Back to resultsA Study of the Safety of REN001 in Patients With Primary Mitochondrial Myopathy
Primary Purpose
Primary Mitochondrial Myopathy
Status
Terminated
Phase
Phase 1
Locations
United Kingdom
Study Type
Interventional
Intervention
REN001
Sponsored by
About this trial
This is an interventional treatment trial for Primary Mitochondrial Myopathy
Eligibility 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
Sites / Locations
- Institute of Neurology, University College London
- Wellcome Centre for Mitochondrial Research
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Group
Arm Description
REN001 Low Dose
Outcomes
Primary Outcome Measures
Adverse Events
Number of participants with Adverse Events as a measure of safety and tolerability
Secondary Outcome Measures
Adverse Events
Number of participants with Adverse Events as a measure of safety and tolerability
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03862846
Brief Title
A Study of the Safety of REN001 in Patients With Primary Mitochondrial Myopathy
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
Study Type
Interventional
2. Study Status
Record Verification Date
March 2020
Overall Recruitment Status
Terminated
Why Stopped
COVID-19 Pandemic sufficient data gathered to achieve the study objective
Study Start Date
May 10, 2019 (Actual)
Primary Completion Date
April 23, 2020 (Actual)
Study Completion Date
April 23, 2020 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Reneo Pharma Ltd
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
The purpose of this study is to assess REN001 safety in subjects with primary mitochondrial myopathy
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Mitochondrial Myopathy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
23 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Group
Arm Type
Experimental
Arm Description
REN001 Low Dose
Intervention Type
Drug
Intervention Name(s)
REN001
Intervention Description
Once daily
Primary Outcome Measure Information:
Title
Adverse Events
Description
Number of participants with Adverse Events as a measure of safety and tolerability
Time Frame
Comparing Baseline to Week 12
Secondary Outcome Measure Information:
Title
Adverse Events
Description
Number of participants with Adverse Events as a measure of safety and tolerability
Time Frame
Continous to Week 48
10. Eligibility
Sex
All
Minimum Age & Unit of Time
16 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Grainne Gorman, MD
Organizational Affiliation
Wellcome Centre for Mitochondrial Research
Official's Role
Principal Investigator
Facility Information:
Facility Name
Institute of Neurology, University College London
City
London
ZIP/Postal Code
WC1N 3BG
Country
United Kingdom
Facility Name
Wellcome Centre for Mitochondrial Research
City
Newcastle Upon Tyne
ZIP/Postal Code
NE2 4HH
Country
United Kingdom
12. IPD Sharing Statement
Learn more about this trial
A Study of the Safety of REN001 in Patients With Primary Mitochondrial Myopathy
We'll reach out to this number within 24 hrs