Clinical Trial in Patients With Barth Syndrome- 4TAZPower
Barth Syndrome
De qué trata este estudio
El registro indica que este estudio recluta participantes. La disponibilidad puede variar por centro.
- Tipo de estudio
- De intervención
- Fase
- Fase 4
- Rango de edad
- 5 años - 55 años
- Sexo admitido
- Masculino
- Voluntarios sanos
- No aceptados
- Participantes
- 48 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
Phase 3b/4, randomized, double-blind, parallel-group, placebo-controlled clinical trial to evaluate the efficacy, safety, and pharmacokinetics of a once daily SC injection of elamipretide in subjects with genetically confirmed BTHS for 72 weeks. The primary trial objective is to confirm the efficacy of elamipretide which is approved in the United States(FORZINITY™) under the accelerated approval based on an improvement in knee extensor muscle strength, an intermediate clinical endpoint.
Leer la descripción completa
The SPIBA-401 trial is a post marketing Phase 3b/4, randomized, double-blind, parallel-group, placebo-controlled clinical trial to evaluate the efficacy, safety, and pharmacokinetics of a once daily subcutaneous (SC) injection of elamipretide in subjects with genetically confirmed Barth syndrome (BTHS) for 72 weeks. The primary trial objective is to confirm the efficacy of elamipretide which is approved in the United States under the name FORZINITY™ as a mitochondrial cardiolipin binder indicated to improve muscle strength in adult and pediatric patients with Barth syndrome weighing at least 30 kg. This indication is approved in the United States under accelerated approval based on an improvement in knee extensor muscle strength, an intermediate clinical endpoint.
¿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.
Key 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 ≥ 5 years at time of the Screening Visit
5. Left Ventricular Ejection fraction of ≥ 50% by 3-D Echocardiogram at the Screening Visit.
6. 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.
7. Able to administer Investigational Medicinal Product (IMP) or have an appropriate designee who can administer the IMP (i.e., a capable family member or a caregiver).
8. Subjects with female partners of childbearing potential must be willing to use a highly effective method of contraception (e.g., abstinence, dual method of contraception) from the date they sign the ICF until 28 days after the last dose of IMP.
Key Exclusion Criteria:
1. Unable to perform the required functional tests or undergo echocardiography.
2. 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, is stable, and does not pose additional safety risk to participant.
3. Patients with an implantable cardioverter defibrillator (ICD) and with a known occurrence of ICD discharge in the 3 months prior to the Screening Visit.
4. Current placement on the waiting list for heart transplantation.
5. Hospitalization for heart failure within 6 months prior to the Screening Visit.
6. 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.
7. Has a history of a systemic eosinophilic illness
8. Estimated Glomerular Filtration Rate (eGFR) of \< 30 mL/min at the Screening Visit (using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) 2021 formula for subjects \>16 years of age and the Schwartz 2009 formula for subjects 5-16 years of age).
9. 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.
10. Participation in other investigational drug or device clinical trials within 30 days or 5 half-lives (whichever is longer) of Screening; 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.
11. History of allergic reaction to the IMP or any of its components.
12. Prior participation in any elamipretide trial or expanded access programs.
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
- Asignación al azar
- Enmascaramiento
- Enmascaramiento cuádruple
Elamipretide
Elamipretide - aqueous, sterile 5.0 mL single-patient, ready to use, multi-dose glass vial containing 3.5 mL of elamipretide solution (elamipretide \[80 mg/mL\],
Drug: Elamipretide
Placebo
Matching Placebo-aqueous, sterile 5.0 mL single-patient, ready to use, multi-dose glass vial containing will be composed of 3.5mL of sodium chloride, phosphate buffer, and benzyl alcohol similar to excipients in active drug but without the active drug substance
Drug: Placebo
Elamipretide DRUG
sub cutaneous injection
Placebo DRUG
sub cutaneous injection
Qué evalúan los investigadores
Estas medidas indican qué se estudia. No prueban que un tratamiento funcione.
Medidas principales
Primary Efficacy End Point
Plazo: 72 weeks
-change in the composite normalized score of the three functional tests: Six-minute walk test (6MWT), the triple-timed up and go test (3TUG), and the Five times sit-to-stand test (5XSST) from baseline to 72 weeks of treatment
Medidas secundarias
Secondary Efficacy End Point 1
Plazo: 72 weeks
\- Change in 6Minute Walk Test from Baseline to Week 72
Secondary Efficacy End Point 2
Plazo: 72 weeks
-Change in 3Timed Up and Go Test from Baseline to Week 72
Secondary Efficacy End Point 3
Plazo: 72 weeks
-Change in 5XSit to Stand Test from Baseline to Week 72
Secondary Efficacy End Point 4
Plazo: 72 weeks
-Change in Patient Global Impression of Severity Scale (PGI-S) score from Baseline to Week 72
Secondary Efficacy End Point 5
Plazo: 72 weeks
Change in Clinician Global Impression of Severity Scale (CGI-S) score from Baseline to Week 72
Secondary Efficacy End Point
Plazo: 72 weeks
Change in knee extensor muscle strength as measured by handheld dynamometry (HHD)from Baseline to Week 72
Secondary Efficacy End Point 6
Plazo: 72 weeks
-Change in hip flexor muscle strength as measured by HHD from Baseline to Week 72
Otras medidas
Safety End Point Adverse Event Reporting
Plazo: 72weeks
-Report the # of subjects with adverse events and incidence and severity of such adverse events (AEs)
Revisa las tablas completas y sus limitaciones en el registro oficial.
Abrir resultados oficiales ↗Centros de investigación (4)
El estado general puede diferir del estado de un centro. Confirma la disponibilidad antes de trasladarte.
4 centros
Trial Not Offered in the U.SNeedham, Massachusetts, United StatesTerminado antes de tiempo
Children's Hospital of WestmeadWestmead, North South Wales, AustraliaReclutando
Metabolics and Genetics in Canada (MAGIC)Calgary, Alberta, CanadaReclutando
Bristol Royal Hospital for Children Upper Maudlin Street Paul O'Gorman BuildingBristol, United KingdomReclutando
Equipo y fechas
- Patrocinador principal
- Stealth BioTherapeutics Inc.
- Inicio del estudio
- 2 jul 2026 Real
- Finalización primaria
- 30 sept 2029 Estimado
- Finalización del estudio
- 30 nov 2029 Estimado
- Primera publicación
- 15 abr 2026 Real
- Última actualización
- 9 nov 2026 Real
Contactos oficiales del estudio
Título oficial del estudio
Phase 3b/4, Randomized, Double-Blind, Parallel-Group, Placebo-Controlled, Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Patients 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: 9 nov 2026