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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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03862846
Recruitment Status : Terminated (COVID-19 Pandemic sufficient data gathered to achieve the study objective)
First Posted : March 5, 2019
Last Update Posted : July 16, 2020
Sponsor:
Information provided by (Responsible Party):
Reneo Pharma Ltd

Study Description
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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

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 23 participants
Allocation: N/A
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
Actual Study Start Date : May 10, 2019
Actual Primary Completion Date : April 23, 2020
Actual Study Completion Date : April 23, 2020

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


Outcome Measures
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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


Eligibility Criteria
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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 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
Contacts and Locations
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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


Locations
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United Kingdom
Institute of Neurology, University College London
London, United Kingdom, WC1N 3BG
Wellcome Centre for Mitochondrial Research
Newcastle Upon Tyne, United Kingdom, NE2 4HH
Sponsors and Collaborators
Reneo Pharma Ltd
Investigators
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Principal Investigator: Grainne Gorman, MD Wellcome Centre for Mitochondrial Research
More Information
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Responsible Party: Reneo Pharma Ltd
ClinicalTrials.gov Identifier: NCT03862846    
Other Study ID Numbers: REN001-101
First Posted: March 5, 2019    Key Record Dates
Last Update Posted: July 16, 2020
Last Verified: March 2020

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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