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Phase 2 study of TAK-831 (Takeda)

Description: FARA is announcing open enrollment for a Phase 2 study of TAK-831, sponsored by Takeda. This study will evaluate safety, tolerability, pharmacokinetics and efficacy of multiple doses of TAK-831 in adults with FA. Specifically, this study will look at upper limb motor function and manual dexterity of people who take TAK-831. The study will enroll 65 participants. Participants will be randomly assigned in a 2:1:2 ratio to one of the three treatment groups: TAK-831 high dose, TAK-831 low dose and placebo. Treatment duration is 3 months.

Key Criteria: Adults ages 18-55 years with genetically confirmed FA (homozygous for GAA repeat expansion or compound heterozygous with a point mutation). Must be able to complete the nine-hole pegboard test in less than 150 seconds. For more information on inclusion and exclusion criteria, please contact one of the study site coordinators.

Study Flyer: Click HERE to view the Takeda Study Flyer.

Locations: This study is taking place at several medical centers in the United States.

Children's Hospital of Philadelphia, PA - View the CHOP Study Flyer
Contacts: McKenzie Wells, 267-426-9608 -
or Maya Patel, 267-426-7584 -

University of Iowa, Iowa City, IA
Contact: Carrie Stephan, 319-356-2673 -

University of California Los Angeles, CA
Contact: Aaron Fisher, 310-206-8153 -
or Arjun Sarkar -

University of South Florida, Tampa FL
Contact: Daniel Ekberg, 813-974-5909 -

Ohio State University, Columbus, OH
Contact: Tabi Alexander, 614-293-6953 -

University of Florida, Gainesville, FL
Contact: Blake Meyer, (352) 294-8754 -

Learn more: Here is a brief video where Kyle Bryant talks with Dr. Hao Wang about Takeda, TAK-831 and this study. posting -

Magnetic Resonance Imaging (MRI) Muscle Phenotyping in Mitochondrial Disease

Description: The purpose of the study is to use a new research imaging technique, a kind of magnetic resonance imaging (MRI), to measure important metabolic features of muscle, including mitochondrial function, in people with mitochondrial disease and in healthy individuals.

Key Criteria: Ages 18-65 years

Location: University of Pennsylvania -

It is hoped that this new strategy will help physicians to understand better the health problems of people with mitochondrial disease. Eventually, this could lead to better diagnostic and treatment approaches.




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