A Study of the Safety of REN001 in Patients With Primary Mitochondrial Myopathy

Primary Mitochondrial Myopathy Clinical Trial

Official title:

An Open-label Study to Evaluate the Safety and Tolerability of 12 Weeks Treatment With Oral REN001 in Patients With Primary Mitochondrial Myopathy (PMM), With an Optional Extension of Treatment

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT03862846
• Other study ID #: REN001-101
• Status: Terminated
• Phase: Phase 1
• Start date: May 10, 2019
• Est. completion date: April 23, 2020

Study information

• Verified date: March 2020
• Source: Reneo Pharma Ltd
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to assess REN001 safety in subjects with primary mitochondrial myopathy

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 23
• Est. completion date: April 23, 2020
• Est. primary completion date: April 23, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 16 Years and older

Eligibility

Inclusion Criteria:

• Subjects must give written, signed and dated informed consent

• Confirmed diagnosis of PMM according to the 2016 Rome Consensus recommendations

• Confirmed mitochondrial mutation with evidence of myopathy

• Able to remain on stable medication throughout the study

Exclusion Criteria:

• Documented evidence of ongoing rhabdomyolysis

• Subjects with motor abnormalities other than related to mitochondrial disease

• Treatment with an investigational drug within 3 months prior to Day 1

• Hospitalised within 3 months prior to screening for any major medical condition

• Pregnant or nursing females

Related Conditions & MeSH terms

• Mitochondrial Myopathies
• Muscular Diseases
• Primary Mitochondrial Myopathy

Intervention

Drug:
• REN001
Once daily

Locations

Country	Name	City	State
United Kingdom	Institute of Neurology, University College London	London
United Kingdom	Wellcome Centre for Mitochondrial Research	Newcastle Upon Tyne

Sponsors (1)

Lead Sponsor	Collaborator
Reneo Pharma Ltd

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Adverse Events	Number of participants with Adverse Events as a measure of safety and tolerability	Comparing Baseline to Week 12
Secondary	Adverse Events	Number of participants with Adverse Events as a measure of safety and tolerability	Continous to Week 48