Cysteamine

Generic Name: Cysteamine

Brand Names: Cystagon, Cystaran, Procysbi, Cystadrops

Drug Type: Small Molecule

Chemical Formula: C_₂H_₇NS

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.

Several preparations of cysteamine exist for the treatment of cystinosis manifestations, some in capsule form, and others in ophthalmic solution form. In particular, cystine deposits on the eye can cause significant discomfort throughout the day and require frequent treatment with eye drops, typically every waking hour.

On August 25th 2020, the first ophthalmic solution for cystinosis requiring only 4 daily treatments was granted FDA approval. Cysteamine eye drops are a practical and effective option for those affected by ocular cystinosis. Marketed by Recordati Rare Diseases Inc., CYSTADROPS® reduce the burden of multiple frequent medications normally administered to those with cystinosis.

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

Clinical Trials

Ivabradine Versus Beta-blockers in the Treatment of Inappropriate Sinus Tachycardia

Phase 3

Completed

Conditions: Inappropriate Sinus Tachycardia

Interventions: Drug: Beta blocker
Drug: Ivabradine

First Posted Date: 2012-08-06

Last Posted Date: 2016-09-30

Lead Sponsor: Policlinico Casilino ASL RMB

Target Recruit Count: 20

Registration Number: NCT01657136

Locations: 🇮🇹
Policlinico Casilino, Rome, Italy

Study in Healthy Adults to Determine the Effect That Food Has on the Absorption and Delivery of the Drug Cystagon™

Not Applicable

Completed

Conditions: Nephropathic Cystinosis
Cystinosis

Interventions: Drug: Cysteamine bitartrate

First Posted Date: 2011-09-13

Last Posted Date: 2013-10-09

Lead Sponsor: University of California, San Diego

Target Recruit Count: 8

Registration Number: NCT01432561

Locations: 🇺🇸
University of California, San Diego Center for Clinical Research Services (CCR), La Jolla, California, United States

Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103)

Phase 3

Completed

Conditions: Cystinosis

Interventions: Drug: Cysteamine Bitartrate Delayed-release Capsules

First Posted Date: 2010-09-09

Last Posted Date: 2024-12-27

Lead Sponsor: Amgen

Target Recruit Count: 60

Registration Number: NCT01197378

Locations: 🇺🇸
Stanford University Medical School, Stanford, California, United States
🇫🇷
Robert Debre Hospital, Paris, France
🇺🇸
Texas Children's Hospital/Baylor University, Houston, Texas, United States

and more 7 locations

An Open Trial of Cysteamine Treatment in Schizophrenia

Not Applicable

Terminated

Conditions: Schizoaffective
Schizophrenia

Interventions: Drug: (Cystagon) Cysteamine Bitartrate

First Posted Date: 2010-06-08

Last Posted Date: 2014-11-20

Lead Sponsor: Augusta University

Target Recruit Count: 3

Registration Number: NCT01139125

Locations: 🇺🇸
Georgia Regents University- Dept of Psychiatry, Augusta, Georgia, United States
🇺🇸
Georgia Health Sciences University - Dept of Psychiatry, Augusta, Georgia, United States

Flecainide for Catecholaminergic Polymorphic Ventricular Tachycardia

Not Applicable

Completed

Conditions: Catecholaminergic Polymorphic Ventricular Tachycardia

Interventions: Drug: Flecainide Acetate
Drug: Placebo
Drug: Beta blocker

First Posted Date: 2010-05-05

Last Posted Date: 2017-06-14

Lead Sponsor: Vanderbilt University Medical Center

Target Recruit Count: 14

Registration Number: NCT01117454

Locations: 🇺🇸
University of California Los Angeles, Los Angeles, California, United States
🇺🇸
Nationwide Children's Hospital, Columbus, Ohio, United States
🇺🇸
MetroHealth Medical Center, Cleveland, Ohio, United States

and more 7 locations

Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis

Phase 3

Completed

Conditions: Cystinosis

Interventions: Drug: Cysteamine Bitartrate Delayed-release Capsules (RP103)
Drug: Cystagon® (Cysteamine Bitartrate)

First Posted Date: 2009-10-23

Last Posted Date: 2024-12-19

Lead Sponsor: Amgen

Target Recruit Count: 43

Registration Number: NCT01000961

Locations: 🇫🇷
Villeneuve-Lapeyronie Hospital, Montpellier, France
🇺🇸
Stanford University Medical School, Stanford, California, United States
🇳🇱
Radboud University Nijmegen Medical Center, Nijmegen, Netherlands

and more 5 locations

Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis

Phase 1

Completed

Conditions: Cystinosis

Interventions: Drug: Cystagon®
Drug: RP103

First Posted Date: 2009-03-31

Last Posted Date: 2024-12-19

Lead Sponsor: Amgen

Target Recruit Count: 9

Registration Number: NCT00872729

Locations: 🇺🇸
University of California San Diego Medical Center, San Diego, California, United States

Collaborative Systematic Overview of Randomised Controlled Trials of Beta-Blockers in the Treatment of Heart Failure

Conditions: Heart Failure

Interventions: Other: Placebo
Drug: Beta blocker

First Posted Date: 2009-01-30

Last Posted Date: 2012-05-09

Lead Sponsor: Royal Brompton & Harefield NHS Foundation Trust

Target Recruit Count: 18240

Registration Number: NCT00832442

Locations: 🇬🇧
Clinical Trials & Evaluation Unit, Royal Brompton Hospital, London, United Kingdom
🇬🇧
Centre for Statistics in Medicine, University of Oxford, Oxford, United Kingdom