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Cysteamine Bitartrate

Phase 3

Cystinosis | Small molecule | Rare Disease |Amgen Inc.|Last Updated: Dec 27, 2024

Success Probability

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment60

FDA Designations

No designations recorded

Clinical trial landscape

Cysteamine Bitartrate · 2 trials · 2 indications

Phase 3 1Phase 2 1
NCT01197378Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103)Cystinosis
COMPLETED60 Analytics
PHASE3COMPLETED
Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103)
CystinosisUnlock trial analytics

Study Endpoints

Primary Endpoints

Number of Participants With Treatment-emergent Adverse Events
From first dose of study drug to 7 days after the last dose; median duration of treatment was 1461 days.

Drug-related adverse events (AEs) are AEs the investigator assessed as having relation to drug of 'possibly', 'probably' or 'definitely'. The severity of AEs was categorized according to the Common Terminology Criteria for Adverse Events (CTCAE), Version 3.0 as follows: * Mild (Grade 1): experience is minor and does not cause significant discomfort to subject or change in activities of daily living (ADL); subject is aware of symptoms but symptoms are easily tolerated; * Moderate (Grade 2): experience is an inconvenience or concern to the subject and causes interference with ADL, but the subject is able to continue with ADL. * Severe (Grade 3): experience significantly interferes with ADL and the subject is incapacitated and/or unable to continue with ADL * Life-threatening (Grade 4): experience that, in the view of the Investigator, places the subject at immediate risk of death from the event as it occurred.

Change From Baseline in Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Sections I-IV
Baseline through Week 24

The NPMDS evaluates the progression of mitochondrial disease in pediatric patients in 4 domains: I - Current Function (vision, hearing, communication, feeding, and mobility) with scores ranging from 0 to 21; II -System Specific Involvement (seizures, encephalopathy, bleeding diathesis or coagulation defects, gastrointestinal, endocrine, respiratory, cardiovascular, renal, liver, and blood) with scores ranging from 0 to 30. III - Current Clinical Assessment (growth and development over past 6 months, vision, strabismus and eye movement, myopathy, ataxia, pyramidal, extrapyramidal, and neuropathy) with scores ranging from 0 to 28; and IV - Quality of Life with scores ranging from 0 to 25. For sections I-III, higher scores reflect more severe disease. For Section IV, a higher score reflects a lower quality of life.

Secondary Endpoints

Trough Plasma Cysteamine Concentration
Day 1 (predose) and Month 6, Years 1, 1.5, 2, 3, 4 and 5 at 0.5 hours post-dose
White Blood Cell Cystine Concentration
Day 1 (predose) and Month 6, Years 1, 1.5, 2, 3, 4 and 5 at 0.5 hours post-dose
Change From Baseline in Glutathione
Baseline and Weeks 4, 8, 12, 16, 20, 24
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Cysteamine BitartrateEXPERIMENTALCysteamine bitartrate delayed-release capsules were administered twice daily for up to 96 months.
Cysteamine Bitartrate Delayed-releaseEXPERIMENTALCysteamine bitartrate delayed-release capsules were administered twice daily following a dose-escalation design with a progressive weekly dose increase over the first 6 weeks. The starting dose was 0.2 g/m²/day, up to a maximum dose of 1.3 g/m²/day. Participants remained on their highest tolerated dose until Week 24.

Interventions

NameTypeDescription
Cysteamine Bitartrate Delayed-release CapsulesDRUGParticipants who entered the trial from the RP103-03 study continued treatment with cysteamine bitartrate every 12 hours at the last dose level prescribed during their participation in that study. Participants not entering the trial from Study RP103-03 were started on twice a day administration of cysteamine bitartrate at a total daily RP103 dose of 70% of their pre-study total daily stable Cystagon® dose.
Cysteamine BitartrateDRUGCysteamine Bitartrate Delayed-release capsules
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Eligibility Criteria

Age Range1 Year to N/A
SexALL
Healthy VolunteersNo
Study Sites10

Inclusion Criteria: * Male and female subjects must have completed the last visit of Study RP103-03 and be willing to continue with RP103 treatment. OR for patients who did not complete the RP103-03 study: * Male and female subjects must have cystinosis. * Subjects must be on a stable dose of Cys...

Countries:United StatesFranceNetherlands
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Frequently asked questions about Cysteamine Bitartrate

What is Cysteamine Bitartrate used for?

Cysteamine Bitartrate is used for cystinosis, a rare disease. It is an investigational small molecule being developed by Amgen Inc. (AMGN) and is currently in Phase 3 clinical development. The drug is intended to treat patients with cystinosis, though it is not yet approved.

Who makes Cysteamine Bitartrate?

Cysteamine Bitartrate is being developed by Amgen Inc., a biopharmaceutical company traded on NASDAQ under the ticker AMGN. The drug is currently in Phase 3 clinical development for the treatment of cystinosis, a rare disease.

What phase is Cysteamine Bitartrate in?

Cysteamine Bitartrate is in Phase 3 clinical development. It is an investigational drug for cystinosis, a rare disease, and is not yet approved. The Phase 3 trial has been completed, with 60 participants enrolled.

What clinical trials is Cysteamine Bitartrate in?

Cysteamine Bitartrate has one completed Phase 3 trial, NCT01197378, titled "Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103)". The trial enrolled 60 participants with cystinosis and was conducted in the United States, France, and the Netherlands.

Is Cysteamine Bitartrate the same as RP103?

Yes, Cysteamine Bitartrate is also known as RP103. The Phase 3 clinical trial NCT01197378 refers to the drug as "Cysteamine Bitartrate Delayed-release Capsules (RP103)". This alternative name is used in clinical research for the same investigational drug.