AN OPEN-LABEL, MULTI-CENTRE STUDY TO EVALUATE THE LONG-TERM SAFETY AND TOLERABILITY OF REN001 IN SUBJECTS WITH PRIMARY MITOCHONDRIAL MYOPATHY (PMM)
- Conditions
- PMMPrimary Mitochondrial Myopathy1002929910029305
- Registration Number
- NL-OMON53482
- Lead Sponsor
- Reneo Pharma Ltd
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Pending
- Sex
- Not specified
- Target Recruitment
- 25
1. mtDNA-PMM subjects:Completed treatment in the STRIDE study or was
participating in Study REN001-101, and in the opinion of the Investigator and
the Sponsor have been compliant with the study requirements.
Or
nDNA-PMM subjects: Subjects aged 18 years or older with known nuclear (nDNA)
pathogenic
variants with a major muscle phenotype consisting of objective myopathy with
poor exercise tolerance. Proof of pathogenicity must be provided. Must be able
to walk at least 100m in the screening 12MWT and the limitations in walk test
must be primarily due to the energy deficit and not due to ataxia or any other
condition.
For subjects under 25 years old only: confirmation of bone growth plate closure
by wrist radiograph.
2. Have PMM which continues to be primarily characterized by exercise
intolerance or active muscle pain.
3. Willing and able to swallow gelatin capsules.
4. Concomitant medications (including supplements) intended for the treatment
of PMM or other co-morbidities likely to remain stable throughout participation
in the study where clinically possible.
5. Signed and dated informed consent document indicating that the subject has
been informed of all pertinent aspects of the study.
6. Females should be either of non-child-bearing potential or must agree to use
highly effective methods of contraception from baseline through to
approximately 30 days after last dose of study drug. Males with partners who
are WOCBP must also use contraception from baseline through to 14 weeks after
last dose of study drug.
1. Anticipated to need a PPAR agonist other than REN001 during the study.
2. Anticipated to need drugs during the study with a narrow therapeutic index
and Breast Cancer Resistant Protein (BCRP) mediated absorption, distribution,
metabolism and excretion (ADME).
3. Intent to donate blood, or blood components during the study or within one
month after completion of the study.
4. Current drug dependency. Use of opiates/cannabis for medical reasons is
acceptable with prescription evidence or at the Investigator*s discretion.
5. Current alcohol dependency.
6. Any medical or psychiatric condition or laboratory abnormality that may
increase the risk associated with study participation or interfere with the
interpretation of study results and, in the judgment of the Investigator and
Medical Monitor, would make the subject inappropriate for entry into this
study.
7. Pregnant or nursing females.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>The safety and tolerability of REN001 will be assessed by the following<br /><br>parameters:<br /><br><br /><br>•Number and severity of adverse events (AE)<br /><br>•Number of AEs leading to study drug discontinuation<br /><br>•Number of serious adverse events (SAEs)<br /><br>•Number of adverse events of special interest (AESIs)<br /><br>•Number of AEs leading to death<br /><br><br /><br>These end points will be evaluated throughout the study.</p><br>
- Secondary Outcome Measures
Name Time Method