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

• Sponsor: Reneo Pharma Ltd (Industry)
• Overall Status: Completed
• CT.gov ID: NCT04226274
• Collaborator: (none)
• Enrollment: 19
• Locations: 2
• Arm: 1
• Actual Duration (Months): 22
• Patients Per Site: 9.5
• Patients Per Site Per Month: 0.4

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
McArdle Disease	Drug: REN001	Phase 1

Study Design

• Study Type: Interventional
• Actual Enrollment: 19 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• 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)
• Actual Study Start Date: Dec 10, 2019
• Actual Primary Completion Date: Oct 11, 2021
• Actual Study Completion Date: Oct 11, 2021

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures

1. Adverse Events [up to Week 12]

   Number of participants with Adverse Events (AEs) as a measure of safety and tolerability

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Instituto de Investigación Hospital 12 de Octubre	Madrid		Spain	28041
2	National Hospital for Neurology and Neurosurgery, Queens Square	London		United Kingdom	WC1 3BG

Sponsors and Collaborators

• Reneo Pharma Ltd

Investigators

• Principal Investigator: Rosaline Quinlivan, MD, MRC Centre for Neuromuscular Diseases, London

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Reneo Pharma Ltd
• ClinicalTrials.gov Identifier: NCT04226274
• Other Study ID Numbers: REN001-103
• First Posted: Jan 13, 2020
• Last Update Posted: Jan 12, 2022
• Last Verified: Jan 1, 2022

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Glycogen Storage Disease Type V

Study Results

No Results Posted as of Jan 12, 2022