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

July 14, 2020 updated by: Reneo Pharma Ltd

An Open-label Study to Evaluate the Safety and Tolerability of 12 Weeks Treatment With Oral REN001 in Patients With Primary Mitochondrial Myopathy (PMM), With an Optional Extension of Treatment

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

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

23

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United Kingdom
      • London, United Kingdom, WC1N 3BG
        • Institute of Neurology, University College London
      • Newcastle Upon Tyne, United Kingdom, NE2 4HH
        • Wellcome Centre for Mitochondrial Research

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

14 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Group
REN001 Low Dose
Drug: REN001
Once daily

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Adverse Events
Time Frame: Comparing Baseline to Week 12
Number of participants with Adverse Events as a measure of safety and tolerability
Comparing Baseline to Week 12

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Adverse Events
Time Frame: Continous to Week 48
Number of participants with Adverse Events as a measure of safety and tolerability
Continous to Week 48

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Reneo Pharma Ltd

Investigators

  • Principal Investigator: Grainne Gorman, MD, Wellcome Centre for Mitochondrial Research

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 10, 2019

Primary Completion (Actual)

April 23, 2020

Study Completion (Actual)

April 23, 2020

Study Registration Dates

First Submitted

February 28, 2019

First Submitted That Met QC Criteria

March 1, 2019

First Posted (Actual)

March 5, 2019

Study Record Updates

Last Update Posted (Actual)

July 16, 2020

Last Update Submitted That Met QC Criteria

July 14, 2020

Last Verified

March 1, 2020

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • REN001-101

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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