Clinical trial NCT03098797: A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome
Official title: A Phase 2 Randomized, Double-Blind, Placebo-Controlled Crossover Trial to Evaluate the Safety and Efficacy of Subcutaneous Injections of Elamipretide in Subjects With Genetically Confirmed Barth Syndrome Followed by Open-Label Treatment
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Barth Syndrome
Keywords: Barth Syndrome
elamipretide
MTP-131
Stealth
Stealth BT
BTHS
Lead sponsor: Stealth BioTherapeutics Inc.
Sponsor class: INDUSTRY
First posted: 2017-04-04
Enrollment: 12
Sex: MALE
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Intervention model description: 28 weeks, double-blinded crossover followed by a 168-week open label long term safety \& tolerability trial.
Primary purpose: TREATMENT

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

Detailed 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.

Eligibility criteria: 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

Primary outcomes:
measure: Part 1: Distance Walked During the 6-Minute Walk Test (6MWT) by Visit
timeFrame: Pre-dose to Week 12 (end of treatment)
measure: Part 1: Total Fatigue Score Based on the BTHS-SA by Visit.
timeFrame: Pre-dose, Weeks 1, 4, 8, and 12 (end of treatment)
Secondary outcomes:
measure: Part 2: Distance Walked During the 6MWT
timeFrame: Baseline to Weeks 12, 24, 36, 48, 72, 96, 168, 192
measure: Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit
timeFrame: Baseline to Weeks 12, 24, 36, 48, 72, 96, 120, 144, 168, 192
measure: Part 1: Muscle Strength as Measured by HHD by Visit
timeFrame: Pre-dose to Week 12 (end of treatment)
measure: Part 2: Change From Baseline: Muscle Strength by HHD (Newtons) by Visit
timeFrame: Baseline to Weeks 12, 24, 36, 48, 72, 96,168, 192
measure: Part 1: 5XSST by Visit
timeFrame: Pre-dose to Week 12 (end of treatment)
measure: Part 2: Change From Baseline: 5X Sit to Stand by Visit
timeFrame: Baseline to Weeks 12, 24, 36, 48, 72, 96, 168, 192
measure: Part 1: SWAY Application Balance Assessments by Visit
timeFrame: Pre-dose to Week 12 (end of treatment)
measure: Part 2: Change From Baseline: SWAY Application Balance Assessment
timeFrame: Baseline to Weeks 12, 24, 36, 48, 72, 96,168, 192
measure: Part 1: Patient Global Impression Scales of Symptoms
timeFrame: Pre-dose, Week 1, Week 12 (end of treatment)
measure: Part 2: Change From Baseline: Patient Global Impression Scales of Symptoms by Visit
timeFrame: Baseline to Weeks 12, 24, 36, 48, 72, 96, 120, 144, 168, 192
measure: Part 1: Part 1: Clinician Global Impression
timeFrame: Pre-dose to Week 12 (end of treatment)
measure: Part 2: Change From Baseline: CGI Symptom Scale
timeFrame: Baseline to Weeks 12, 24, 36, 48, 72, 96, 120, 144, 168, 192
Other outcomes:
Locations:
city: Baltimore
country: United States
facility: McKusick-Nathans Institute of Genetic Medicine, Johns Hopkins University School of Medicine
state: Maryland
Study officials:
affiliation: McKusick-Nathans Institute of Genetic Medicine, Johns Hopkins University, Baltimore, MD, USA
name: Hilary Vernon, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: