Drugs / Cysteamine
last change Aug 2026 re-read 2 minutes ago

Cysteamine

Developed for
cystinosis · hyperpigmentation of the skin · asthma · corneal deposit · cystic fibrosis · fatty liver disease · HIV infectious disease · Huntington disease
+6 more · inborn mitochondrial metabolism disorder · major depressive disorder · pneumonia · schizoaffective disorder · schizophrenia · skin atrophy
Investigated by
Amgen · National Eye Institute (NEI) · Raptor Pharmaceuticals Corp. · Augusta University · Australian and New Zealand Intensive Care Research Centre · Berry Consultants
+27 more · Chang Gung Memorial Hospital · Children's Hospital Medical Center, Cincinnati · China Medical University Hospital · Cystic Fibrosis Trust · FDA Office of Orphan Products Development · Global Coalition for Adaptive Research · Hasanuddin University · Icahn School of Medicine at Mount Sinai · Intensive Care National Audit & Research Centre · Joel Lavine · Leadiant Biosciences, Inc. · Medical Research Institute of New Zealand · Mylan Laboratories · NHS Grampian · Nat Intensive Care Surveillance - MORU · National University Hospital, Singapore · RECORDATI GROUP · Scientis Pharma SA · St. Marianna University School of Medicine · The Eye Center and The Eye Foundation for Research in Ophthalmology · UMC Utrecht · Unity Health · University Hospital, Angers · University of Aberdeen · University of California, San Diego · University of Huddersfield · University of Pittsburgh Medical Center

Regulatory milestones approvals, filings & regulatory actions · 3 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
Refused (EMA) EU (EMA) Dropcys 2016-06-24 europa.eu
Approved EU (EMA) Procysbi 2013-09-05 europa.eu
Approved US (FDA) CYSTARAN 2012-10-02 fda.gov

Trials 19 · a red edge is where a trial was stopped

PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 12 trials
Phase 1 NCT03883984 Jun 2019 → Feb 2021 asthma Children's Hospital Medical Center, Cincinnati Completed No outcome recorded
Phase 1 NCT00001736 May 1998 → Mar 2001 cystinosis National Eye Institute (NEI) Completed No outcome recorded
Phase 1/22 trials
Phase 1/2 NCT02212431 Aug 2014 → Mar 2015 cystic fibrosis University of Aberdeen Completed No outcome recorded
Phase 1/2 NCT00799578 Oct 2008 → Jun 2009 fatty liver disease Joel Lavine Completed No outcome recorded
Phase 24 trials
Phase 2 NCT07477288 Mar → Sep 2025 skin atrophy Hasanuddin University Completed No outcome recorded
Phase 2 NCT02023866 May 2014 → Oct 2016 inborn mitochondrial metabolism disorder Amgen Completed No outcome recorded
Phase 2 NCT00001213 Apr 1986 → Jul 2013 cystinosis National Eye Institute (NEI) Completed No outcome recorded
Phase 2 NCT00002110 HIV infectious disease Mylan Laboratories Completed No outcome recorded
Phase 2/31 trial
Phase 2/3 NCT02101957 Oct 2010 → Jul 2015 Huntington disease University Hospital, Angers Unknown No outcome recorded
Phase 34 trials
Phase 3 NCT04125927 Sep 2020 → Dec 2022 cystinosis RECORDATI GROUP Completed No outcome recorded
Phase 3 NCT05969587 Nov 2019 → Sep 2020 hyperpigmentation of the skin China Medical University Hospital Completed No outcome recorded
Phase 3 NCT02735707 Apr 2016 → Feb 2026 overdue pneumonia UMC Utrecht Recruiting No outcome recorded
Phase 3 NCT01197378 Aug 2010 → Jun 2017 cystinosis Amgen Completed No outcome recorded
Phase not applicable6 trials
NCT05206318 Dec 2021 → Jun 2022 hyperpigmentation of the skin Chang Gung Memorial Hospital Unknown No outcome recorded
NCT01432561 Sep → Dec 2011 nephropathic cystinosis University of California, San Diego Completed No outcome recorded
NCT01139125 Sep 2009 → Jun 2012 schizoaffective disorder, schizophrenia Augusta University Terminated No outcome recorded
NCT00715559 Jul 2008 → May 2009 major depressive disorder Icahn School of Medicine at Mount Sinai Terminated No outcome recorded
NCT02766855 Jan 2004 → Dec 2012 corneal deposit, nephropathic cystinosis The Eye Center and The Eye Foundation for Research in Ophthalmology Completed No outcome recorded
NCT00010426 Dec 1999 → Feb 2001 cystinosis FDA Office of Orphan Products Development Completed No outcome recorded
Also used as a comparator or background therapy in 3 trials
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1/21 trial
Phase 1/2 NCT05994534 Comparator Oct 2023 → Aug 2026 overdue cystinosis Nacuity Pharmaceuticals, Inc. Active not recruiting No outcome recorded
Phase 21 trial
Phase 2 NCT03000348 Comparator Dec 2016 → Apr 2018 cystic fibrosis NovaBiotics Ltd. Completed No outcome recorded
Phase not applicable1 trial
NCT06278948 Comparator Oct 2023 → Jun 2024 hyperpigmentation of the skin Cosmetique Active International Unknown No outcome recorded

Evidence & citations 13 cited values

Every value below carries the sentence it was read from. 24 sources stand behind the page.

FieldValueCited text
Known as Cysteamine “Multicentric Trial of the Treatment of Huntington's Disease by Cysteamine (RP103)” NCT02101957
19

Cysteamine 5%” NCT06278948

NCT00002110

NCT05206318

NCT00001213

NCT03000348

NCT05969587

NCT03883984

NCT07477288

NCT02766855

NCT01139125

NCT02023866

NCT00001736

NCT00799578

NCT01197378

NCT01432561

NCT05994534

NCT02735707

NCT02212431

NCT00715559

Known as b-mercaptoethylamine “Cysteamine (b-mercaptoethylamine) hydrochloride is the stable amino-thiol that acts as an antioxidant.” NCT05969587
Known as Cyspera “5% cysteamine cream (Cyspera®) was acquired from Scientis APAC Pte. Ltd. (Singapore, Singapore).” NCT05969587
Known as Cystagon “The current study drug, cysteamine bitartrate (Cystagon), is FDA approved for the treatment nephropathic cystinosis” NCT00715559
2

“The current marketed therapy is Cystagon® (cysteamine bitartrate immediate release)” NCT01197378

“(Cystagon) Cysteamine Bitartrate” NCT01139125

Known as Cysteamine bitartrate “The current study drug, cysteamine bitartrate (Cystagon), is FDA approved for the treatment nephropathic cystinosis” NCT00715559
3

“To determine the long-term effects of delayed-release cysteamine bitartrate (DR-CYS) based on our previous work” PMID 24948347 Jun 2014

Cysteamine bitartrate readily traverses cellular membranes and is a potent antioxidant.” PMID 21395631 Mar 2011

“(Cystagon) Cysteamine BitartrateNCT01139125

Known as cysteamine hydrochloride ClinicalTrials.gov intervention name — accepted as the source's own label NCT00010426
Known as DR-CYS “To determine the long-term effects of delayed-release cysteamine bitartrate (DR-CYS) based on our previous work” PMID 24948347 Jun 2014
Known as EC-cysteamine “received twice-daily EC-cysteamine for 24 weeks.” PMID 21395631 Mar 2011
Known as Mercaptamine ClinicalTrials.gov intervention name — accepted as the source's own label NCT04125927
Known as Mercaptoethylamine “A Phase II/III Study of Cysteamine (Mercaptoethylamine) and Zidovudine for the Treatment of HIV Disease” NCT00002110
Known as RP103 “Multicentric Trial of the Treatment of Huntington's Disease by Cysteamine (RP103)” NCT02101957
1

“cysteamine bitartrate delayed-release capsules (RP103)” NCT02023866

Action Inhibit “Cysteamine is a thiolic compound that inhibit tyrosinase and peroxidase activity of melanocytes” NCT05969587
Route Topical “cysteamine 0.5% topical solution” NCT00001213