Study to Evaluate Pharmacokinetics, Safety, and Tolerability of Subcutaneous Injections of Elamipretide in Pediatric Subjects With Barth Syndrome
Barth Syndrome
De qué trata este estudio
El registro no identifica actualmente este estudio como reclutando. Consulta su estado antes de planificar una evaluación.
- Tipo de estudio
- De intervención
- Fase
- Fase 1
- Rango de edad
- No especificado - No especificado
- Sexo admitido
- Masculino
- Voluntarios sanos
- No aceptados
- Participantes
- 6 Estimado
Estos son puntos de partida, no una decisión de elegibilidad. El equipo debe revisar todos los requisitos.
Las explicaciones y la navegación están en español. Los textos proporcionados por el registro, incluidos títulos, resúmenes y criterios, se conservan en su idioma original. Puedes pedir orientación en español a WCR.
Acerca de la investigación
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.
Leer la descripción completa
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.
¿Quién podría participar?
La lista siguiente contiene el texto del patrocinador. Puede incluir condiciones, tratamientos previos, resultados de pruebas y otros requisitos. Aparecer en tu búsqueda no significa que califiques.
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.
Tratamiento, grupos y participación
El registro puede describir tratamientos y duración sin incluir un calendario completo. Pide al equipo las visitas, procedimientos, riesgos, costos y alternativas. Mantén tu atención habitual salvo que tu médico te indique un cambio.
- Asignación
- No aplica
- Enmascaramiento
- Sin enmascaramiento
Elamipretide
SC injection daily
Drug: Elamipretide
Elamipretide DRUG
SC injection daily
Qué evalúan los investigadores
Estas medidas indican qué se estudia. No prueban que un tratamiento funcione.
Medidas principales
Maximum plasma concentration of elamipretide and its metabolites
Plazo: 24 hours
Characterization of the maximum plasma concentration (Cmax) of elamipretide and its metabolites utilizing population PK modeling.
Area under the plasma concentration time curve from 0 to 24 hours
Plazo: 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.
Medidas secundarias
Safety Assessments: Adverse Events and Serious Adverse Events
Plazo: 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)
Change from baseline of Clinical Hematology
Plazo: 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.
Change in Clinical Chemistry from Baseline
Plazo: 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.
Change in 12 Lead Electrocardiogram ECG QT Interval from Baseline
Plazo: 4 weeks
Change in findings of 12 Lead Electrocardiogram ECG QT Interval from baseline
Change in 12 Lead Electrocardiogram ECG heart rate from Baseline
Plazo: 4 weeks
Change in 12 Lead Electrocardiogram ECG heart Rate from Baseline
Change in Vital Signs from Baseline: Temperature
Plazo: 4 weeks
Change in temperature measure (Fahrenheit or Celsius) from baseline to week 4
Change in Vital Signs from Baseline: Respiratory Rate
Plazo: 4 weeks
Change in respiratory rate (breaths per minute) from baseline to week 4
Change in Vital Signs from Baseline: Blood Pressure
Plazo: 4 weeks
Change in resting (5 minutes) Blood Pressure (mm Hg) from baseline to week 4
Change in Vital Signs from Baseline: heart rate
Plazo: 4 weeks
Change in Heart Rate (beats per minute) from baseline to week 4
Revisa las tablas completas y sus limitaciones en el registro oficial.
Abrir resultados oficiales ↗Centros de investigación (0)
El estado general puede diferir del estado de un centro. Confirma la disponibilidad antes de trasladarte.
No hay centros indicados. Usa el contacto oficial del estudio.
Equipo y fechas
- Patrocinador principal
- Stealth BioTherapeutics Inc.
- Inicio del estudio
- 2026-11 Estimado
- Finalización primaria
- 2027-05 Estimado
- Finalización del estudio
- 2027-06 Estimado
- Primera publicación
- 10 nov 2026 Real
- Última actualización
- 10 nov 2026 Real
Contactos oficiales del estudio
Título oficial del estudio
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
Consulta a Wellness Clinical Research
Tu mensaje va a WCR, incluso si otra organización realiza este estudio. WCR puede orientarte, pero podría no realizar este estudio, tener acceso a su equipo ni poder inscribirte.
Fuente: API v2 de ClinicalTrials.gov. Patrocinadores e investigadores envían la información. La NLM no aprueba la seguridad ni la ciencia de todos los estudios registrados. Aviso del registro.
Consultado: 12 nov 2026 · Última actualización del registro: 10 nov 2026