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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03862846
Recruitment Status : Not yet recruiting
First Posted : March 5, 2019
Last Update Posted : March 5, 2019
Sponsor:
Information provided by (Responsible Party):
Reneo Pharma Ltd

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

Condition or disease Intervention/treatment Phase
Primary Mitochondrial Myopathy Drug: REN001 Phase 1

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 24 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Estimated Study Start Date : April 2019
Estimated Primary Completion Date : April 2020
Estimated Study Completion Date : May 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Muscle Disorders

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




Primary Outcome Measures :
  1. Adverse Events [ Time Frame: Comparing Baseline to Week 12 ]
    Number of participants with Adverse Events as a measure of safety and tolerability


Secondary Outcome Measures :
  1. Adverse Events [ Time Frame: Continous to Week 48 ]
    Number of participants with Adverse Events as a measure of safety and tolerability



Information from the National Library of Medicine

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Ages Eligible for Study:   16 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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 mediation 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03862846


Contacts
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Contact: Lynn Purkins, PhD 07538521498 lpurkins@reneopharma.com

Sponsors and Collaborators
Reneo Pharma Ltd
Investigators
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Principal Investigator: Grainne Gorman, MD Wellcome Centre for Mitochondrial Research

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Responsible Party: Reneo Pharma Ltd
ClinicalTrials.gov Identifier: NCT03862846     History of Changes
Other Study ID Numbers: REN001-101
First Posted: March 5, 2019    Key Record Dates
Last Update Posted: March 5, 2019
Last Verified: February 2019

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Muscular Diseases
Mitochondrial Myopathies
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Mitochondrial Diseases
Metabolic Diseases