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

A Phase 1b, Open-label Study to Evaluate the Safety and Tolerability of 12 Weeks Treatment With Oral REN001 in Patients With McArdle Disease (Glycogen Storage Disorder 5)

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

Study Overview

• Status: Completed
• Conditions: McArdle Disease
• Intervention / Treatment: Drug: REN001

• Study Type: Interventional
• Enrollment (Actual): 19
• Phase: Phase 1

Contacts and Locations

Study Locations

• Spain
  • Madrid, Spain, 28041
    • Instituto de Investigación Hospital 12 de Octubre
• United Kingdom
  • London, United Kingdom, WC1 3BG
    • National Hospital for Neurology and Neurosurgery, Queens Square

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: REN001; Oral	Drug: REN001; Once daily for 12 weeks

What is the study measuring?

Primary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Reneo Pharma Ltd
• Investigators: Principal Investigator: Rosaline Quinlivan, MD, MRC Centre for Neuromuscular Diseases, London

Study record dates

Study Major Dates

• Study Start (Actual): December 10, 2019
• Primary Completion (Actual): October 11, 2021
• Study Completion (Actual): October 11, 2021

Study Registration Dates

• First Submitted: January 9, 2020
• First Submitted That Met QC Criteria: January 10, 2020
• First Posted (Actual): January 13, 2020

Study Record Updates

• Last Update Posted (Actual): January 12, 2022
• Last Update Submitted That Met QC Criteria: January 11, 2022
• Last Verified: January 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Genetic Diseases, Inborn; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Glycogen Storage Disease; Glycogen Storage Disease Type V
• Other Study ID Numbers: REN001-103

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No