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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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04226274
Recruitment Status : Recruiting
First Posted : January 13, 2020
Last Update Posted : January 13, 2020
Sponsor:
Information provided by (Responsible Party):
Reneo Pharma Ltd

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

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 16 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
Estimated Primary Completion Date : March 2021
Estimated Study Completion Date : March 2021


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




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



Information from the National Library of Medicine

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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

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


Contacts
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Contact: Lynn Purkins, PhD +44 (0) 1304 809360 lpurkins@reneopharma.com

Locations
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Spain
Instituto de Investigación Hospital 12 de Octubre Recruiting
Madrid, Spain, 28041
Principal Investigator: Cristina Domínguez González, MD         
United Kingdom
National Hospital for Neurology and Neurosurgery, Queens Square Recruiting
London, United Kingdom, WC1 3BG
Principal Investigator: Roseline Quinlivan, MD         
Sponsors and Collaborators
Reneo Pharma Ltd
Investigators
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Principal Investigator: Rosaline Quinlivan, MD MRC Centre for Neuromuscular Diseases, London

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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 13, 2020
Last Verified: January 2020

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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