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

NCT03098797 · Status: COMPLETED · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2024-04-16

Study results available
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Summary

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

Conditions

  • Barth Syndrome

Interventions

DRUG

Elamipretide

40 mg daily subcutaneous injection for 12 weeks

DRUG

Placebo

daily subcutaneous injection for 12 weeks

Sponsors & Collaborators

  • Stealth BioTherapeutics Inc.

    lead INDUSTRY

Principal Investigators

  • Hilary Vernon, MD, PhD · McKusick-Nathans Institute of Genetic Medicine, Johns Hopkins University, Baltimore, MD, USA

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
CROSSOVER

Eligibility

Min Age
12 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-07-05
Primary Completion
2018-10-05
Completion
2021-10-11
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03098797 on ClinicalTrials.gov