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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04226274
Recruitment Status : Completed
First Posted : January 13, 2020
Last Update Posted : January 12, 2022
Sponsor:
Information provided by (Responsible Party):
Reneo Pharma Ltd

Study Description
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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
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Study Type : Interventional  (Clinical Trial)
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 : December 10, 2019
Actual Primary Completion Date : October 11, 2021
Actual Study Completion Date : October 11, 2021

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: REN001
Oral
 Drug: REN001
Once daily for 12 weeks


Outcome Measures
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Primary Outcome Measures :
  1. Adverse Events [ Time Frame: up to Week 12 ]
    Number of participants with Adverse Events (AEs) as a measure of safety and tolerability


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04226274


Locations
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Spain
Instituto de Investigación Hospital 12 de Octubre
Madrid, Spain, 28041
United Kingdom
National Hospital for Neurology and Neurosurgery, Queens Square
London, United Kingdom, WC1 3BG
Sponsors and Collaborators
Reneo Pharma Ltd
Investigators
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Principal Investigator: Rosaline Quinlivan, MD MRC Centre for Neuromuscular Diseases, London
More Information
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Responsible Party: Reneo Pharma Ltd
ClinicalTrials.gov Identifier: NCT04226274    
Other Study ID Numbers: REN001-103
First Posted: January 13, 2020    Key Record Dates
Last Update Posted: January 12, 2022
Last Verified: January 2022

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Glycogen Storage Disease Type V
Glycogen Storage Disease
Carbohydrate Metabolism, Inborn Errors
 Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases