Phase
Condition
Spinocerebellar Disorders
Friedreich's Ataxia
Dyskinesias
Treatment
Nomlabofusp
Placebo
Clinical Study ID
Ages 2-17 All Genders
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
Subject has genetically confirmed diagnosis of FRDA manifested by homozygous GAArepeat expansions, with repeat sizing (if available) included on the diagnosisreport.
Male or female subjects ≥ 2 to < 18 years of age at screening.
Subjects must weigh ≥ 10.0 kg.
Subject must be able to traverse a distance of 25 feet with or without someassistive device (e.g., cane, walker, crutches, self-propelled wheelchair) and meetthe following requirements:
Be able to sit upright with thighs together and arms crossed without requiringsupport on more than 2 sides;
Be able to transfer from bed to chair independently or with assistance if, inthe opinion of the investigator, the degree of physical disability does notresult in undue risk to the subject while participating in the study; and
Perform basic age-appropriate daily care, such as feeding themselves andpersonal hygiene, with minimal assistance.
Exclusion
Exclusion Criteria:
Subjects who are confirmed as compound heterozygous (GAA repeat expansion on only 1allele) for FRDA.
Subject has any condition, disease, or situation, including a cardiac condition ordisease, that in the opinion of the investigator could confound the results of thestudy or put the subject at undue risk, making participation inadvisable.
Subjects currently receiving or having received omaveloxolone within 30 days priorto Screening.
Study Design
Study Description
Connect with a study center
Uncommon Cures
Chevy Chase, Maryland 20815
United StatesActive - Recruiting
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