Back to resultsA Study of the Safety of REN001 in Patients With McArdle Disease
Primary Purpose
McArdle Disease
Status
Completed
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
REN001
Sponsored by
About this trial
This is an interventional treatment trial for McArdle Disease
Eligibility 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
Sites / Locations
- Instituto de Investigación Hospital 12 de Octubre
- National Hospital for Neurology and Neurosurgery, Queens Square
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
REN001
Arm Description
Oral
Outcomes
Primary Outcome Measures
Adverse Events
Number of participants with Adverse Events (AEs) as a measure of safety and tolerability
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04226274
Brief Title
A Study of the Safety of REN001 in Patients With McArdle Disease
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)
Study Type
Interventional
2. Study Status
Record Verification Date
January 2022
Overall Recruitment Status
Completed
Study Start Date
December 10, 2019 (Actual)
Primary Completion Date
October 11, 2021 (Actual)
Study Completion Date
October 11, 2021 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Reneo Pharma Ltd
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
The purpose of this study is to assess REN001 safety in subjects with McArdle Disease
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
McArdle Disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
19 (Actual)
8. Arms, Groups, and Interventions
Arm Title
REN001
Arm Type
Experimental
Arm Description
Oral
Intervention Type
Drug
Intervention Name(s)
REN001
Intervention Description
Once daily for 12 weeks
Primary Outcome Measure Information:
Title
Adverse Events
Description
Number of participants with Adverse Events (AEs) as a measure of safety and tolerability
Time Frame
up to Week 12
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Rosaline Quinlivan, MD
Organizational Affiliation
MRC Centre for Neuromuscular Diseases, London
Official's Role
Principal Investigator
Facility Information:
Facility Name
Instituto de Investigación Hospital 12 de Octubre
City
Madrid
ZIP/Postal Code
28041
Country
Spain
Facility Name
National Hospital for Neurology and Neurosurgery, Queens Square
City
London
ZIP/Postal Code
WC1 3BG
Country
United Kingdom
12. IPD Sharing Statement
Learn more about this trial
A Study of the Safety of REN001 in Patients With McArdle Disease
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