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Trial record 1 of 1 for:    04535609
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A Study of the Efficacy and Safety of 24 Week Treatment With REN001 in Patients With Primary Mitochondrial Myopathy (STRIDE)

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ClinicalTrials.gov Identifier: NCT04535609
Recruitment Status : Not yet recruiting
First Posted : September 2, 2020
Last Update Posted : September 2, 2020
Sponsor:
Information provided by (Responsible Party):
Reneo Pharma Ltd

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.

Condition or disease Intervention/treatment Phase
Primary Mitochondrial Myopathy Drug: REN001 Drug: Placebo Phase 2 Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 200 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official 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
Estimated Study Start Date : February 2021
Estimated Primary Completion Date : December 2022
Estimated Study Completion Date : December 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Muscle Disorders

Arm Intervention/treatment
Experimental: REN001
Once daily
Drug: REN001
Once daily

Placebo Comparator: Matched placebo
Once daily
Drug: Placebo
Once daily




Primary Outcome Measures :
  1. Change from Baseline in distance walked during the Walk Test [ Time Frame: Week 24 ]
    Distance walked in meters


Secondary Outcome Measures :
  1. Change from Baseline at Week 24 in the Modified Fatigue Impact Scale (MFIS) physical sub-scale score [ Time Frame: Week 24 ]
    Physical Scale 0-36 scoring

  2. Patient Global Impression of Change (PGIC) score at Week 24 [ Time Frame: Week 24 ]
    7 point scale: Very much improved, Moderately improved, Minimally improved, No change, Minimally worse, Moderately worse, Very much worse


Other Outcome Measures:
  1. Incidence and severity of adverse events, serious adverse events, and withdrawals due to adverse events [ Time Frame: Baseline to Week 24 ]
    Number and Severity

  2. Change from Baseline in number of sit to stands in the sit to stand test [ Time Frame: Week 24 ]
    Individual counts

  3. Change from Baseline in number of steps [ Time Frame: Week 24 ]
    Step count from pedometer

  4. Change from Baseline in Patient Global Impression of Severity (PGIS) score [ Time Frame: Week 24 ]
    5 point scale: Absent, Mild, Moderate, Severe, Very Severe

  5. Change from Baseline in MFIS total, cognitive and psychosocial sub-scale scores [ Time Frame: Week 24 ]
    Total MFIS 0-84, cognitive 0-40, psychosocial 0-8

  6. Change from Baseline in Brief Pain Inventory pain severity and pain interference scores [ Time Frame: Week 24 ]
    Numerical rating scale 0-10



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04535609


Contacts
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Contact: Clinical Trial Coordinator +44 (0)1304 809360 clintrialinfo@reneopharma.com

Sponsors and Collaborators
Reneo Pharma Ltd
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Responsible Party: Reneo Pharma Ltd
ClinicalTrials.gov Identifier: NCT04535609    
Other Study ID Numbers: REN001-201
First Posted: September 2, 2020    Key Record Dates
Last Update Posted: September 2, 2020
Last Verified: August 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Muscular Diseases
Mitochondrial Myopathies
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Mitochondrial Diseases
Metabolic Diseases