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

Reneo Pharma

Status and phase: Terminated; Phase 1

Conditions: Primary Mitochondrial Myopathy

Treatments: Drug: REN001

Study type: Interventional

Funder types: Industry

Identifiers

NCT03862846

REN001-101

Details and patient eligibility

About

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

Enrollment: 23 patients

Sex: All

Ages: 16+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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23 participants in 1 patient group

Group

Experimental group

Description:

REN001 Low Dose

Treatment:

Drug: REN001