Trial Review

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT04535609




Registration number
NCT04535609
Ethics application status
Date submitted
27/08/2020
Date registered
2/09/2020
Date last updated
8/05/2024

Titles & IDs
Public title
An Efficacy and Safety Study of 24 Week Treatment With Mavodelpar (REN001) in Primary Mitochondrial Myopathy Patients
Scientific title
A Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of 24 Weeks Treatment With REN001 in Patients With Primary Mitochondrial Myopathy
Secondary ID [1] 0 0
REN001-201
Universal Trial Number (UTN)
Trial acronym
STRIDE
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Primary Mitochondrial Myopathy 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Mavodelpar
Treatment: Drugs - Placebo

Experimental: Mavodelpar - Once daily

Placebo Comparator: Matched placebo - Once daily


Treatment: Drugs: Mavodelpar
Once daily

Treatment: Drugs: Placebo
Once daily
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change in Distance Walked During a 12 Minute Walk Test
Timepoint [1] 0 0
Baseline to Week 24
Secondary outcome [1] 0 0
Change in PROMIS Short Form - Fatigue 13a (FACIT-fatigue) Scores
Timepoint [1] 0 0
Baseline to Week 24

Eligibility
Key inclusion criteria
1. Subjects age 18 years or older with PMM as defined by the International Workshop:
Outcome measures and clinical trial readiness in primary mitochondrial myopathies in
children and adult (Mancuso et al 2017).

2. A confirmed PMM diagnosis due to known pathogenic gene mutation or deletion of the
mitochondrial genome. The Sponsor may authorize local genetic testing at Screening, if
required, but results must be available prior to randomization of the subject.

3. Documented PMM primarily characterized by exercise intolerance or active muscle pain.

4. Subjects must be ambulatory and able to perform the walking tests independently
(walking aids are allowed).

5. Have no changes to any therapeutic exercise regimen within 30 days prior to Day 1 and
be willing to remain on the same therapeutic exercise regimen for the duration of the
study.

6. Females should be either of non-child-bearing potential or must agree to use highly
effective methods of contraception from Screening through to 30 days after last dose
in the study. Males with partners who are WOCBP must also use contraception.

7. Concomitant medications (including supplements) must be stable for at least 1 month
prior to enrolment and throughout participation in the study.

8. Evidence of a personally signed and dated informed consent document indicating that
the subject has been informed of all pertinent aspects of the study.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion:

1. Participation in a prior REN001 (previously known as HPP-593) study.

2. Currently taking or anticipated to need a PPAR agonist during the study.

3. Subjects with bone deformities or motor abnormalities other than related to the
mitochondrial myopathy that may interfere with the outcome measures.

4. Clinically significant kidney disease or impairment calculated as eGFR Grade 2 or
above <60ml/min/1.73m2 using the Chronic Kidney Disease Epidemiology Collaboration
(CKD-EPI) creatinine equation at Screening.

5. Clinically significant liver disease or impairment of AST or ALT Grade 2 or above
(>2.5 x ULN), or Total bilirubin > 1.6 x ULN or >ULN with other signs and symptoms of
hepatotoxicity at Screening.

6. Subjects with uncontrolled diabetes and/or a Screening HbA1c of =11%.

7. Evidence of significant concomitant clinical disease that may need a change in
management during the study or could interfere with the conduct or safety of this
study. (Stable well-controlled chronic conditions such hypercholesterolemia,
gastroesophageal reflux, or depression under control with medication (other than
tricyclic antidepressants), are acceptable provided the symptoms and medications would
not be predicted to compromise safety or interfere with the tests and interpretations
of this study.)

8. Subjects with a history of cancer. A history of in situ basal cell carcinoma in the
skin is allowed.

9. Clinically significant cardiac disease and/or clinically significant ECG abnormalities
such as 2nd degree heart block, symptomatic tachyarrhythmia or unstable arrythmia
(right bundle branch block, left fascicular block and long PR interval are not
excluded) that in the opinion of the Investigator should exclude the subject from
completing exercise tests.

10. Evidence of hospitalization for rhabdomyolysis within the year prior to enrolment.

11. Pregnant or nursing females.

12. History of sensitivity to PPAR agonists.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
21/05/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
5/10/2023
Sample size
Target
Accrual to date
Final
213
Recruitment in Australia
Recruitment state(s)
NSW,SA,VIC
Recruitment hospital [1] 0 0
Royal North Shore Hospital - St. Leonards
Recruitment hospital [2] 0 0
PARC Clinical Research - Adelaide
Recruitment hospital [3] 0 0
The Alfred Hospital - Melbourne
Recruitment postcode(s) [1] 0 0
2065 - St. Leonards
Recruitment postcode(s) [2] 0 0
5000 - Adelaide
Recruitment postcode(s) [3] 0 0
3004 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
Massachusetts
Country [4] 0 0
United States of America
State/province [4] 0 0
New York
Country [5] 0 0
United States of America
State/province [5] 0 0
Ohio
Country [6] 0 0
United States of America
State/province [6] 0 0
Pennsylvania
Country [7] 0 0
United States of America
State/province [7] 0 0
Texas
Country [8] 0 0
Belgium
State/province [8] 0 0
Leuven
Country [9] 0 0
Canada
State/province [9] 0 0
Alberta
Country [10] 0 0
Canada
State/province [10] 0 0
British Columbia
Country [11] 0 0
Czechia
State/province [11] 0 0
Prague
Country [12] 0 0
Denmark
State/province [12] 0 0
Copenhagen
Country [13] 0 0
France
State/province [13] 0 0
Grand Est
Country [14] 0 0
France
State/province [14] 0 0
Hauts De France
Country [15] 0 0
France
State/province [15] 0 0
Ile-de-France
Country [16] 0 0
France
State/province [16] 0 0
Pays De La Loire
Country [17] 0 0
France
State/province [17] 0 0
Bron
Country [18] 0 0
France
State/province [18] 0 0
Nice
Country [19] 0 0
Germany
State/province [19] 0 0
Bonn
Country [20] 0 0
Germany
State/province [20] 0 0
Munich
Country [21] 0 0
Hungary
State/province [21] 0 0
Budapest
Country [22] 0 0
Hungary
State/province [22] 0 0
Pécs
Country [23] 0 0
Italy
State/province [23] 0 0
Lazio
Country [24] 0 0
Italy
State/province [24] 0 0
Lombardia
Country [25] 0 0
Italy
State/province [25] 0 0
Sicilia
Country [26] 0 0
Italy
State/province [26] 0 0
Toscana
Country [27] 0 0
Italy
State/province [27] 0 0
Bologna
Country [28] 0 0
Netherlands
State/province [28] 0 0
Nijmegen
Country [29] 0 0
New Zealand
State/province [29] 0 0
Auckland
Country [30] 0 0
Norway
State/province [30] 0 0
Bergen
Country [31] 0 0
Spain
State/province [31] 0 0
Barcelona
Country [32] 0 0
Spain
State/province [32] 0 0
Madrid
Country [33] 0 0
Spain
State/province [33] 0 0
Valencia
Country [34] 0 0
United Kingdom
State/province [34] 0 0
Greater London
Country [35] 0 0
United Kingdom
State/province [35] 0 0
Greater Manchester
Country [36] 0 0
United Kingdom
State/province [36] 0 0
Tyne And Wear

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Reneo Pharma Ltd
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This is a randomized, double-blind, placebo-controlled, parallel group, multi-centre, study
designed to investigate the efficacy and safety of REN001 administered once daily over a
24-week period to patients with PMM.
Trial website
https://clinicaltrials.gov/ct2/show/NCT04535609
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Amel Karaa, MD
Address 0 0
Massachusetts General Hospital (MGH)
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT04535609