NCT03862846

Brief Summary

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

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
23

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started May 2019

Geographic Reach
1 country

2 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

February 28, 2019

Completed
5 days until next milestone

First Posted

Study publicly available on registry

March 5, 2019

Completed
2 months until next milestone

Study Start

First participant enrolled

May 10, 2019

Completed
12 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 23, 2020

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 23, 2020

Completed
Last Updated

July 16, 2020

Status Verified

March 1, 2020

Enrollment Period

12 months

First QC Date

February 28, 2019

Last Update Submit

July 14, 2020

Conditions

Primary Mitochondrial Myopathy

Outcome Measures

Primary Outcomes (1)

  • Adverse Events

    Number of participants with Adverse Events as a measure of safety and tolerability

    Comparing Baseline to Week 12

Secondary Outcomes (1)

  • Adverse Events

    Continous to Week 48

Study Arms (1)

Group

EXPERIMENTAL

REN001 Low Dose

Drug: REN001

Interventions

REN001DRUG

Once daily

Group

Eligibility Criteria

Age16 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Institute of Neurology, University College London

London, WC1N 3BG, United Kingdom

Location

Wellcome Centre for Mitochondrial Research

Newcastle upon Tyne, NE2 4HH, United Kingdom

Location

Study Officials

  • Grainne Gorman, MD

    Wellcome Centre for Mitochondrial Research

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 28, 2019

First Posted

March 5, 2019

Study Start

May 10, 2019

Primary Completion

April 23, 2020

Study Completion

April 23, 2020

Last Updated

July 16, 2020

Record last verified: 2020-03

Locations

GB(2)