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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

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