Trial · NCT02023866
Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease
NCT02023866, Phase 2, Completed, Amgen; results first posted 2017-11-13
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease. Trials, record NCT02023866. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02023866
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease
- NCT number
- NCT02023866
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 36 participants, actual
- Lead sponsor
- Amgen
- Conditions
- Inherited Mitochondrial Disease, Including Leigh Syndrome
- Interventions
- Cysteamine Bitartrate
- First posted
- 2013-12-30
- Last updated
- 2024-12-27
- Results first posted
- 2017-11-13
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | 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 | Change From Baseline in Glutathione | Baseline and Weeks 4, 8, 12, 16, 20, 24 | |
| Secondary | Change From Baseline in Glutathione Disulfide | Baseline and Weeks 4, 8, 12, 16, 20, 24 | |
| Secondary | Change From Baseline in Lactic Acid | Baseline and Weeks 4, 8, 12, 16, 20, 24 | |
| Secondary | Change From Baseline in 6 Minute Walk Test | Baseline and Weeks 4, 8, 12, 16, 20, 24 | The investigator selected the two most preeminent symptoms for each participant during the screening visit from the following: Myopathy, dystonia, ataxia, retarded motor development, reduced activities of daily living, and vision. The 2 symptoms selected for each participant were then assessed at each subsequent study visit. Myopathy was assessed using the 6 minute walk test, which measures the distance walked in a 6 minute walk test. |
| Secondary | Change From Baseline in Jamar Dynamometer Hand Strength | Baseline and Weeks 4, 8, 12, 16, 20, 24 | The investigator selected the two most preeminent symptoms for each participant during the screening visit from the following: myopathy, dystonia, ataxia, retarded motor development, reduced activities of daily living, and vision. The 2 symptoms selected for each participant were then assessed at each subsequent study visit. Myopathy was assessed using standard grip strength evaluation, which measures hand strength in both hands using a Jamar dynamometer. |
| Secondary | Change From Baseline in Barry-Albright Dystonia Scale Total Score | Baseline and Weeks 4, 8, 12, 16, 20, 24 | The investigator selected the two most preeminent symptoms for each participant during the screening visit from the following: myopathy, dystonia, ataxia, retarded motor development, reduced activities of daily living, and vision. The 2 symptoms selected for each participant were then assessed at each subsequent study visit. Dystonia symptoms were assessed using the Barry-Albright Dystonia Scale for Dystonia. Participants were assessed for dystonia in each of the following regions: eyes, mouth, neck, trunk, and each upper and lower extremity (8 body regions) on a scale from 0 (absent) to 4 (severe symptoms). The individual scores were summed to calculate the total score which ranges from 0 (dystonia absent) to 32 (severe dystonia). |
| Secondary | Change From Baseline in Friedreich Ataxia Rating Scale | Baseline and Weeks 4, 8, 12, 16, 20, 24 | The investigator selected the two most preeminent symptoms for each participant during the screening visit from the following: myopathy, dystonia, ataxia, retarded motor development, reduced activities of daily living, and vision. The 2 symptoms selected for each participant were then assessed at each subsequent study visit. Ataxia was assessed using the Friedreich Ataxia Rating Scale (FARS). FARS comprises a functional ataxia staging score of overall mobility (score 0 to 6), an assessment of the activities of daily living (ADL) (score 0 to 36) and a neurological assessment (score from 0 to 117) which is composed of bulbar (score 0-11), upper limb (score 0- 36) and lower limb (score 0-16), peripheral nerve (score 0-26) and upright stability/gait (score 0-28). The scores were summed to calculate the total score which ranges from 0 to 159. A higher score indicates a greater level of disability. |
| Secondary | Change From Baseline in Gross Motor Function | Baseline and Weeks 4, 8, 12, 16, 20, 24 | The investigator selected the two most preeminent symptoms for each participant during the screening visit from the following: myopathy, dystonia, ataxia, retarded motor development, reduced activities of daily living, and vision. The 2 symptoms selected for each participant were then assessed at each subsequent study visit. Retarded motor development was assessed using the Gross Motor Function Measure (GMFM)-88 which consists of 88 items scored on a scale of 0 to 3: 0: Does not initiate the task; 1. Initiates the task (completes \< 10%); 2. Partially completes the task (10 to 99%); 3. Completes the task (100%). The 88 items are grouped into five dimensions: 1) lying and rolling, 2) sitting, 3) crawling and kneeling, 4) standing, and 5) walking, running and jumping. Scores are expressed as a percentage of the maximum score for that dimension. The total score is the average of the 5 the percentage scores where higher scores indicate better performance. |
| Secondary | Change From Baseline in Modified Lansky Play Performance Scale | Baseline and Weeks 4, 8, 12, 16, 20, 24 | The investigator selected the 2 most preeminent symptoms for each participant during the screening visit from the following: myopathy, dystonia, ataxia, retarded motor development, reduced activities of daily living, and vision. The 2 symptoms were assessed at each subsequent study visit. Reduced activities of daily living was assessed using the modified Lansky Play Performance Scale, completed by parents based on their child's activity in the past week, where 100=fully active; 90=minor restrictions in strenuous physical activity; 80=active, gets tired more quickly; 70=greater restriction of play, less time spent in play activity; 60=up and around, active play minimal; quieter activities; 50=lying around much of the day; no active playing, all quiet play and activities; 40=mainly in bed; quiet activities; 30=bedbound; needs assistance even for quiet play; 20=sleeps often; play limited to very passive activities; 10=doesn't play or get out of bed; 5=unresponsive 0=dead |
Source ClinicalTrials.gov
Outcomes10
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Sections I-IV | Baseline through Week 24 | Completers Analysis Set included all participants who received at least one dose of study drug (RP103) with at least one post-baseline NPMDS assessment and an evaluable Week 24 NPMDS assessment within the protocol specified window. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in Glutathione | Baseline and Weeks 4, 8, 12, 16, 20, 24 | The pharmacodynamic (PD) analysis set included all participants who received at least one dose of study drug and had at least one post-baseline PD assessment. Participants with available data at each time point are included in the analysis. | µmol/L | MEAN |
| SECONDARY | Change From Baseline in Glutathione Disulfide | Baseline and Weeks 4, 8, 12, 16, 20, 24 | PD analysis set participants with available data at each time point. | µmol/L | MEAN |
| SECONDARY | Change From Baseline in Lactic Acid | Baseline and Weeks 4, 8, 12, 16, 20, 24 | PD analysis set participants with available data at each time point. | mmol/L | MEAN |
| SECONDARY | Change From Baseline in 6 Minute Walk Test | Baseline and Weeks 4, 8, 12, 16, 20, 24 | Participants who received at least one dose of study drug (RP103) and had at least one post-baseline 6 minute walk test assessment and for whom myopathy was prespecified as a preeminent symptom and with available data at each time point. | meters | MEAN |
| SECONDARY | Change From Baseline in Jamar Dynamometer Hand Strength | Baseline and Weeks 4, 8, 12, 16, 20, 24 | Participants who received at least one dose of study drug (RP103) and had at least one post-baseline Jamar hand strength assessment and for whom myopathy was prespecified as a preeminent symptom and with available data at each time point. | kg | MEAN |
| SECONDARY | Change From Baseline in Barry-Albright Dystonia Scale Total Score | Baseline and Weeks 4, 8, 12, 16, 20, 24 | Participants who received at least one dose of study drug (RP103) and had at least one post-baseline dystonia assessment and for whom dystonia was prespecified as a preeminent symptom and with available data at each time point. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in Friedreich Ataxia Rating Scale | Baseline and Weeks 4, 8, 12, 16, 20, 24 | Participants who received at least one dose of study drug (RP103) and had at least one post-baseline ataxia assessment and for whom ataxia was prespecified as a preeminent symptom and with available data at each time point. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in Gross Motor Function | Baseline and Weeks 4, 8, 12, 16, 20, 24 | Participants who received at least one dose of study drug (RP103) and had at least one post-baseline gross motor function assessment and for whom retarded motor development was prespecified as a preeminent symptom and with available data at each time point. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in Modified Lansky Play Performance Scale | Baseline and Weeks 4, 8, 12, 16, 20, 24 | Participants who received at least one dose of study drug (RP103) and had at least one post-baseline Lansky play performance scale assessment and for whom reduced activities of daily living was prespecified as a preeminent symptom and with available data at each time point. | units on a scale | MEAN |
Source ClinicalTrials.gov
Results by group64
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Sections I-IV | Cysteamine Bitartrate Delayed-release | Section I | -0.3 | Standard Deviation 0.7 | units on a scale |
| Change From Baseline in Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Sections I-IV | Cysteamine Bitartrate Delayed-release | Section II | 0.1 | Standard Deviation 1.1 | units on a scale |
| Change From Baseline in Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Sections I-IV | Cysteamine Bitartrate Delayed-release | Section III | -0.6 | Standard Deviation 1.2 | units on a scale |
| Change From Baseline in Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Sections I-IV | Cysteamine Bitartrate Delayed-release | Section IV | 0.0 | Standard Deviation 4.0 | units on a scale |
| Change From Baseline in Glutathione | Cysteamine Bitartrate Delayed-release | Week 4 | 16.8 | Standard Deviation 277.1 | µmol/L |
| Change From Baseline in Glutathione | Cysteamine Bitartrate Delayed-release | Week 8 | 141.5 | Standard Deviation 275.5 | µmol/L |
| Change From Baseline in Glutathione | Cysteamine Bitartrate Delayed-release | Week 12 | 10.2 | Standard Deviation 419.4 | µmol/L |
| Change From Baseline in Glutathione | Cysteamine Bitartrate Delayed-release | Week 16 | 88.7 | Standard Deviation 343.9 | µmol/L |
| Change From Baseline in Glutathione | Cysteamine Bitartrate Delayed-release | Week 20 | 51.4 | Standard Deviation 207.8 | µmol/L |
| Change From Baseline in Glutathione | Cysteamine Bitartrate Delayed-release | Week 24 | 122.4 | Standard Deviation 244.8 | µmol/L |
1 to 10 of 64Source ClinicalTrials.gov
See all 64 results by groupParticipant 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 | 36 | |
| Overall Study | COMPLETED | Cysteamine Bitartrate Delayed-release | 30 | |
| Overall Study | NOT COMPLETED | Cysteamine Bitartrate Delayed-release | 6 |
Source ClinicalTrials.gov
Adverse events135
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 | Sinus tachycardia | Cardiac disorders | Cysteamine Bitartrate Delayed-release | 1 | 36 | |
| other | Motion sickness | Ear and labyrinth disorders | Cysteamine Bitartrate Delayed-release | 1 | 36 | |
| other | Eye movement disorder | Eye disorders | Cysteamine Bitartrate Delayed-release | 1 | 36 | |
| other | Eye pain | Eye disorders | Cysteamine Bitartrate Delayed-release | 1 | 36 | |
| other | Eyelid oedema | Eye disorders | Cysteamine Bitartrate Delayed-release | 1 | 36 | |
| other | Eyelid ptosis | Eye disorders | Cysteamine Bitartrate Delayed-release | 2 | 36 | |
| other | Mydriasis | Eye disorders | Cysteamine Bitartrate Delayed-release | 1 | 36 | |
| other | Ocular hyperaemia | Eye disorders | Cysteamine Bitartrate Delayed-release | 1 | 36 | |
| other | Abdominal discomfort | Gastrointestinal disorders | Cysteamine Bitartrate Delayed-release | 1 | 36 | |
| other | Abdominal distension | Gastrointestinal disorders | Cysteamine Bitartrate Delayed-release | 1 | 36 |
1 to 10 of 135Source ClinicalTrials.gov
See all 135 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 | 1 | 36 |
| Total, other adverse events | Cysteamine Bitartrate Delayed-release | 35 | 36 |
| Total, serious adverse events | Cysteamine Bitartrate Delayed-release | 11 | 36 |
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/NCT02023866
GET https://conductscience.org/api/trials/v1/NCT02023866/results?page=2