NCT04226274

CompletedPhase 1

A Study of the Safety of REN001 in Patients With McArdle Dis...

Reneo Pharma Ltd Source

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Other study identifiers

Sponsor study identifier

REN001-103

Study Type

interventional

Actual enrollment (reported)

19

Locations

2 countries

Sites

2

Timeline

Study Start

December 10, 2019

Actual (reported)

Primary Completion

October 11, 2021

Actual (reported)

Study Completion

October 11, 2021

Actual (reported)

Brief Summary

The purpose of this study is to assess REN001 safety in subjects with McArdle Disease

Registry Overview

Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.

Recruitment status: completed
Actual enrollment (reported): 19 participants
Study completion date: October 11, 2021
Actual (reported)
Listed locations: 2 countries · 2 sites

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.

Study Start
December 10, 2019
Actual (reported)
First Submitted
January 9, 2020
Actual (reported)
First Posted
January 13, 2020
Actual (reported)
Primary Completion
October 11, 2021
Actual (reported)
Study Completion
October 11, 2021
Actual (reported)

Last Updated: January 12, 2022
Status Verified: January 2022
Last Update Submitted: January 11, 2022

Conditions

McArdle Disease

Outcome Measures

Primary Outcomes (1)

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

Study Arms (1)

REN001

EXPERIMENTAL

Oral

Drug: REN001

Interventions

REN001Drug

Once daily for 12 weeks

REN001

Eligibility Criteria

Age18 Years+

Sexall

Healthy VolunteersNo

Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

Subjects must give written, signed and dated informed consent
Confirmed diagnosis of McArdle Disease

Sponsors & Collaborators

Reneo Pharma Ltd
Industrylead

Study Sites (2)

Instituto de Investigación Hospital 12 de Octubre

Madrid, 28041, Spain

National Hospital for Neurology and Neurosurgery, Queens Square

London, WC1 3BG, United Kingdom

MeSH Terms

Conditions

Glycogen Storage Disease Type V

Condition Hierarchy (Ancestors)

Glycogen Storage DiseaseCarbohydrate Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

Rosaline Quinlivan, MD
MRC Centre for Neuromuscular Diseases, London
PRINCIPAL INVESTIGATOR

Enrollment

19 participants (actual enrollment reported by the registry).

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

January 9, 2020

First Posted

January 13, 2020

Study Start

December 10, 2019

Actual (reported)

NCT04694547

Ketogenic Diet Survey in Patients With McArdle Disease (GSDV)

Completed

NCT03843606

Modified Ketogenic Diet in Patients With McArdle Disease Part A

Not ApplicableCompleted

NCT03844022

MRI in McArdle Disease (GSDV)

Completed

NCT03112889

Sodium Valproate for GSDV

Phase 2Completed

NCT02919631

Triheptanoin in Mc Ardle

Phase 2Completed

NCT02432768

The Effect of Triheptanoin in Adults With McArdle Disease (Glycogen Storage Disease Type V)

Phase 2Completed

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.

You may not qualify if:

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

Contact the study team to confirm eligibility.

Primary Completion

October 11, 2021

Actual (reported)

Study Completion

October 11, 2021

Actual (reported)

Last Updated

January 12, 2022

Record last verified: 2022-01

Source record

Download the complete ClinicalTrials.gov record imported by Syfrah, including fields not shown in this page. The registry may contain a newer revision.

Source: ClinicalTrials.gov.

JSON downloads include source attribution and retrieval details alongside the unchanged original record or the separately normalized values.

Registry identifier: NCT04226274
Data provider: ClinicalTrials.gov
Source updated: 2022-01-12
Normalization version: ctgov-legacy-1

Open registry record

Source retrieved: 2026-10-25 22:32:48 UTC

Locations

ES(1)GB(1)

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

Study Start

First Submitted

First Posted

Primary Completion

Study Completion

Conditions

Outcome Measures

Primary Outcomes (1)

Study Arms (1)

REN001

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (2)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Study Officials

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Source record

Locations