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.
...

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)

Phase 3
Completed
Conditions
Interventions
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

and more 3 locations

A Study of the Dosing, Efficacy, and Safety of Oral Cysteamine in Adult Patients With Cystic Fibrosis Exacerbations

Phase 2
Completed
Conditions
Interventions
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

and more 14 locations

Efficacy of Topical Cysteamine in Nephropathic Cyctinosis

Not Applicable
Completed
Conditions
Interventions
First Posted Date
2016-05-10
Last Posted Date
2016-05-10
Lead Sponsor
The Eye Center and The Eye Foundation for Research in Ophthalmology
Target Recruit Count
26
Registration Number
NCT02766855

Randomized, Embedded, Multifactorial Adaptive Platform Trial for Community- Acquired Pneumonia

Phase 3
Recruiting
Conditions
Interventions
Other: No systemic corticosteroid
Other: No antiviral agent for COVID-19
Other: No immune modulation for COVID-19
Drug: Standard course macrolide
Other: No antiviral agent for influenza
Drug: Local standard venous thromboprophylaxis
Other: No immunoglobulin
Other: No simvastatin
Other: No cysteamine
Other: No Immune Modulator for Influenza
Other: No vitamin C
Other: No antiplatelet
Procedure: Clinician-preferred mechanical ventilation strategy
Other: No renin-angiotensin system inhibitor
Other: No endothelial modulator
Drug: Ceftriaxone
Drug: Moxifloxacin or Levofloxacin
Drug: Extended course macrolide
Drug: Fixed-duration Hydrocortisone
Drug: Five-days oseltamivir
Drug: Lopinavir / Ritonavir
Biological: Convalescent plasma
Drug: Angiotensin converting enzyme inhibitor
Drug: Nirmatrelvir/ritonavir
Drug: Therapeutic dose anticoagulation
Procedure: Protocolised mechanical ventilation strategy
Drug: Interferon beta-1a
Drug: Vitamin C
Drug: Simvastatin
Drug: Aspirin
Drug: Eritoran
Drug: Cysteamine
Drug: Imatinib
Drug: Shock-dependent hydrocortisone
Drug: Ten-days oseltamivir
Drug: P2Y12 inhibitor
Drug: Angiotensin Receptor Blockers
Drug: Conventional low dose thromboprophylaxis
Biological: Delayed administration of convalescent plasma
Drug: Piperacillin-tazobactam
Drug: Hydroxychloroquine
Drug: Anakinra
Drug: Apremilast
Drug: Baricitinib
Drug: Remdesivir
Drug: Fixed-duration higher dose Hydrocortisone
Drug: Hydroxychloroquine + lopinavir/ritonavir
Drug: Intermediate dose thromboprophylaxis
Drug: Nirmatrelvir/ritonavir + remdesivir
Drug: ARB + DMX-200
Drug: Ceftaroline
Drug: Baloxavir Marboxil
Drug: Tocilizumab
Drug: Amoxicillin-clavulanate
Drug: Fixed-duration dexamethasone
Drug: Five-days oseltamivir + baloxavir marboxil
Drug: Continuation of therapeutic dose anticoagulation
Drug: Ivermectin
Drug: Sarilumab
Drug: 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

and more 405 locations

First Study of Oral Cysteamine in Cystic Fibrosis

Phase 1
Completed
Conditions
Interventions
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)

Phase 1
Completed
Conditions
Interventions
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

Recruiting
Conditions
Interventions
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

Phase 1
Completed
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

Phase 2
Completed
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

and more 3 locations

Cysteamine Eye Drops to Treat Corneal Crystals in Cystinosis

Phase 2
Completed
Conditions
Interventions
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

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