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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


The Friedreich’s Ataxia Health Index Study (FA-HI)

Title of Study: The Friedreich's Ataxia Health Index Study

Principal Investigator: Chad Heatwole, MD, MS-CI

Institutional Affiliation: University of Rochester, Department of Neurology

Study Description: This study aims to identify the symptoms that have the greatest impact on quality-of-life for individuals with FA. The results of the study will help guide future research involving FA patients through the development of a patient-reported measure of health.

The study involves completing a survey to include questions on demographics and symptoms of FA.

WHO CAN PARTICIPATE?

  • U.S. residents
  • Participants with a confirmed diagnosis of FA
  • Adults, ages 18 or older
  • Children, ages 8-17, with parental consent; if the child is age 13-17, only the child should complete the survey
  • Parents of children age 0-12; both parent and child can complete the survey for children ages 8-12.

Please see this Recruitment flyer to access the survey links.
 

 

CLIN-1601-102 - Larimar Therapeutics Phase I study of CTI-1601, a frataxin replacement therapy

Participants are needed for a Phase I research study to test an investigational medication in individuals with Friedreich's ataxia.

You May Be Eligible if You:

  • are 18 years of age or older,
  • have genetically confirmed Friedreich’s Ataxia with a copy of the genetic result (homozygous GAA repeat expansions), and
  • are able to go the distance of 25 feet with or without an assistive device or pushing oneself using a manual wheelchair.

Individuals should also be able to transfer from a bed to a chair with minimal assistance (e.g., being able to stand with support during a transfer) and perform basic daily care, such as feeding themselves and personal hygiene, with minimal assistance.

Please see this Recruitment Flyer for other Key Inclusion Criteria and contact information.

More information can also be found at Clinicaltrials.gov https://clinicaltrials.gov/ct2/show/NCT04176991.

Retrotope RT001-006 Phase 2/3 trial for protection against oxidative damage in the central nervous system

The Retrotope RT001-006 trial involves the study drug RT001, which may protect against lipid peroxidation, the process that is believed to cause disability in many neurodegenerative diseases, including Friedreich’s ataxia.

This study will involve five visits to a research clinic over a 12-month period. Each site visit can be done over multiple days. If you should decide to participate, you will be reimbursed for reasonable travel expenses.

To qualify, you must:

  • Have been diagnosed with Friedreich's ataxia
  • Be between 12 and 50 years old
  • Be able to walk at least 25-feet within 1 minute, with or without an assistive device
  • Not be actively enrolled in another clinical trial
  • Not have participated in the previous RT001 trial

 
For enrollment at Children's Hospital of Philadelphia only -
Please see this flyer to contact the study coordinator at CHOP.

For enrollment at our Long Beach, CA site -
Please see this flyer to contact the study coordinator.

For enrollment at our UCLA site -
Please see this flyer to contact the study coordinator.

For enrollment at other sites -
Please see this flyer to contact the study coordinator.

 

Methods of measuring neuropathy study - University of Rochester

Volunteers, ages 6‐40 needed for Research Study

Dr. David Herrmann (Dept. of Neurology) at the University of Rochester in upstate NY, is conducting a research study of Friedreich’s Ataxia. The study will evaluate how well different methods work, to measure how Friedreich’s Ataxia progresses

People with FA are needed for this study

Study procedures:
  • Brief neurological exam
  • Sensory testing
  • Photograph a small area of your hand & foot, using a microscope that can look into the skin
  • Skin biopsy – optional for some subjects
Altogether, the each study visit takes up to 8 hours

You will be asked to complete 4 study visits over about 2 years.

You will be paid $100 for each completed study visit. Some funds are available for travel expenses for people who have FA and people in their family without FA.

For more information, please contact Beth Wood: (585)276‐6248 - ElizabethP_Wood@urmc.rochester.edu



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