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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

19 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-12-10

Study Completion Date

2021-10-11

Brief Summary

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

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Detailed Description

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Conditions

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McArdle Disease

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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REN001

Oral

Group Type EXPERIMENTAL

REN001

Intervention Type DRUG

Once daily for 12 weeks

Interventions

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REN001

Once daily for 12 weeks

Intervention Type DRUG

Eligibility Criteria

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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

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No