Cysteamine
- Generic Name
- Cysteamine
- Brand Names
- Cystagon, Cystaran, Procysbi, Cystadrops
- Drug Type
- Small Molecule
- Chemical Formula
- C2H7NS
- CAS Number
- 60-23-1
- Unique Ingredient Identifier
- 5UX2SD1KE2
- Background
Cystinosis is a rare disease caused by mutations in the CTNS gene that encodes for cystinosin, a protein responsible for transporting cystine out of the cell lysosome. A defect in cystinosin function is followed by cystine accumulation throughout the body, especially the eyes and kidneys.
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- Indication
The bitartrate salt of cysteamine is used for the oral treatment of nephropathic cystinosis and cystinuria in adults and children ≥6 years old. The hydrochloride salt, used in eye drop preparations, is indicated for the treatment of corneal cystine crystal accumulation in patients with cystinosis.
- Associated Conditions
- Cystinosis, Nephropathic, Corneal cystine crystal accumulation
- Associated Therapies
- -
Cystadrops in Pediatric Cystinosis Patients From Six Months to Less Than Two Years Old (SCOB2)
- First Posted Date
- 2019-10-14
- Last Posted Date
- 2024-02-05
- Lead Sponsor
- Recordati Rare Diseases
- Target Recruit Count
- 5
- Registration Number
- NCT04125927
- Locations
- 🇧🇪
UZ Leuven, Leuven, Belgium
🇫🇷Hôpital Necker-Enfants Malades, Paris, France
🇬🇧Great Ormond Street Hospital, London, United Kingdom
A Study of the Dosing, Efficacy, and Safety of Oral Cysteamine in Adult Patients With Cystic Fibrosis Exacerbations
- First Posted Date
- 2016-12-22
- Last Posted Date
- 2021-04-14
- Lead Sponsor
- NovaBiotics Ltd.
- Target Recruit Count
- 91
- Registration Number
- NCT03000348
- Locations
- 🇺🇸
West Virginia University, Morgantown, West Virginia, United States
🇺🇸Albany Medical College, Albany, New York, United States
🇬🇧Aberdeen Royal Infirmary, Aberdeen, Scotland, United Kingdom
Efficacy of Topical Cysteamine in Nephropathic Cyctinosis
- Conditions
- Interventions
- First Posted Date
- 2016-05-10
- Last Posted Date
- 2016-05-10
- Target Recruit Count
- 26
- Registration Number
- NCT02766855
Randomized, Embedded, Multifactorial Adaptive Platform Trial for Community- Acquired Pneumonia
- Conditions
- Interventions
- Other: No systemic corticosteroidOther: No antiviral agent for COVID-19Other: No immune modulation for COVID-19Drug: Standard course macrolideOther: No antiviral agent for influenzaDrug: Local standard venous thromboprophylaxisOther: No immunoglobulinOther: No simvastatinOther: No cysteamineOther: No Immune Modulator for InfluenzaOther: No vitamin COther: No antiplateletProcedure: Clinician-preferred mechanical ventilation strategyOther: No renin-angiotensin system inhibitorOther: No endothelial modulatorDrug: CeftriaxoneDrug: Moxifloxacin or LevofloxacinDrug: Extended course macrolideDrug: Fixed-duration HydrocortisoneDrug: Five-days oseltamivirDrug: Lopinavir / RitonavirBiological: Convalescent plasmaDrug: Angiotensin converting enzyme inhibitorDrug: Nirmatrelvir/ritonavirDrug: Therapeutic dose anticoagulationProcedure: Protocolised mechanical ventilation strategyDrug: Interferon beta-1aDrug: Vitamin CDrug: SimvastatinDrug: AspirinDrug: EritoranDrug: CysteamineDrug: ImatinibDrug: Shock-dependent hydrocortisoneDrug: Ten-days oseltamivirDrug: P2Y12 inhibitorDrug: Angiotensin Receptor BlockersDrug: Conventional low dose thromboprophylaxisBiological: Delayed administration of convalescent plasmaDrug: Piperacillin-tazobactamDrug: HydroxychloroquineDrug: AnakinraDrug: ApremilastDrug: BaricitinibDrug: RemdesivirDrug: Fixed-duration higher dose HydrocortisoneDrug: Hydroxychloroquine + lopinavir/ritonavirDrug: Intermediate dose thromboprophylaxisDrug: Nirmatrelvir/ritonavir + remdesivirDrug: ARB + DMX-200Drug: CeftarolineDrug: Baloxavir MarboxilDrug: TocilizumabDrug: Amoxicillin-clavulanateDrug: Fixed-duration dexamethasoneDrug: Five-days oseltamivir + baloxavir marboxilDrug: Continuation of therapeutic dose anticoagulationDrug: IvermectinDrug: SarilumabDrug: Ten-days oseltamivir + baloxavir marboxil
- First Posted Date
- 2016-04-13
- Last Posted Date
- 2024-07-12
- Lead Sponsor
- UMC Utrecht
- Target Recruit Count
- 20000
- Registration Number
- NCT02735707
- Locations
- 🇨🇦
Toronto Western Hospital, Toronto, Ontario, Canada
🇺🇸University of Illinois Health, Chicago, Illinois, United States
🇦🇺Bankstown-Lidcombe Hospital, Bankstown, New South Wales, Australia
First Study of Oral Cysteamine in Cystic Fibrosis
- First Posted Date
- 2014-08-08
- Last Posted Date
- 2015-11-05
- Lead Sponsor
- University of Aberdeen
- Target Recruit Count
- 10
- Registration Number
- NCT02212431
- Locations
- 🇬🇧
Aberdeen Royal Infirmary, Aberdeen, United Kingdom
A Preliminary Study to Evaluate Cysteamine Therapy in Human Subjects With Non-Alcoholic Steatohepatitis (NASH)
- First Posted Date
- 2008-12-01
- Last Posted Date
- 2014-01-31
- Lead Sponsor
- Joel Lavine
- Target Recruit Count
- 13
- Registration Number
- NCT00799578
- Locations
- 🇺🇸
University of California, San Diego School of Medicine General Clinic Research Center, San Diego, California, United States
Use of Cysteamine in the Treatment of Cystinosis
- First Posted Date
- 2006-08-02
- Last Posted Date
- 2024-12-06
- Lead Sponsor
- National Human Genome Research Institute (NHGRI)
- Target Recruit Count
- 330
- Registration Number
- NCT00359684
- Locations
- 🇺🇸
National Institutes of Health Clinical Center, Bethesda, Maryland, United States
New Cysteamine Eye Drops Formulation to Treat Corneal Crystals in Cystinosis
- Conditions
- First Posted Date
- 2002-12-10
- Last Posted Date
- 2008-03-04
- Lead Sponsor
- National Eye Institute (NEI)
- Target Recruit Count
- 51
- Registration Number
- NCT00001736
- Locations
- 🇺🇸
National Eye Institute (NEI), Bethesda, Maryland, United States
A Phase II/III Study of Cysteamine (Mercaptoethylamine) and Zidovudine for the Treatment of HIV Disease
- Conditions
- First Posted Date
- 2001-08-31
- Last Posted Date
- 2005-06-24
- Lead Sponsor
- Mylan Laboratories
- Target Recruit Count
- 300
- Registration Number
- NCT00002110
- Locations
- 🇺🇸
Independent Research Nurses Inc, Cranston, Rhode Island, United States
🇺🇸Infectious Disease Research Institute Inc, Tampa, Florida, United States
🇺🇸Tulane Univ Med School, New Orleans, Louisiana, United States
Cysteamine Eye Drops to Treat Corneal Crystals in Cystinosis
- First Posted Date
- 1999-11-04
- Last Posted Date
- 2014-07-22
- Lead Sponsor
- National Eye Institute (NEI)
- Target Recruit Count
- 328
- Registration Number
- NCT00001213
- Locations
- 🇺🇸
National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, United States
Drug Development Updates
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