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

NCT04226274 | Completed Phase 1

• 1 other identifier: REN001-103
• Study Type: interventional
• Target: 19
• Locations: 2 countries
• Sites: 2

Brief Summary

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

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

REN001 DRUG

Once daily for 12 weeks

REN001

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

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

Sponsors & Collaborators

• Reneo Pharma Ltd: lead

Study Sites (2)

Instituto de Investigación Hospital 12 de Octubre

Madrid, 28041, Spain

Location

National Hospital for Neurology and Neurosurgery, Queens Square

London, WC1 3BG, United Kingdom

Location

MeSH Terms

Conditions

Glycogen Storage Disease Type V

Condition Hierarchy (Ancestors)

Glycogen Storage Disease; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Rosaline Quinlivan, MD

  MRC Centre for Neuromuscular Diseases, London

  PRINCIPAL INVESTIGATOR

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
• Primary Completion: October 11, 2021
• Study Completion: October 11, 2021
• Last Updated: January 12, 2022

Record last verified: 2022-01

Locations

ES (1); GB (1)