development
 1999-12

(data from https://clinicaltrials.gov, updated on 2026-07-24)

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

Compliance for Clinical Use

Mechanism and Biochemical Profile

Cysteamine HCl functions as an autophagy-stimulatory aminothiol that directly reacts with lysosomal cystine to form cysteine and a mixed cysteamine-cysteine disulfide. This chemical conversion facilitates cystine transport out of lysosomes via functional amino acid carriers, thereby preventing intracellular crystal accumulation and cellular damage in conditions such as nephropathic cystinosis.

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.