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

Stealth BioTherapeutics Inc.12 enrolled

Overview

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

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Barth Syndrome

Interventions

ElamipretideDRUG

40 mg daily subcutaneous injection for 12 weeks

PlaceboDRUG

daily subcutaneous injection for 12 weeks

Eligibility

Sex: MALEMin age: 12 Years

Medical Language ↔ Plain English

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

Locations (1)

McKusick-Nathans Institute of Genetic Medicine, Johns Hopkins University School of Medicine

Baltimore, Maryland, United States

Outcomes

Primary Outcomes

Part 1: Distance Walked During the 6-Minute Walk Test (6MWT) by Visit

Average distance walked in meters during the 6-minute walk test (6MWT) at end of treatment, where end of treatment means end of Visit 5 (Week 12) for Period 1 and end of Visit 10 (Week 12) for Period 2. Results were pooled for each 12 week period.

Time frame: Pre-dose to Week 12 (end of treatment)

Part 1: Total Fatigue Score Based on the BTHS-SA by Visit.

Total Fatigue Score (Q1, Q2, and Q4) Based on the BarTH Syndrome Symptom Assessment (BTHS-SA) by visit at predose, Weeks 1, 4, 8, and 12 (end of treatment). The BTHS-SA is 3-question fatigue assessment using a 0 to 4-point scale for each question where no fatigue =0 and very severe fatigue =4, with scores from all 3 questions combined and averaged for all subjects; a lower score means better outcome, higher score means a worse outcome. Combined Min score=0, max score =12. Weekly values are based on the weekly average of the last 7 days before the site or visiting nurse visit for both periods. Results from each 12-week period were combined, where end of treatment for Period 1 =Visit 5 (week 12), and End of Period 2 = Visit 10 (week 12)

Time frame: Pre-dose, Weeks 1, 4, 8, and 12 (end of treatment)

Secondary Outcomes

Part 2: Distance Walked During the 6MWT

Change from baseline for distance walked in meters during the 6-minute walk test (6MWT) by visit where Baseline= Part 1 Period 1 Pre-dose

Time frame: Baseline to Weeks 12, 24, 36, 48, 72, 96, 168, 192

Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit

Change from Baseline (mean) in Total Fatigue Score (Q1, Q2, and Q4) Based on the BarTH Syndrome Symptom Assessment (BTHS-SA) by visit. The BTHS-SA is 3-question fatigue assessment using a 0-4 point scale for each question where no fatigue =0 and very severe fatigue =4, with scores from all 3 questions combined and averaged for all subjects by visit; a lower score means better outcome, higher score means a worse outcome. Change from baseline: an increase in scores would mean worse outcome, a decrease in scores means better outcome. Combined Min score=-12, max score =12. Weekly values are based on the weekly average of the last 7 days before the site or visiting nurse visit for both periods. Baseline= Period 1 Predose.

Data from ClinicalTrials.gov

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