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Completed

NCT Number: NCT04226274

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

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

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Key information

Conditions

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

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Instituto de Investigación Hospital 12 de Octubre, Madrid, Spain

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

REN001

Drug

Once daily for 12 weeks

Primary outcomes

  1. Adverse Events

    Time frame: up to Week 12

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

Sponsors and collaborators

Lead sponsor

Reneo Pharma Ltd

Industry

Registry information

Official study 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)

Important dates

Study start
2019
Primary completion
2021
Study completion
2021
First posted
Jan 13, 2020
Registry last updated
Jan 12, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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