A Study of the Safety of REN001 in Patients With Primary Mitochondrial Myopathy
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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
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Primary Mitochondrial Myopathy | Drug: REN001 | Phase 1 |
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 |

Arm | Intervention/treatment |
---|---|
Experimental: Group
REN001 Low Dose
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Drug: REN001
Once daily |
- Adverse Events [ Time Frame: Comparing Baseline to Week 12 ]Number of participants with Adverse Events as a measure of safety and tolerability
- Adverse Events [ Time Frame: Continous to Week 48 ]Number of participants with Adverse Events as a measure of safety and tolerability

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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 |
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

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
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 |
Principal Investigator: | Grainne Gorman, MD | Wellcome Centre for Mitochondrial Research |
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 |
Studies a U.S. FDA-regulated Drug Product: | No |
Studies a U.S. FDA-regulated Device Product: | No |
Muscular Diseases Mitochondrial Myopathies Musculoskeletal Diseases Neuromuscular Diseases |
Nervous System Diseases Mitochondrial Diseases Metabolic Diseases |