Study to Evaluate Pharmacokinetics, Safety, and Tolerability of Subcutaneous Injections of Elamipretide in Pediatric Subjects With Barth Syndrome (SPIBA-101)

November 3, 2026 updated by: Stealth BioTherapeutics Inc.

A Phase 1 Open-Label Trial to Evaluate the Pharmacokinetics, Safety, and Tolerability of Subcutaneous Injections of Elamipretide in Pediatric Subjects With Genetically Confirmed Barth Syndrome

This Phase 1, open-label trial will enroll approximately 6 subjects who have BTHS weighing between 7 and 30 kg. Subjects will receive a weight-based dose of elamipretide daily via SC injection for 4 weeks. PK samples will be collected on Day 1 and at week 4 to determine PK profile in BTHS patients weighing between 7 and 30kg.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Not yet recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

In this Phase 1, open-label trial to evaluate the PK, safety, and tolerability of elamipretide, approximately 6 pediatric subjects with genetically confirmed BTHS weighing between 7 and 30 kg will be enrolled to receive daily dosing via SC injection for 4 weeks.

To characterize the PK of elamipretide, PK sampling will be conducted at Day 1 and Week 4. The study is intended to characterize PK and safety across a broader range of body weights that haven't been studied in prior trials.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
6

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

Inclusion Criteria:

  1. Willing and able to provide signed informed consent form (ICF) prior to participation in any trial-related procedures. If applicable, informed consent in writing from parent(s) or legally-acceptable representative(s) and, informed assent from subject (if age appropriate according to local requirements) should be provided.
  2. Agrees to adhere to the trial requirements for the length of the trial.
  3. Must have genetically confirmed Barth Syndrome (pathogenic variant in the TAZ gene)
  4. Male aged ≥ 1 year and weighing between 7 and 30 kg at time of the Baseline Visit
  5. For subjects with a medical history of cardiomyopathy, must be on a stable regimen (unchanged and constant) of background heart failure medications for at least 3 months prior to the Screening Visit.
  6. Able to administer IMP or have an appropriate designee who can administer the IMP (i.e., a capable family member or a caregiver).

Exclusion Criteria:

  1. History of solid organ transplant, except successful cardiac transplantation >12 months prior to Screening, if, in the opinion of the Investigator, there is no evidence of organ rejection and post-transplant pharmacotherapy, the subject is stable, and participation in the trial does not pose additional safety risk.
  2. Current placement on the waiting list for heart transplantation.
  3. Hospitalization for heart failure within 6 months prior to the Screening/Baseline Visit
  4. Any disease or medical condition that in the opinion of the Investigator would prevent the subject from successfully participating in the trial and reliably completing the assessments or might confound trial results.
  5. eGFR of < 30 mL/min at the Screening Visit (for purposes of Screening, eGFR can be confirmed locally at the clinical site. Historical values within 3 months prior to Screening can be accepted).
  6. Active malignancy or any other cancer from which the subject has been cancer-free for < 2 years. Localized squamous or non-invasive basal cell skin carcinomas are allowed, if appropriately treated prior to Screening/Baseline.
  7. Participation in other investigational drug or device clinical trials within 30 days or 5 half-lives (whichever is longer) of Screening/Baseline; or is currently enrolled in a non-interventional clinical trial that, in the opinion of the Investigator, may be potentially confounding to the results of the current trial. NOTE: Subjects currently treated with elamipretide through an EAP are eligible to participate in the SPIBA-101 study.
  8. History of allergic reaction to the IMP or any of its components.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Elamipretide

SC injection daily
Drug: Elamipretide

SC injection daily

Other Names:
  • FORZINITY

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Maximum plasma concentration of elamipretide and its metabolites
Time Frame: 24 hours

Characterization of the maximum plasma concentration (Cmax) of elamipretide and its metabolites utilizing population PK modeling.
24 hours
Area under the plasma concentration time curve from 0 to 24 hours
Time Frame: 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.
24 hours

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Safety Assessments: Adverse Events and Serious Adverse Events
Time Frame: 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)
4 weeks
Change from baseline of Clinical Hematology
Time Frame: 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.
4 Weeks
Change in Clinical Chemistry from Baseline
Time Frame: 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.
4 weeks
Change in 12 Lead Electrocardiogram ECG QT Interval from Baseline
Time Frame: 4 weeks

Change in findings of 12 Lead Electrocardiogram ECG QT Interval from baseline
4 weeks
Change in 12 Lead Electrocardiogram ECG heart rate from Baseline
Time Frame: 4 weeks

Change in 12 Lead Electrocardiogram ECG heart Rate from Baseline
4 weeks
Change in Vital Signs from Baseline: Temperature
Time Frame: 4 weeks

Change in temperature measure (Fahrenheit or Celsius) from baseline to week 4
4 weeks
Change in Vital Signs from Baseline: Respiratory Rate
Time Frame: 4 weeks

Change in respiratory rate (breaths per minute) from baseline to week 4
4 weeks
Change in Vital Signs from Baseline: Blood Pressure
Time Frame: 4 weeks

Change in resting (5 minutes) Blood Pressure (mm Hg) from baseline to week 4
4 weeks
Change in Vital Signs from Baseline: heart rate
Time Frame: 4 weeks

Change in Heart Rate (beats per minute) from baseline to week 4
4 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Stealth BioTherapeutics Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
November 1, 2026

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
May 1, 2027

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
June 1, 2027

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
October 23, 2026

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 3, 2026

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
November 10, 2026

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
November 10, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 3, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
November 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • SPIBA-101

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Cookie Settings

We use cookies to ensure that we give you the best experience on our website. For more details carefully read our Privacy and Cookies Policy. ...>>>You can change your preferences or withdraw your consent at any time by deleting the cookies from your website or computer as described in the policy. Please accept it and choose your preferences by ticking the corresponding boxes in the "Manage Settings" section below. Please carefully read our Terms of Service before you proceed with using any part of this website.
«Manage Settings