Trials Sponsors Conditions Drugs Pricing

Conditions

Primary Mitochondrial Myopathy

Outcome Measures

Primary Outcomes (1)

Number of Participants With Mild, Moderate, Severe TEAEs, TEAEs Leading to Study Discontinuation, All TEAEs and All TESAEs
Number of participants with AEs and severity
Baseline through study termination, an average of 12.1 months

Secondary Outcomes (9)

Absolute Values, Changes From Baseline, and Incidence of Potentially Clinically Significant Changes in Laboratory Safety Tests, Electrocardiograms, Supine Vital Signs, and Eye Assessments
Study Termination
Change in Distance Walked During a 12 Minute Walk Test
Baseline to Month 24
Change in Modified Fatigue Impact Scale (MFIS) Score
Baseline to Month 24
Change in Patient Global Impression of Severity (PGIS) Score
Baseline to Month 24
Change in Brief Pain Inventory (BPI) Score
Baseline to Month 24
+4 more secondary outcomes

Study Arms (1)

REN001

EXPERIMENTAL

100 mg once daily

Drug: REN001

Interventions

REN001Drug

Once daily dosing

REN001

Eligibility Criteria

Age18 Years+

Sexall

Healthy VolunteersNo

Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

mtDNA-PMM subjects: Completed treatment in STRIDE or was participating in Study REN001-101 when the study stopped due to the COVID-19 pandemic, 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.

Sponsors & Collaborators

Reneo Pharma Ltd
Industrylead

Study Sites (29)

Royal North Shore Hospital

St Leonards, New South Wales, 2065, Australia

PARC Clinical Research

Adelaide, South Australia, 5000, Australia

The Alfred Hospital

Melbourne, Victoria, 3004, Australia

University Hospital Leuven

Leuven, 3000, Belgium

M.A.G.I.C. Clinic (Metabolics and Genetics in Calgary)

Calgary, Alberta, T2E 7Z4, Canada

Vancouver General Hospital

Vancouver, V5Z 1M9, Canada

Copenhagen Neuromuscular Center

Copenhagen, 2100, Denmark

Results Point of Contact

Title: Alex Dorenbaum, MD, Chief Medical Officer
Organization: Reneo Pharmaceuticals, Inc.

Study Officials

Grainne Gorman, MD
Newcastle Hospital NHS Foundation Trust
PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee: No
Restriction Type: OTHER
Restrictive Agreement: Yes

Enrollment

155 participants (actual enrollment reported by the registry).

Study Design

Study Type: interventional
Phase: phase 2 3
Allocation: NA
Masking: NONE
Purpose: TREATMENT
Intervention Model: SINGLE GROUP
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR

Study Record Dates

First Submitted

February 24, 2022

First Posted

March 4, 2022

Study Start

February 1, 2022

Actual (reported)

You may not qualify if:

Anticipated to need a peroxisome proliferator-activated receptor (PPAR) agonist other than REN001 during the study.
Intent to donate blood, or blood components during the study or within one month after completion of the study.
Current drug dependency. Use of opiates/cannabis for medical reasons is acceptable with prescription evidence or at the Investigator's discretion.
Current alcohol dependency.
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.
Pregnant or nursing female
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.
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.
Subjects with uncontrolled diabetes and/or a Screening HbA1c of ≥11%.
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.)
Subjects with a history of cancer. A history of in situ basal cell carcinoma in the skin is allowed.
Clinically significant cardiac disease and/or clinically significant ECG abnormalities such as 2nd degree heart block, symptomatic tachyarrhythmia or unstable arrhythmia (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.

Study Stopped

An Open Label, Long Term Safety Study of REN001 in Primary Mitochondrial Myopathy Patients (Stride Ahead)

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

Study Start

First Submitted

First Posted

Primary Completion

Study Completion

Results Posted

Conditions

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (9)

Study Arms (1)

REN001

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (29)

Results Point of Contact

Study Officials

Publication Agreements

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Source record

Documents

Locations

Study Stopped

An Open Label, Long Term Safety Study of REN001 in Primary Mitochondrial Myopathy Patients (Stride Ahead)

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

Study Start

First Submitted

First Posted

Primary Completion

Study Completion

Results Posted

Conditions

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (9)

Study Arms (1)

REN001

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (29)

Results Point of Contact

Study Officials

Publication Agreements

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Source record

Documents

Locations