TRACK-FA Clinical Trial Seeks Participants

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by Steve Bryson, PhD |

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In addition to three recruiting clinical studies that were announced in June, an additional trial is seeking participants with Friedreich’s ataxia (FA) to investigate how the disease affects the brain, spinal cord, and cognition.

Listed in a monthly newsletter released by the Friedreich’s Ataxia Research Alliance (FARA), the TRACK-FA study (NCT04349514) will use MRIs to track the natural history of brain and spinal cord changes in FA patients and correlate the findings with disease progression.

Researchers will pay particular attention to data collected from brain and spinal cord regions known to be affected in FA patients, in addition to cognitive data and results from tissue analysis. This collection of data may be used in future clinical trials to monitor treatment effectiveness.

The study is enrolling about 300 participants, ages 5 and older, at six sites in the U.S. and Australia. Additional locations in Brazil and Germany are expected to open soon.

Participants will visit the clinic on three occasions, about 12 months apart, which involves functional assessments, blood tests, and brain and spinal cord MRI.

Eligible patients include those with more than 55 repeats of the GAA sequence in both their FXN genes (one from each parent) or a GAA repeat expansion in one gene copy plus another mutation associated with loss of protein function in the second gene. Age of disease onset and disease duration of less than 25 also is required.

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Normally, the FXN gene contains a region where the GAA sequence is repeated five to 33 times. In almost all FA cases, there is a repeat expansion, in which the number of GAA repeats ranges from 66 to more than 1,000 in both FXN copies (one from each biological parent). Of note, “G” stands for guanine and “A” for adenine, two DNA building blocks.

The FXN gene provides instructions to make the frataxin protein, which is essential for the proper function of energy-producing mitochondria. A frataxin deficiency due to FXN defects leads to impaired energy production within cells, damaging nerves and muscles, causing ataxia, or uncontrolled body movements.

Three other clinical trials investigating disease progression and therapeutic interventions also are recruiting FA patients.

The Phase 2/3 MOVE-FA study (NCT04577352) is seeking 126 children and young adults with FA, ages 7 and older, to test the safety and efficacy of the investigational therapy vatiquinone (PTC743).

Launched late last year, MOVE-FA is enrolling at sites in the U.S., Canada, Brazil, Australia, and Germany. Locations in France and New Zealand are now open for eligibility screening, and additional sites in Italy and Spain are expected to open soon.

Researchers at the Children’s Hospital of Philadelphia and the University of Pennsylvania seek 14 more participants, ages 10 to 40, for a study (NCT04192136) evaluating the impact of the dietary supplement nicotinamide riboside (NR) combined with exercise.

The trial started in the fall of 2020, but is expected to run through December 2024. Enrollment information is available here.

Finally, participants are needed for the IDEA study (NCT04268147) to evaluate the impact of wearing sensors that measure body coordination to monitor disease progression and the severity of ataxia, or uncoordinated movements. This may allow remote data collected during future clinical trials.

Currently enrolling up to 144 individuals at sites in Baltimore, Boston, Chicago, Portland, or Los Angeles, the IDEA study is open to FA patients ages 12 to 30 who were diagnosed when they were 5 to 25 years old.