Study to Evaluate Pharmacokinetics, Safety, and Tolerability of Subcutaneous Injections of Elamipretide in Pediatric Subjects With Barth Syndrome
NCT07927972, Phase 1, Not yet recruiting, Stealth BioTherapeutics Inc.
Trial 10
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
| Field | Value | Source |
|---|---|---|
| Title | Study to Evaluate Pharmacokinetics, Safety, and Tolerability of Subcutaneous Injections of Elamipretide in Pediatric Subjects With Barth Syndrome | ClinicalTrials.gov |
| NCT number | NCT07927972 | ClinicalTrials.gov |
| Status | Not yet recruiting | ClinicalTrials.gov |
| Phases | Phase 1 | ClinicalTrials.gov |
| Enrollment | 6 participants, estimated | ClinicalTrials.gov |
| Lead sponsor | Stealth BioTherapeutics Inc. | ClinicalTrials.gov |
| Conditions | Barth Syndrome | ClinicalTrials.gov |
| Interventions | Elamipretide | ClinicalTrials.gov |
| First posted | 2026-11-10 | ClinicalTrials.gov |
| Last updated | 2026-11-10 | ClinicalTrials.gov |
Registered outcomes 11
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description | Source |
|---|---|---|---|---|
| Primary | Maximum plasma concentration of elamipretide and its metabolites | 24 hours | Characterization of the maximum plasma concentration (Cmax) of elamipretide and its metabolites utilizing population PK modeling. | ClinicalTrials.gov |
| Primary | Area under the plasma concentration time curve from 0 to 24 hours | 24 hours | Characterization of the area under the plasma concentration time curve from 0 to 24 hours of elamipretide and its metabolites (AUC0-24) utilizing population PK modeling. | ClinicalTrials.gov |
| Secondary | Safety Assessments: Adverse Events and Serious Adverse Events | 4 weeks | The incidence and severity of adverse events (AEs) and serious adverse events (SAEs) coded to system organ class and preferred term using the Medical Dictionary for Regulatory Activities (MedDRA) from baseline through 4 weeks. Report the # of subjects with adverse events and incidence and severity of such adverse events (AEs) | ClinicalTrials.gov |
| Secondary | Change from baseline of Clinical Hematology | 4 Weeks | Change from baseline in clinical hematology utilizing descriptive statistics of change relative to baseline and shift tables to show number of subjects who are low, normal, high at baseline verses week 4. | ClinicalTrials.gov |
| Secondary | Change in Clinical Chemistry from Baseline | 4 weeks | Change from baseline in Clinical Chemistry utilizing descriptive statistics of change relative to baseline and shift tables to show number of subjects who are low, normal, high at baseline verses week 4. | ClinicalTrials.gov |
| Secondary | Change in 12 Lead Electrocardiogram ECG QT Interval from Baseline | 4 weeks | Change in findings of 12 Lead Electrocardiogram ECG QT Interval from baseline | ClinicalTrials.gov |
| Secondary | Change in 12 Lead Electrocardiogram ECG heart rate from Baseline | 4 weeks | Change in 12 Lead Electrocardiogram ECG heart Rate from Baseline | ClinicalTrials.gov |
| Secondary | Change in Vital Signs from Baseline: Temperature | 4 weeks | Change in temperature measure (Fahrenheit or Celsius) from baseline to week 4 | ClinicalTrials.gov |
| Secondary | Change in Vital Signs from Baseline: Respiratory Rate | 4 weeks | Change in respiratory rate (breaths per minute) from baseline to week 4 | ClinicalTrials.gov |
| Secondary | Change in Vital Signs from Baseline: Blood Pressure | 4 weeks | Change in resting (5 minutes) Blood Pressure (mm Hg) from baseline to week 4 | ClinicalTrials.gov |
| Secondary | Change in Vital Signs from Baseline: heart rate | 4 weeks | Change in Heart Rate (beats per minute) from baseline to week 4 | ClinicalTrials.gov |
Outcomes 0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as | Source |
|---|
Results by group 0
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units | Source |
|---|
Participant flow 0
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted | Source |
|---|
Adverse events 0
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 | Source |
|---|
Adverse event totals 0
The totals the trial posted for each group.
| Total | Group | Affected | At risk | Source |
|---|
Papers 0
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as | Source |
|---|
Grants 0
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.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years | Source |
|---|
Trial comparisons 0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|
Tests 0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
| Test | Named as | Source |
|---|
Source 5
| Field | Value | Source |
|---|---|---|
| Release | trials-2026-11-12-0.4.0 | ClinicalTrials.gov |
| Release manifest SHA-256 | 0f4a8d8c48aee2a7573ae0fa94ecbeab70c1adbe449bbc95c424bc7d3e97b9d4 | ClinicalTrials.gov |
| Registry capture | 2026-11-12 | ClinicalTrials.gov |
| Results copy, AACT | 2026-03-23 | ClinicalTrials.gov |
| NIH clinical studies file | 2026-11-13 | ClinicalTrials.gov |
Release trials-2026-11-12-0.4.0
ClinicalTrials.gov's posted results, through CTTI's AACT: ClinicalTrials.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.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
ClinicalTrials.gov: none stated ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
NIH RePORTER's clinical studies and project files: Public domain (NIH RePORTER)