FARA - Clinical Trials

TRACK-FA study: bioimaging of brain and spinal cord

TRACK-FA is a natural history study to investigate brain and spinal cord changes in individuals with Friedreich ataxia. If imaging differences in the central nervous system are correlated with progression of FA, these differences could be used in future clinical trials to monitor treatment outcome.

There are 3 study visits, each approximately 12 months apart. At each study visit, participants will be asked to complete -

Neurologic and functional testing
Blood draw
Brain and spinal cord MRI scan

Inclusion criteria (partial):

Age >5 years with genetically confirmed diagnosis of FA.
GAA repeat expansion >55 in length on both alleles of the FXN gene or GAA repeat expansion on one allele and another mutation type on the other allele (e.g., point mutation, gene deletion).
Age of disease onset less than 25 years.
Disease duration less than 25 years.
Able to read and converse in English.

Exclusion criteria (partial):

MRI contraindications such as pacemaker, other metallic surgical implants, metallic braces.
Pregnancy
Other ongoing medical conditions, including psychiatric and neurological diagnoses (speak with the site investigator).

Additional information can be found on the clinicaltrials.gov website (NCT04349514)

Compensation is provided for time and travel expenses.

Sites open for enrollment

University of Florida, Gainesville
Principal Investigators: Sub H Subramony, MD, Thomas Mareci, PhD, Manuela Corti, PhD
Study coordinator: Samantha Norman
Email: Samantha.Norman@peds.ufl.edu
Phone: (352) 273-8218

University of Minnesota, Minneapolis
Principal Investigators: Pierre-Gilles Henry, PhD, Christophe Lenglet, PhD
Study coordinator: Diane Hutter
Email: hutte019@umn.edu
Phone: (612) 625-2350

Children’s Hospital of Philadelphia
Principal investigators: William Gaetz, PhD, Timothy PL Robets, PhD, David Lynch, MD, PhD
Study coordinator: Leah Gaetz
Email: GaetzL@chop.edu
Phone: (267) 275-6352

Monash University, Clayton, Victoria, Australia
Principal Investigators: Nellie Georgiou-Karistianis, PhD, Ian Harding, PhD, Louise Corben, PhD, Martin Delatycki, PhD
Study coordinator: Karen Harris
Email: Karen.Harris1@monash.edu
Phone: +61 3 9905 3487

Exercise/NAD+ study at CHOP & Penn

Exercise/NAD+ study

Research clinicians at the Children’s Hospital of Philadelphia and University of Pennsylvania are enrolling participants in a study to evaluate the effects of a dietary supplement combined with exercise. The primary outcome measure is changes in aerobic capacity, which is the body’s ability to take in, transport and use oxygen during exercise.

This study will be enrolling over 4 years, starting in 2020, with a target enrollment of 72 participants. Compensation is provided for study time.

Eligibility criteria include:

Genetically-confirmed diagnosis of FA
Age 10-40 years old
Able to complete exercise testing

Participation is this study involves completion of two (2) in-person clinic visits, each lasting 2 days, over a 12-week period. During the study period, you will be asked to complete blood testing, exercise testing, administration of a dietary supplement, MRI scans, oral glucose tolerance testing (with a non-FDA approved stable isotope product) and optional muscle biopsies.

If you are interested in learning more about this study, please contact Anna DeDio through email dedioa@email.chop.edu or by phone (267)-425-1998.

PTC-743: MOVE-FA Study

PTC-743: MOVE-FA Study

MOVE-FA is a Phase 2/3 interventional study with planned enrollment sites in Australia, Brazil, Canada, the U.S. and throughout the EU. Eligibility criteria include:

Genetically confirmed FA with two GAA repeat expansions (currently, point mutations and deletions are excluded from the study)
Children ages 7-17 and Adults age 18 and older
Ability to walk 10 feet in one minute with or without assistance
Be able to swallow capsules
Not have participated in another interventional study in the 2 months prior to enrollment in MOVE-FA

Participation in MOVE-FA will last for 2 years and will involve about 10 in-person study visits, as well as study phone calls in-between visits. Participants will need to take the study drug or placebo three times a day for 72 weeks. After those 72 weeks, all study participants will receive the study drug for an additional 24 weeks during the open-label extension phase.

This interventional study will monitor the safety and efficacy of the drug Vatiquinone, which is expected to decrease the activity of an enzyme that allows free iron to trigger a type of programmed cell death that occurs more frequently in people affected with FA.

Enrollment sites worldwide will become available in December 2020 through June 2021.

An informational webinar on PTC-743 can be viewed here:

ENROLLMENT is OPEN at the following institutions:

Children's Hospital of Philadelphia
Investigator: Dr. Dave Lynch
Study coordinator: Courtney Park
Email: ParkCC@email.chop.edu
Phone: 267-426-9567

University of South Florida
Investigator: Dr. Teresa Zesiewicz
Study coordinator: Mary Freeman
Email: mfreema4@usf.edu
Phone: 813-974-4685

UCLA
Investigator: Dr. Susan Perlman
Study coordinator: Aaron Fisher
Email: adfisher@mednet.ucla.edu
Phone: 310-206-8153

University of Iowa
Investigator: Dr. Kathy Mathews
Study Coordinator: Evgenia Folts
Email: Evgenia-Folts@uiowa.edu
Phone: 319-335-7617

Murdoch Children's Research Institute, Victoria, Australia
Investigator: Dr. Martin Delatycki
Study coordinator: Geneieve Tai
Email: geneieve.tai@mcri.edu.au
Phone: +61 3 8341 6374

Centre Hospitalier de l'Universite de Montreal, Canada
Investigator: Dr. Antoine Duquette
Study coordinator: Martine Comeau
Email: martine.comeau.chum@ssss.gouv.qc.ca
Phone: 514-890-8000 post 30154

Instrumented Data Exchange for Ataxia (IDEA study)

Participants are needed for a research study to test body-worn sensors to measure movement during simple tests of coordination. The goal of this study is to evaluate progression and severity of ataxia.The outcomes of this study are critical for allowing remote data collection during future clinical trials.

Eligibility criteria include:

Be 12-30 years of age with FA diagnosed between ages 5-25
Be able to walk 10 feet independently
Be able to sit and stand unassisted for at least 30 seconds

Participants can be involved in an investigational drug trial and still be eligible for the IDEA study.

The study involves visits every 6 months over 2 years at a clinic site. Involvement in the study provides an Apple watch.

Compensation is available for travel to study sites.

Participants are needed at all study sites in Baltimore (Johns Hopkins), Boston (MGH), Chicago (University of Chicago), Los Angeles (UCLA) or Portland (Oregon Health & Science University).

For additional eligibility criteria, please contact the site study coordinator:

University of Chicago
Study coordinator: Hannah Casey
Email: hannahcasey@uchicago.edu
Phone: (773) 702-4610

Oregon Health & Sciences University
Study coordinator: Austin Prewitt
Email: prewitta@ohsu.edu

Johns Hopkins Medical Institute
Study coordinator: Michelle Joyce
Email: mjoyce14@jhmi.edu

Massachusetts General Hospital
Study coordinator: Brigitte Jacoby
Email: BJACOBY@mgh.harvard.edu

UCLA
Study coordinator: Aaron Fisher
Email: ADFisher@mednet.ucla.edu

Burke Institute - Seated Exercise Study

Home Based Tele-Exercise for People with Chronic Neurological Impairments
The seated exercise study will include:

Adaptive Boxing
Adaptive Power Posture
Adaptive High-Intensity Interval Training (HIIT)

Who can participate?

18-75 years of age
Diagnosis of a chronic neurological impairment (at least 6 months post injury)
Ability to commit to 12-week program (3 times a week)
Medically stable, no contraindications to exercise
Access to high speed internet on a device with webcam
Currently exercising two (2) days or less per week
Can follow instructions in English

More information is available below.

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Clinical Trials