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NCT02473445 · ClinicalTrials.gov registry record · Phase 2
A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease
A Phase 2 study, sponsored by Amgen.
- Terminated
- Registry status
- Phase 2
- Development phase
- 22
- Enrollment target
NCT02473445: Clinical Trial Phase 2 study, sponsored by Amgen.
NCT02473445 is a Phase 2 study that was terminated before completion, run by Amgen. The registered enrollment target is 22 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (83% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02473445, a Phase 2 study, was terminated before completion, sponsored by Amgen.
- TERMINATED
- Registry status
- Phase 2
- Development phase
- 22 participants
- Enrollment target
Study Summary
A long-term extension study to assess the safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) in children with inherited mitochondrial diseases who previously enrolled into study RP103-MITO-001 (NCT02023866).
Primary Outcome
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
Interventions
- DRUG Cysteamine Bitartrate
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 22 participants |
| Start Date | 2015-05-19 |
| Est. Completion | 2017-03-06 |
| Phase | Phase 2 |
Why NCT02473445 stopped before completion
NCT02473445 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 22 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (83% lower).
The record links to 0 conditions, and to 1 intervention - of which Cysteamine Bitartrate is the first listed.
NCT02473445 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT02473445 about?
NCT02473445 is a clinical study titled "A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease". A long-term extension study to assess the safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) in children with inherited mitochondrial diseases who previously enrolled into study RP103-MITO-001 (NCT02023866).
What is the current status of trial NCT02473445?
This trial is currently terminated. It is a Phase 2 study. The enrollment target is 22 participants. The study started on 2015-05-19. Estimated completion is 2017-03-06.
What interventions are being tested in trial NCT02473445?
The interventions under investigation include: Cysteamine Bitartrate (DRUG).
Who is sponsoring clinical trial NCT02473445?
This trial is sponsored by Amgen, which has 558 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT02473445's enrollment target sits among peer trials
22 1790th of 2000 higher than 207 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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