Trial · NCT02473445
A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease
NCT02473445, Phase 2, Terminated, Amgen; results first posted 2018-05-11
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease. Trials, record NCT02473445. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02473445
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease
- NCT number
- NCT02473445
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 22 participants, actual
- Lead sponsor
- Amgen
- Conditions
- Mitochondrial Diseases
- Interventions
- Cysteamine Bitartrate
- First posted
- 2015-06-16
- Last updated
- 2024-12-27
- Results first posted
- 2018-05-11
- Limitations and caveats
- The study was closed prematurely due to lack of efficacy demonstrated in base study RP103-MITO-001 and no efficacy analyses were conducted.
Source ClinicalTrials.gov
Registered outcomes3
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change in Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Score | Baseline, every 3 months and Study Exit (up to 24 Months) | 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; 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 | Change Over Time in Two of the Most Pre-eminent Symptoms | Baseline, every 3 months and Study Exit (up to 24 Months) | The two pre-eminent symptoms previously identified in study RP103-MITO-001 were to be continued to be assessed during the extension study. Symptoms included myopathy, dystonia, ataxia, retarded motor development, reduced activities of daily living, and vision. |
| Secondary | Change Over Time in Pharmacodynamic Biomarkers | Baseline, every 3 months and Study Exit (up to 24 Months) | Change from baseline in glutathione, glutathione disulfide, and lactate analyses were not performed as the study was prematurely terminated. |
Source ClinicalTrials.gov
Outcomes3
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change in Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Score | Baseline, every 3 months and Study Exit (up to 24 Months) | The study was closed prematurely due to lack of efficacy demonstrated in base study RP103-MITO-001. As a result, only a limited amount of data was collected for patients that were enrolled prior to termination. The decision was made that the data were not complete enough, and no analyses were conducted. | ||
| SECONDARY | Change Over Time in Two of the Most Pre-eminent Symptoms | Baseline, every 3 months and Study Exit (up to 24 Months) | The study was closed prematurely due to lack of efficacy demonstrated in base study RP103-MITO-001. As a result, only a limited amount of data was collected for patients that were enrolled prior to termination. The decision was made that the data were not complete enough, and no analyses were conducted. | ||
| SECONDARY | Change Over Time in Pharmacodynamic Biomarkers | Baseline, every 3 months and Study Exit (up to 24 Months) | The study was closed prematurely due to lack of efficacy demonstrated in base study RP103-MITO-001. As a result, only a limited amount of data was collected for patients that were enrolled prior to termination. The decision was made that the data were not complete enough, and no analyses were conducted. |
Source ClinicalTrials.gov
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Cysteamine Bitartrate Delayed-release | 22 | |
| Overall Study | COMPLETED | Cysteamine Bitartrate Delayed-release | 0 | |
| Overall Study | NOT COMPLETED | Cysteamine Bitartrate Delayed-release | 22 |
Source ClinicalTrials.gov
Adverse events35
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Abdominal pain | Gastrointestinal disorders | Cysteamine Bitartrate Delayed-release | 2 | 22 | |
| other | Nausea | Gastrointestinal disorders | Cysteamine Bitartrate Delayed-release | 2 | 22 | |
| other | Vomiting | Gastrointestinal disorders | Cysteamine Bitartrate Delayed-release | 11 | 22 | |
| other | Asthenia | General disorders | Cysteamine Bitartrate Delayed-release | 2 | 22 | |
| other | Pyrexia | General disorders | Cysteamine Bitartrate Delayed-release | 5 | 22 | |
| other | Ataxia | Nervous system disorders | Cysteamine Bitartrate Delayed-release | 2 | 22 | |
| other | Convulsion | Nervous system disorders | Cysteamine Bitartrate Delayed-release | 2 | 22 | |
| other | Headache | Nervous system disorders | Cysteamine Bitartrate Delayed-release | 2 | 22 | |
| other | Lethargy | Nervous system disorders | Cysteamine Bitartrate Delayed-release | 2 | 22 | |
| other | Myoclonus | Nervous system disorders | Cysteamine Bitartrate Delayed-release | 2 | 22 |
1 to 10 of 35Source ClinicalTrials.gov
See all 35 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cysteamine Bitartrate Delayed-release | 0 | 22 |
| Total, other adverse events | Cysteamine Bitartrate Delayed-release | 18 | 22 |
| Total, serious adverse events | Cysteamine Bitartrate Delayed-release | 9 | 22 |
Source ClinicalTrials.gov
Papers4
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02473445
GET https://conductscience.org/api/trials/v1/NCT02473445/events?page=2