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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 February 2021. Information on MOVE-FA can be found 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: 319-335-7498

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.

Eligibility criteria:

  • 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

Please see the flyers below for additional details about the study and to contact a study coordinator:

Johns Hopkins University flyer

Oregon Health & Sciences University (OHSU) flyer

University of Chicago flyer - study contact for additional enrollment sites at Mass General and UCLA

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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Characterization of the Cardiac Phenotype of Friedreich’s Ataxia

Heart and Eye Study at Weill Cornell Medicine

This study is currently recruiting 20 participants ages 12-17 diagnosed with FA along with their unaffected siblings in the same age range. This study is designed to characterize the cardiac manifestations of FA using exercise, MRI, ECHO and blood tests. Additionally, this study will include corneal confocal microscopy (CCM), a non-invasive eye evaluation. Study participants and their siblings will have 2 visits in 1 year to Weill Cornell Medicine in New York City; rapid COVID-19 testing will be performed at the hospital prior to study visit. Financial support is available for travel and hotel. For eligibility screening and additional study information, please contact the study coordinator through email at cora@med.cornell.edu.

More information is available HERE

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