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.
Locations: This study is taking place at several medical centers in the United States.
Children's Hospital of Philadelphia, PA - View Study Flyer
Contacts: McKenzie Wells, 267-426-9608 - firstname.lastname@example.org
or Maya Patel, 267-426-7584 - email@example.com
University of Iowa, Iowa City, IA
Contact: Carrie Stephan, 319-356-2673 - firstname.lastname@example.org
University of California Los Angeles, CA
Contact: Aaron Fisher, 310-206-8153 - email@example.com
or Arjun Sarkar, firstname.lastname@example.org
University of South Florida, Tampa FL
Contact: USF Ataxia Center, 813-974-5909
Ohio State University, Columbus, OH (Not Yet Enrolling)
Contact: To Be Posted
Learn more: Here is a brief video where Kyle Bryant talks with Dr. Hao Wang about Takeda, TAK-831 and this study.
Clinicaltrials.gov posting - www.clinicaltrials.gov/ct2/show/NCT03214588