A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome (TAZPOWER)

Stealth BioTherapeutics

Status and phase: Completed; Phase 3; Phase 2

Conditions: Barth Syndrome

Treatments: Drug: Elamipretide; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT03098797

SPIBA-201

Details and patient eligibility

About

A randomized, double-blind cross over trial to evaluate the safety, efficacy, and tolerability of elamipretide in subjects with Barth syndrome.

Full description

A phase 2 randomized, double-blind, placebo-controlled crossover trial to evaluate the safety, tolerability, and efficacy of subcutaneous injections of elamipretide in subjects with genetically confirmed Barth syndrome followed by open-label treatment extension. Part 1 was a randomized, double-blind, placebo-controlled, crossover trial to assess safety, tolerability, and efficacy single daily subcutaneous (SC) doses of 40 mg elamipretide administered for 12 weeks in subjects with Barth syndrome. Part 2: This was an open-label extension trial to assess the long-term safety, tolerability, and longitudinal trends in efficacy single daily SC doses of 40 mg elamipretide for up to 192 weeks.

Enrollment: 12 patients

Sex: Male

Ages: 12+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Genetically confirmed Barth Syndrome

• Male aged 12 and above

• At the screening visit, eGFR must meet the following:

  1. Body weight >30 kg AND eGFR ≥ 90mL/min at screening
  2. Body weight >40kg AND eGFR ≥60 but <90mL/min/ 1.73m² at screening

• Ambulatory and impaired during the baseline 6MWT

• On stable medication for 30 days prior to the baseline visit

Exclusion criteria

• Participated in another interventional clinical trial within 30 days of or is currently enrolled in a non-interventional clinical trial at the baseline visit potentially confounding with this trial
• Prior or current medical condition that would prevent the subject from safely participating in the trial
• Undergone any inpatient hospitalizations within 30 days of the baseline visit
• Is undergoing an apparent pubertal growth spurt
• Has uncontrolled hypertension
• History of substance abused within the year before the baseline visit or is likely to be uncompliant
• History of heart transplantation or current placement on the waiting list for a heart transplant
• For subjects with an ICD: known occurrence of ICD discharge in the 3 months prior to the baseline visit
• For subjects without an ICD: expected to undergo an implantation of an ICD during the conduct of the study
• Currently receiving treatment with chemotherapeutic agents or immunosuppressant agents or has received prior radiation therapy to the chest
• Recipient of stem cell or gene therapy or is currently being treated by a therapeutic investigational device

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Quadruple Blind

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12 participants in 2 patient groups, including a placebo group

Elamipretide, then Placebo

Experimental group

Description:

Experimental Part 1: 12 weeks of single daily SC doses of 40 mg elamipretide in Treatment Period 1 followed by 12 weeks of treatment with placebo in Treatment Period 2 (separated by a 4 week washout period). Experimental Open Label Extension Part 2: All subjects will receive Elamipretide 40mg SC daily injections for up to 168 weeks

Treatment:

Drug: Elamipretide

Drug: Placebo

Placebo , then Elamipretide

Placebo Comparator group

Description:

Placebo Comparator Part 1: 12 weeks of single daily SC doses of placebo in Treatment Period 1 followed by 12 weeks of treatment with 40 mg elamipretide in Treatment Period 2 (separated by a 4 week washout period). Placebo Comparator Open Label Extension Part 2: All subjects will receive Elamipretide 40mg SC daily injections for up to 168 weeks

Treatment:

Drug: Elamipretide

Drug: Placebo