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BioMarin Pharmaceutical & Sanguine Biosciences -Biomarker Research Study

Description: BioMarin Pharmaceutical and Sanguine Biosciences are seeking additional individuals with FA and FA Carriers (biological parents and genetically confirmed siblings) who live in Northern California, in the extended San Francisco Bay Area, to participate in their Biomarker Research study.

The purpose of the study is to explore potential biomarkers that may serve as biochemical measurements of the effects of FA, and may be used to monitor therapeutic effects of a future treatment.

Key Inclusion Criteria: Adults 18 years and older who were diagnosed with FA prior to age 20, or a related Carrier has one FA disease allele (biological parent or genetically confirmed siblings). Individuals or Carriers who have GAA repeat length >100 and/or a single nucleotide point mutation. Patients must be able to donate blood 4 times over 4-6 months, at home visits, and must live in the extended San Francisco Bay Area. Carriers may donate blood 1 time.

To learn more about the Inclusion and Exclusion Criteria and the process for participation in the study, including the link to the Study Landing Page, see the attached flyer below.

Please contact the Clinical Research Coordinator with any questions.

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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 -

Cohort 9, MOXIe, Phase 2 Study of RTA 408

We are pleased to announce that 62 patients have been enrolled in the MOXIe study. Thank you to all those who have contacted coordinators about this study and are participating. The study sites need more volunteers to be enrolled to complete the next and final cohort for Part 1 of the study.

Reata and the clinical sites are preparing for the ninth cohort of 8 patients to test at a dose of 300mg of study drug at the following sites:

· University of Florida, Gainesville, FL
· Ohio State University, Columbus, OH
· University of South Florida, Tampa, FL
· Emory University, Atlanta, GA
· Children's Hospital of Philadelphia, Philadelphia, PA
· University of California, Los Angeles, CA
· Murdoch Children's Research Institute, Melbourne, Australia
· Medical University Innsbruck, Austria

Before you call or email the study coordinators, please note the following:
- You must be between ages 16-40 to participate (some sites are age 18-40)
- You must have a confirmed diagnosis of Friedreich ataxia (confirmed by formal genetic testing)
- You must be able to perform maximal exercise testing on a recumbent bicycle for at least 10 minutes
- You are only permitted to participate in one clinical drug trial at a time. If you are currently participating in a different drug study, and have questions about this rule and your options, please contact the study coordinator or study doctor in charge of the study that you are currently participating in.

**If you are reading this as a parent of an individual with Friedreich ataxia, please note that you will not be allowed to enroll your child in this study if he/she is over age 18. Instead, please forward this email and have your son/daughter contact us directly.

This will be a multi-site study. A list of sites can be found HERE.

Study Coordinators:

University of Florida: Aika Konn/ Tel.: (352) 273-6003; Email:

The Ohio State University: Louisa Mezache / Tel.: (614)-685-3030; Email:

University of South Florida: Kevin Allison, study coordinator/ Tel.: (813) 974-5909; Email:

Emory University: Becky McMurray / Tel.: 404-712-7013; Email:

University of California Los Angeles: Aaron Fisher 310-206-8153

Children's Hospital of Philadelphia: Lauren Seyer/Tel.: (267) 426-9738; Email:

Thank you very much for your help and for your ongoing support of clinical research in Friedreich ataxia.
tel: 484-879-6160

A Study of the Safety and Effectiveness of (+) Epicatechin for Treating Patients with Friedreich's Ataxia

Description: The purpose of this study is to test the safety and effectiveness of synthetically produced (+) Epicatechin for treating patients who have Friedreich's Ataxia, a neurological disorder.

Key Criteria: Ages 10-50 years

Location: Mayo Clinic (Rochester, MN) -

Please review the study flyer below, and contact the Study Coordinator with any questions.

Study Flyer PDF



A study of walking and balance in Friedreich's Ataxia

Description: The USF Ataxia Research Center, located at the Morsani Center for Advanced Health Care, is looking for people with Friedreich’s ataxia for a research study to evaluate changes in walking and balance in people with Friedreich’s ataxia. The study involves 3-4 single day visits over a 12 month period.

Key Criteria: Ages 7 and older

Location: USF Ataxia Research Center (Tampa, FL)

Please review the study advertisement below and contact the Study Coordinator, phone: 813-974-5909 or email: with any questions.

Study Flyer PDF



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