Primary Mitochondrial Myopathy MedDRA version: 20.0 Level: PT Classification code 10027710 Term: Mitochondrial myopathy System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: Mavodelpar
Product Code: REN001
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: Mavodelpar
Current Sponsor code: REN001
Other descriptive name: Sodium (4-{(E)-3-(4-fluorophenyl)-
Sponsors
Reneo Pharma Ltd.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1.mtDNA-PMM subjects: Completed treatment in the STRIDE study or participated in Study REN001-101, and in the opinion of the Investigator and Sponsor had 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 characterised by exercise intolerance or active muscle pain. 3.Willing and able to swallow the REN001 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. If subjects are transferred to a commercial supply of REN001 they must be advised to adhere to the contraceptive requirements. Are the trial subjects under 18? no Number of subjects for this age range: 0 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 180 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 20
Exclusion criteria
Exclusion criteria: 1.Anticipated to need a peroxisome proliferator-activated receptor (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) (See Table 1). 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, psychiatric or laboratory condition 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate long-term safety and tolerability of REN001 in subjects with PMM.;Secondary Objective: To assess patients with PMM who are receiving long-term treatment with REN001 in terms of PMM associated symptoms, exercise endurance, quality of life (QoL) and work productivity.;Primary end point(s): For mtDNA and nDNA subjects: •Number and severity of adverse events (AE) •Number of AEs leading to study drug discontinuation •Number of serious adverse events (SAEs) •Number of adverse events of special interest (AESIs) •Number of AEs leading to death ;Timepoint(s) of evaluation of this end point: throughout the study | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): For mtDNA and nDNA subjects: Absolute values, changes from baseline, and incidence of potentially clinically significant changes in: •Laboratory safety tests •Electrocardiograms (ECG) •Supine vital signs •Eye assessments For mtDNA subjects: Absolute values and changes from baseline in: •Distance walked during the 12-Minute Walk Test (12MWT) •Modified Fatigue Impact Scale (MFIS) total scores and sub-scale scores •Patient Global Impression of Severity (PGIS) scores for fatigue and muscle symptoms •Brief Pain Inventory (BPI) pain severity and pain interference scores •Patient Reported Outcomes Measurement Information System (PROMIS) Short Form-Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue 13a scores •36 Item Short Form Health Survey (SF-36) domain scores (7-day recall) •Work Productivity and Activity Impairment Questionnaire: Specific Health Problem (WPAI:SHP) scores •First 6-minute walk distance and last 6-minute walk distance •Phenotypic description question •PGIC (muscle and fatigue symptoms) scores ;Timepoint(s) of evaluation of this end point: throughout the study | — |
Countries
Australia, Belgium, Canada, Denmark, France, Germany, Hungary, Italy, Netherlands, New Zealand, Spain, United Kingdom
Contacts
Public ContactLynn Purkins
Reneo Pharma Ltd
Outcome results
None listed