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

McArdle Disease Clinical Trial

Official title:

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04226274
• Other study ID #: REN001-103
• Status: Completed
• Phase: Phase 1
• Start date: December 10, 2019
• Est. completion date: October 11, 2021

Study information

• Verified date: January 2022
• Source: Reneo Pharma Ltd
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 19
• Est. completion date: October 11, 2021
• Est. primary completion date: October 11, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Glycogen Storage Disease Type V
• McArdle Disease

Intervention

Drug:
• REN001
Once daily for 12 weeks

Locations

Country	Name	City	State
Spain	Instituto de Investigación Hospital 12 de Octubre	Madrid
United Kingdom	National Hospital for Neurology and Neurosurgery, Queens Square	London

Sponsors (1)

Lead Sponsor	Collaborator
Reneo Pharma Ltd

Countries where clinical trial is conducted

Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Adverse Events	Number of participants with Adverse Events (AEs) as a measure of safety and tolerability	up to Week 12