Primary Mitochondrial MyopathyPhase 1NCT03862846
A Study of the Safety of REN001 in Patients With Primary Mitochondrial Myopathy
A Phase 1 study testing REN001 in people with Primary Mitochondrial Myopathy, run by Reneo Pharma Ltd at 2 sites in United Kingdom.
Trial summary
The purpose of this study is to assess REN001 safety in subjects with primary mitochondrial myopathy Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Primary Mitochondrial Myopathy
- What is being tested?
- REN001
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 23 participants
- Where
- 2 sitesUnited Kingdom
- Study dates
- 10 May 2019to 23 Apr 2020
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
2 sites in 1 country.
United Kingdom2 sites
Institute of Neurology, University College London
London, WC1N 3BG
Wellcome Centre for Mitochondrial Research
Newcastle upon Tyne, NE2 4HH
About this study
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
The purpose of this study is to assess REN001 safety in subjects with primary mitochondrial myopathy
- Conditions
- Primary Mitochondrial Myopathy
- Being tested
- REN001
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
REN001Drug
Once daily
| Study group | Role | What they receive |
|---|---|---|
| Group | Experimental | REN001 Low Dose Receives: Drug: REN001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events
Number of participants with Adverse Events as a measure of safety and tolerability
Measured: Comparing Baseline to Week 12
Secondary outcomes (1)
Adverse Events
Number of participants with Adverse Events as a measure of safety and tolerability
Measured: Continous to Week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Mar 2019
- Study start10 May 2019
- Primary completion23 Apr 2020
- Study completion23 Apr 2020
Why it stopped: COVID-19 Pandemic sufficient data gathered to achieve the study objective
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Mitochondrial MyopathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 23 participants (actual)
Sponsor and investigators
- Lead sponsor
- Reneo Pharma Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Grainne Gorman, MD, Principal investigator, Wellcome Centre for Mitochondrial Research
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03862846
- Sponsor's protocol ID
- REN001-101
- Status last verified
- Mar 2020
- First submitted
- 28 Feb 2019
- Last updated
- 16 Jul 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Jul 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03862846
- Acronym / sponsor ID
- REN001-101
- Conditions
- Primary Mitochondrial Myopathy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: COVID-19 Pandemic sufficient data gathered to achieve the study objective
- Enrolment
- 23 participants (actual)
- Lead sponsor
- Reneo Pharma LtdIndustry (pharma, biotech, devices)
- Study start
- 10 May 2019 (actual)
- Primary completion
- 23 Apr 2020 (actual)
- Study completion
- 23 Apr 2020 (actual)
- First posted
- 5 Mar 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to assess REN001 safety in subjects with primary mitochondrial myopathy
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Group | Experimental | Drug: REN001REN001 Low Dose |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| REN001Once daily | Drug | Group |
Primary outcome
Adverse Events
Time frame: Comparing Baseline to Week 12
Number of participants with Adverse Events as a measure of safety and tolerability
1 secondary outcome
Adverse Events
Time frame: Continous to Week 48
Results and safety
The study was stopped early (reason given: COVID-19 Pandemic sufficient data gathered to achieve the study objective). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jul 2020.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Grainne Gorman, MD principal investigator
Wellcome Centre for Mitochondrial Research
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing REN001 in people with Primary Mitochondrial Myopathy, run by Reneo Pharma Ltd at 2 sites in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 16 Jul 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: COVID-19 Pandemic sufficient data gathered to achieve the study objective). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse Events (time frame: Comparing Baseline to Week 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Jul 2020.