A Study of the Safety of REN001 in Patients With McArdle... | NCT04226274

Studies
A Study of the Safety of REN001 in Patients With McArdle Disease

NCT04226274

Sponsor

Reneo Pharma Ltd

Status

Completed

Last Update Posted

Jan 12, 2022Actual

Enrollment

19Actual

Phase

Phase 1

Conditions

McArdle Disease

Interventions

REN001

Countries

Spain
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

Jan 2022

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Dec 10, 2019Actual

Primary Completion Date

Oct 11, 2021Actual

Completion Date

Oct 11, 2021Actual

First Submitted Date

Jan 9, 2020

First Submission Date that Met QC Criteria

Jan 10, 2020

First Posted Date

Jan 13, 2020Actual

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

Jan 11, 2022

Last Update Posted Date

Jan 12, 2022Actual

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

19Actual

Label	Type	Description	Intervention Names
REN001	Experimental	Oral	Drug: REN001

Name	Type	Description	Arm Group Labels	Other Names
REN001	Drug	Once daily for 12 weeks	REN001

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Adverse Events	Number of participants with Adverse Events (AEs) as a measure of safety and tolerability	up to Week 12

Secondary Outcomes

Not provided

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Subjects must give written, signed and dated informed consent
Confirmed diagnosis of McArdle Disease
Subjects must be able to remain on stable medication through the study and specifically must not commence or have changes to agents that affect metabolism such as medication for diabetes
Follow a stable dietary regimen as documented by a 3-day dietary record obtained during the screening period.

Exclusion Criteria:

Documented history of ongoing rhabdomyolysis
Evidence of acute crisis from their underlying disease
Currently following or planning to start a ketogenic diet
Treatment with an investigational drug within 1 month or within 5 half-lives, whichever is longer
Have been hospitalized within the 3 months prior to screening for any major medical condition
Severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the Investigator in discussion with the Medical Monitor, would make the subject inappropriate for entry into this study
Pregnant or nursing females

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

Not provided

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Rosaline Quinlivan, MD	MRC Centre for Neuromuscular Diseases, London	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Instituto de Investigación Hospital 12 de Octubre		Madrid		28041	Spain
National Hospital for Neurology and Neurosurgery, Queens Square

Condition Browse Module

MeSH Terms

ID	Term
D006012	Glycogen Storage Disease Type V

Ancestor Terms

ID	Term
D006008	Glycogen Storage Disease
D002239	Carbohydrate Metabolism, Inborn Errors
D008661	Metabolism, Inborn Errors
D030342	Genetic Diseases, Inborn

Browse Leaves

Not provided

Browse Branches

Not provided