Accept Cookies?
Provided by OpenGlobal E-commerce

Please wait while your page loads ...

FARAFARA Cure FA

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 - wellsm@email.chop.edu
or Maya Patel, 267-426-7584 - patelm2@email.chop.edu

University of Iowa, Iowa City, IA
Contact: Carrie Stephan, 319-356-2673 - carrie-stephan@uiowa.edu

University of California Los Angeles, CA
Contact: Aaron Fisher, 310-206-8153 - adfisher@mednet.ucla.edu
or Arjun Sarkar - asarkar@mednet.ucla.edu

University of South Florida, Tampa FL
Contact: Daniel Ekberg, 813-974-5909 - daniel.ekberg@health.usf.edu

Ohio State University, Columbus, OH
Contact: Tabi Alexander, 614-293-6953 - tabitha.alexander@osumc.edu

University of Florida, Gainesville, FL
Contact: Blake Meyer, (352) 294-8754 - blakemeyer@ufl.edu

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

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

ClinicalTrials.gov - https://clinicaltrials.gov/ct2/show/NCT02154711

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.

 

 

SHARE

FacebookTwitterLinkedInYoutube
michelle-h.jpg