Advancing Clinical Trials for FXTAS

Unlike a typical grant, the funding for the ACT for FXTAS study is a cooperative agreement where NINDS scientists actively participate in the project, ensuring close collaboration. The goal is to be fully prepared for upcoming clinical trials for FXTAS — knowing exactly what to measure and how to measure it.

The NFXF is truly excited about this initiative. We recently committed to funding the biobanking effort of this study because strong biomarkers and outcome measures are essential for developing effective therapies. This biobank promises to accelerate discoveries and bring us closer to meaningful, measurable outcomes for those living with FXTAS. We’re proud to support this work and inspired by the dedication driving it forward.

Also see: $55 Million Federal Grant to Help Researchers Build Tools for FXTAS Treatment Trials (UC Davis Health News)

Key Takeaways

• The ACT for FXTAS project recently received $5.5 million in federal funding from NINDS, providing crucial support to prepare for future clinical trials.
• Researchers from UC Davis, University of Michigan, and Rush University are working together to advance FXTAS research.
• Scientists from NINDS are active partners in the project, helping guide milestones and study design.
• The project is preparing for new treatments, including gene therapies and other targeted approaches for FXTAS.
• A strong research network is being built across multiple centers, with trained staff, standardized equipment, and ready participants.
• Patients and their families are deeply involved through advisory boards and focus groups to ensure the research is meaningful and patient-friendly.
• Advanced tools, such as MRI scans, rating scales, and wearable sensors, are being used to track symptoms and disease progression.
• The NFXF is helping by providing funding support for the project’s biobank, helping collect and store patient samples for future biomarker studies.
• Studying this rare disease also helps scientists learn more about other movement disorders and neurodegenerative conditions.
• Federal funding is essential because industry rarely supports early-stage clinical trial preparation for rare diseases.
• The study plans to enroll 100 participants over the next two years, and families are encouraged to contact site coordinators if they are interested in joining.

Interested in Participating?

The study is looking for English-speaking men and women ages 50 to 85 with a diagnosis of FXTAS.

The study will involve:

• Motor function assessments
• Medical history and physical exam
• Cognitive testing
• Brain magnetic resonance imaging (MRI)
• Blood draw
• Questionnaires

Participants will receive compensation for their time and reimbursement of all study-approved travel expenses to attend clinic visits.

If you’d like to learn more and be considered for this research opportunity, please fill out this form and your contact information will be sent to the study team.