A Study of the Safety of REN001 in Patients With Primary Mitochondrial Myopathy

Phase 1TerminatedNCT03862846

Reneo Pharma Ltd23 enrolled

Overview

The purpose of this study is to assess REN001 safety in subjects with primary mitochondrial myopathy

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Primary Mitochondrial Myopathy

Interventions

REN001DRUG

Once daily

Eligibility

Sex: ALLMin age: 16 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Subjects must give written, signed and dated informed consent * Confirmed diagnosis of PMM according to the 2016 Rome Consensus recommendations * Confirmed mitochondrial mutation with evidence of myopathy * Able to remain on stable medication throughout the study Exclusion Criteria: * Documented evidence of ongoing rhabdomyolysis * Subjects with motor abnormalities other than related to mitochondrial disease * Treatment with an investigational drug within 3 months prior to Day 1 * Hospitalised within 3 months prior to screening for any major medical condition * Pregnant or nursing females

Locations (2)

Institute of Neurology, University College London

London, United Kingdom

Wellcome Centre for Mitochondrial Research

Newcastle upon Tyne, United Kingdom

Outcomes

Primary Outcomes

Adverse Events

Number of participants with Adverse Events as a measure of safety and tolerability

Time frame: Comparing Baseline to Week 12

Secondary Outcomes

Adverse Events

Number of participants with Adverse Events as a measure of safety and tolerability

Time frame: Continous to Week 48

Data from ClinicalTrials.gov

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