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PTC-743: MOVE-FA Study

PTC-743: MOVE-FA Study

MOVE-FA is a Phase 2/3 interventional study with 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.

This study became fully enrolled in October 2021.

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

This study is being conducted 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

University of Florida, Gainesville
Investigator: Dr. Sub Subramony
Study Coordinator: Amanda Cowsert
Email: Amanda.cowsert@neurology.ufl.edu
Phone: 352-733-2435

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

CHU Sainte-Justine, Canada
Enrolling children <14 years old
Study Coordinator: Fabioloa Breault
Email: fabioloa.breault.hsj@ssss.gouv.qc.ca
Phone: 514-345-4931 x5407

University of Campinas (UNICAMP) , Brazil
Investigator: Dr. Marcondes Cavalcante Franca Junior
Study coordinator: Maria Fernanda Ribeiro Bittar
Email: febittar@unicamp.br
Phone: +55 (19) 3521-9217

France, Institute du Cerveau et de la Molelle epiniere (ICM), Hopital Universitaire Pitie-Salpetriere
Investigator: Dr. Alexandra Durr
Study coordinator: Rania Hilab
Email: rania.hilab@icm-institute.org
Phone: +33 (0) 1 57 27 46 91

Auckland City Hospital, New Zealand
Investigator: Dr. Richard Roxburgh
Study coordinator: Kay Yeoman
Email: kay.yeoman@auckland.ac.nz
Phone: +64 274 790 725

Hospital Sant Joan de Déu Barcelona Unidad de Enfermedades Neuromusculares, Spain
Investigator: Dr. Alejandra Darling
Study Coordinator: Isabel Maria Miquel Aymar
Email: isabelmaria.miquel@sjd.es
Phone: 673 135168

Ospedale Pediatrico Bambino Gesu’ IRCCS, Italy
Investigator: Dr. Enrico Bertini
Study Coordinator: Dr. Antonella Longo
Email: antonella.longo@opbg.net
Phone: 39 06 68592105


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