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A Multicenter, Open-Label Phase 2 Extension Trial to Characterize the Long-term Safety and Tolerability of Subcutaneous Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Disease (PMD)

2016-11-30 15:45:39 | BioPortfolio

Summary

This is a Phase 2 Open-Label extension study to evaluate the long term safety and tolerability of daily elamipretide injections in patients with genetically confirmed Primary Mitochondrial Disease who previously participated in the SPIMM-201 Clinical Trial

Description

This open-label, non-comparative, extension trial will enroll subjects with genetically confirmed PMD who have completed the End-of-Study Visit in the SPIMM-202 trial. Subjects who do not discontinue or withdraw from the trial will receive treatment with 40 mg SC elamipretide for the shortest of the following: 260 weeks; regulatory approval and commercial availability of elamipretide in the subject's respective country; or termination of the clinical development for elamipretide in subjects with PMD.

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Conditions

Primary Mitochondrial Disease

Intervention

ELA4

Location

Akron Children's Hospital
Akron
Ohio
United States

Status

Not yet recruiting

Source

Stealth BioTherapeutics Inc.

Results (where available)

View Results

Links

Published on BioPortfolio: 2016-11-30T15:45:39-0500

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