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

Reneo Pharma

Status and phase

Completed

Phase 1

Conditions

McArdle Disease

Treatments

Drug: REN001

Study type

Interventional

Funder types

Industry

Identifiers

REN001-103

Details and patient eligibility

About

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

Enrollment

19 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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