McArdle Disease
Conditions
Brief summary
The purpose of this study is to assess REN001 safety in subjects with McArdle Disease
Interventions
DRUGREN001
Once daily for 12 weeks
Sponsors
Reneo Pharma Ltd
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Adverse Events | up to Week 12 | Number of participants with Adverse Events (AEs) as a measure of safety and tolerability |
Countries
Spain, United Kingdom
Outcome results
None listed