A Study of the Safety of REN001 in Patients With Primary... | NCT03862846

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

NCT03862846

Sponsor

Reneo Pharma Ltd

Status

Terminated

Last Update Posted

Jul 16, 2020Actual

Enrollment

23Actual

Phase

Phase 1

Conditions

Primary Mitochondrial Myopathy

Interventions

REN001

Countries

United Kingdom

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Mar 2020

Overall Recruitment Status or Expanded Access Status

Terminated

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

COVID-19 Pandemic sufficient data gathered to achieve the study objective

Expanded Access Info

Start Date

May 10, 2019Actual

Primary Completion Date

Apr 23, 2020Actual

Completion Date

Apr 23, 2020Actual

First Submitted Date

Feb 28, 2019

First Submission Date that Met QC Criteria

Mar 1, 2019

First Posted Date

Mar 5, 2019Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Jul 14, 2020

Last Update Posted Date

Jul 16, 2020Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 1

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

23Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Group	Experimental	REN001 Low Dose	Drug: REN001

Interventions

Name	Type	Description	Arm Group Labels	Other Names
REN001	Drug	Once daily	Group

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Adverse Events	Number of participants with Adverse Events as a measure of safety and tolerability	Comparing Baseline to Week 12

Secondary Outcomes

Measure	Description	Time Frame
Adverse Events	Number of participants with Adverse Events as a measure of safety and tolerability	Continous to Week 48

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

16 Years

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Grainne Gorman, MD	Wellcome Centre for Mitochondrial Research	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Institute of Neurology, University College London		London		WC1N 3BG	United Kingdom
Wellcome Centre for Mitochondrial Research