Trial · NCT03098797
A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome
NCT03098797, Phase 2, Phase 3, Completed, Stealth BioTherapeutics Inc.; results first posted 2024-04-16
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome. Trials, record NCT03098797. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03098797
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome
- NCT number
- NCT03098797
- Status
- Completed
- Phases
- Phase 2, Phase 3
- Enrollment
- 12 participants, actual
- Lead sponsor
- Stealth BioTherapeutics Inc.
- Conditions
- Barth Syndrome
- Interventions
- Elamipretide, Placebo
- First posted
- 2017-04-04
- Last updated
- 2024-04-16
- Results first posted
- 2024-04-16
Source ClinicalTrials.gov
Registered outcomes14
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Part 1: Distance Walked During the 6-Minute Walk Test (6MWT) by Visit | Pre-dose to Week 12 (end of treatment) | Average distance walked in meters during the 6-minute walk test (6MWT) at end of treatment, where end of treatment means end of Visit 5 (Week 12) for Period 1 and end of Visit 10 (Week 12) for Period 2. Results were pooled for each 12 week period. |
| Primary | Part 1: Total Fatigue Score Based on the BTHS-SA by Visit. | Pre-dose, Weeks 1, 4, 8, and 12 (end of treatment) | Total Fatigue Score (Q1, Q2, and Q4) Based on the BarTH Syndrome Symptom Assessment (BTHS-SA) by visit at predose, Weeks 1, 4, 8, and 12 (end of treatment). The BTHS-SA is 3-question fatigue assessment using a 0 to 4-point scale for each question where no fatigue =0 and very severe fatigue =4, with scores from all 3 questions combined and averaged for all subjects; a lower score means better outcome, higher score means a worse outcome. Combined Min score=0, max score =12. Weekly values are based on the weekly average of the last 7 days before the site or visiting nurse visit for both periods. Results from each 12-week period were combined, where end of treatment for Period 1 =Visit 5 (week 12), and End of Period 2 = Visit 10 (week 12) |
| Secondary | Part 2: Distance Walked During the 6MWT | Baseline to Weeks 12, 24, 36, 48, 72, 96, 168, 192 | Change from baseline for distance walked in meters during the 6-minute walk test (6MWT) by visit where Baseline= Part 1 Period 1 Pre-dose |
| Secondary | Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit | Baseline to Weeks 12, 24, 36, 48, 72, 96, 120, 144, 168, 192 | Change from Baseline (mean) in Total Fatigue Score (Q1, Q2, and Q4) Based on the BarTH Syndrome Symptom Assessment (BTHS-SA) by visit. The BTHS-SA is 3-question fatigue assessment using a 0-4 point scale for each question where no fatigue =0 and very severe fatigue =4, with scores from all 3 questions combined and averaged for all subjects by visit; a lower score means better outcome, higher score means a worse outcome. Change from baseline: an increase in scores would mean worse outcome, a decrease in scores means better outcome. Combined Min score=-12, max score =12. Weekly values are based on the weekly average of the last 7 days before the site or visiting nurse visit for both periods. Baseline= Period 1 Predose. |
| Secondary | Part 1: Muscle Strength as Measured by HHD by Visit | Pre-dose to Week 12 (end of treatment) | Muscle strength as measured by handheld dynamometry (HHD) in Newtons, where end of treatment means end of Visit 5 (Week 12) for Period 1 and end of Visit 10 (Week 12) for Period 2. Results were pooled for each 12 week period. Handheld dynamometry assesses the average strength of the knee extensors of both legs. |
| Secondary | Part 2: Change From Baseline: Muscle Strength by HHD (Newtons) by Visit | Baseline to Weeks 12, 24, 36, 48, 72, 96,168, 192 | Muscle strength as measured by handheld dynamometry (HHD) in Newtons. Handheld dynamometry assesses the average strength of the knee extensors of both legs. Change from baseline where Baseline= Part 1 Period 1 Pre-dose: the greater positive change, the better the outcome, the smaller positive change (or negative change) the number the worse the outcome. |
| Secondary | Part 1: 5XSST by Visit | Pre-dose to Week 12 (end of treatment) | Time (in seconds) to complete the Five Times Sit-To-Stand, where end of treatment means end of Visit 5 (Week 12) for Period 1 and end of Visit 10 (Week 12) for Period 2. Results were pooled for each 12 week period. |
| Secondary | Part 2: Change From Baseline: 5X Sit to Stand by Visit | Baseline to Weeks 12, 24, 36, 48, 72, 96, 168, 192 | Change in baseline in time (in seconds), where Baseline= Part 1 Period 1 Pre-dose to complete the Five Times Sit-To-Stand by visit. More time means worse outcome, less time means better outcome. |
| Secondary | Part 1: SWAY Application Balance Assessments by Visit | Pre-dose to Week 12 (end of treatment) | SWAY application balance assessment consists of five stances each performed for 10 seconds and scores postural stability on a scale from 0-100 with higher scores indicating better postural stability from Pre-dose to Week 12 (end of treatment), where end of treatment means end of Visit 5 (Week 12) for Period 1 and end of Visit 10 (Week 12) for Period 2. Results were pooled for each 12 week period. |
| Secondary | Part 2: Change From Baseline: SWAY Application Balance Assessment | Baseline to Weeks 12, 24, 36, 48, 72, 96,168, 192 | Change from Baseline: SWAY Application Balance Assessment,where Baseline= Part 1 Period 1 Pre-doseSWAY application balance assessment consists of five stances each performed for 10 seconds and scores postural stability on a scale from 0-100 with higher scores indicating better postural stability. An increase from baseline is a better outcome and a decrease in score is a worse outcome. |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 registered outcomesOutcomes14
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Part 1: Distance Walked During the 6-Minute Walk Test (6MWT) by Visit | Pre-dose to Week 12 (end of treatment) | All participants for whom 6MWT was measured. | meters | MEAN |
| PRIMARY | Part 1: Total Fatigue Score Based on the BTHS-SA by Visit. | Pre-dose, Weeks 1, 4, 8, and 12 (end of treatment) | All participants for whom Total Fatigue Score was measured | score on a scale | MEAN |
| SECONDARY | Part 2: Distance Walked During the 6MWT | Baseline to Weeks 12, 24, 36, 48, 72, 96, 168, 192 | All participants for whom distance walked in 6MWT was measured. | meters | MEAN |
| SECONDARY | Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit | Baseline to Weeks 12, 24, 36, 48, 72, 96, 120, 144, 168, 192 | All participants for whom Total Fatigue Score was measured. | score on a scale | MEAN |
| SECONDARY | Part 1: Muscle Strength as Measured by HHD by Visit | Pre-dose to Week 12 (end of treatment) | All participants for whom muscle strength as measured by handheld dynamometry was measured. | Newtons | MEAN |
| SECONDARY | Part 2: Change From Baseline: Muscle Strength by HHD (Newtons) by Visit | Baseline to Weeks 12, 24, 36, 48, 72, 96,168, 192 | All participants for whom Muscle Strength by HHD (Newtons) was measured. | Newtons | MEAN |
| SECONDARY | Part 1: 5XSST by Visit | Pre-dose to Week 12 (end of treatment) | All participants for whom 5XSST was measured. | seconds | MEAN |
| SECONDARY | Part 2: Change From Baseline: 5X Sit to Stand by Visit | Baseline to Weeks 12, 24, 36, 48, 72, 96, 168, 192 | All participants for whom 5X Sit to Stand was measured. | seconds | MEAN |
| SECONDARY | Part 1: SWAY Application Balance Assessments by Visit | Pre-dose to Week 12 (end of treatment) | All participants for who SWAY application balance was measured. | Score on a scale | MEAN |
| SECONDARY | Part 2: Change From Baseline: SWAY Application Balance Assessment | Baseline to Weeks 12, 24, 36, 48, 72, 96,168, 192 | All participants for whom SWAY Application Balance Assessment was measured. | score on a scale | MEAN |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 outcomesResults by group166
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Part 1: Distance Walked During the 6-Minute Walk Test (6MWT) by Visit | Experimental: Elamipretide | Pre-dose | 400.1 | Standard Deviation 55.07 | meters |
| Part 1: Distance Walked During the 6-Minute Walk Test (6MWT) by Visit | Placebo | Pre-dose | 412.6 | Standard Deviation 60.16 | meters |
| Part 1: Distance Walked During the 6-Minute Walk Test (6MWT) by Visit | Experimental: Elamipretide | Week 12 (End of Treatment | 443.1 | Standard Deviation 65.43 | meters |
| Part 1: Distance Walked During the 6-Minute Walk Test (6MWT) by Visit | Placebo | Week 12 (End of Treatment | 443.9 | Standard Deviation 77.10 | meters |
| Part 1: Total Fatigue Score Based on the BTHS-SA by Visit. | Experimental: Elamipretide | Pre-dose | 7.672 | Standard Deviation 1.9360 | score on a scale |
| Part 1: Total Fatigue Score Based on the BTHS-SA by Visit. | Placebo | Pre-dose | 7.433 | Standard Deviation 1.3848 | score on a scale |
| Part 1: Total Fatigue Score Based on the BTHS-SA by Visit. | Experimental: Elamipretide | Week 1 | 6.837 | Standard Deviation 2.1114 | score on a scale |
| Part 1: Total Fatigue Score Based on the BTHS-SA by Visit. | Placebo | Week 1 | 6.994 | Standard Deviation 1.7093 | score on a scale |
| Part 1: Total Fatigue Score Based on the BTHS-SA by Visit. | Experimental: Elamipretide | Week 4 | 6.220 | Standard Deviation 2.3325 | score on a scale |
| Part 1: Total Fatigue Score Based on the BTHS-SA by Visit. | Placebo | Week 4 | 6.485 | Standard Deviation 1.4447 | score on a scale |
1 to 10 of 166Source ClinicalTrials.gov
See all 166 results by groupParticipant flow30
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| First Intervention (12 Weeks) | STARTED | Experimental: Elamipretide, Then Placebo | 6 | |
| First Intervention (12 Weeks) | STARTED | Placebo, Then Elamipretide | 6 | |
| First Intervention (12 Weeks) | COMPLETED | Experimental: Elamipretide, Then Placebo | 6 | |
| First Intervention (12 Weeks) | COMPLETED | Placebo, Then Elamipretide | 6 | |
| First Intervention (12 Weeks) | NOT COMPLETED | Experimental: Elamipretide, Then Placebo | 0 | |
| First Intervention (12 Weeks) | NOT COMPLETED | Placebo, Then Elamipretide | 0 | |
| Washout (4 Weeks) | STARTED | Experimental: Elamipretide, Then Placebo | 6 | |
| Washout (4 Weeks) | STARTED | Placebo, Then Elamipretide | 6 | |
| Washout (4 Weeks) | COMPLETED | Experimental: Elamipretide, Then Placebo | 6 | |
| Washout (4 Weeks) | COMPLETED | Placebo, Then Elamipretide | 6 |
1 to 10 of 30Source ClinicalTrials.gov
See all 30 participant flowAdverse events508
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 | Injection site bruising | General disorders | Part 2 (OLE): Elamipretide (Sequence AB) | 0 | 5 | |
| other | Injection site bruising | General disorders | Part 2 (OLE): Elamipretide (Sequence BA) | 1 | 5 | |
| other | Injection site bruising | General disorders | Part 1: Elamipretide | 3 | 12 | |
| other | Injection site bruising | General disorders | Part 1: Placebo | 0 | 12 | |
| other | Medical site device erythema | General disorders | Part 2 (OLE): Elamipretide (Sequence AB) | 0 | 5 | |
| other | Medical site device erythema | General disorders | Part 2 (OLE): Elamipretide (Sequence BA) | 0 | 5 | |
| other | Medical site device erythema | General disorders | Part 1: Elamipretide | 1 | 12 | |
| other | Medical site device erythema | General disorders | Part 1: Placebo | 1 | 12 | |
| other | Medical device site irritation | General disorders | Part 2 (OLE): Elamipretide (Sequence AB) | 0 | 5 | |
| other | Medical device site irritation | General disorders | Part 2 (OLE): Elamipretide (Sequence BA) | 1 | 5 |
1 to 10 of 508Source ClinicalTrials.gov
See all 508 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Part 2 (OLE): Elamipretide (Sequence AB) | 0 | 5 |
| Total, all-cause mortality | Part 2 (OLE): Elamipretide (Sequence BA) | 0 | 5 |
| Total, all-cause mortality | Part 1: Elamipretide | 0 | 6 |
| Total, all-cause mortality | Part 1: Placebo | 0 | 6 |
| Total, other adverse events | Part 2 (OLE): Elamipretide (Sequence AB) | 5 | 5 |
| Total, other adverse events | Part 2 (OLE): Elamipretide (Sequence BA) | 5 | 5 |
| Total, other adverse events | Part 1: Elamipretide | 12 | 12 |
| Total, other adverse events | Part 1: Placebo | 12 | 12 |
| Total, serious adverse events | Part 2 (OLE): Elamipretide (Sequence AB) | 1 | 5 |
| Total, serious adverse events | Part 2 (OLE): Elamipretide (Sequence BA) | 4 | 5 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers3
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
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