LEOPARD-FA: Longitudinal Endpoint Optimization to Provide an Assessment of Relevant Drugs in Friedreich’s Ataxia

https://clinicaltrials.gov/ct2/show/NCT05168774

The Children’s Hospital of Philadelphia (CHOP) is recruiting patients with Friedreich ataxia (FRDA) in the United States for a Phase 1/2 clinical trial studying the safety and effects of Elamipretide in the treatment of advanced stage FRDA symptoms, specifically vision loss and cardiac disease in both adults and older children.

This study will include up to 18 patients to be enrolled at the Children’s Hospital of Philadelphia.

Eligibility criteria include:

Inclusion:

• Be 16 years of age or older
• Have genetic confirmation of your FRDA (point mutations allowed)
• Have been diagnosed with FRDA at age 18 years old or younger
• Have vision loss and/or cardiac disease
• Be willing to administer a daily injection (shot)
• Not be actively enrolled in any clinical trial

Exclusion:

• Have any clinically relevant medical or surgical condition that could interfere with the administration of study drug, or compromise your safety or well-being
• Be pregnant, planning a pregnancy, or breastfeeding
• Have a history of substance abuse
• Have any other active cause of optic neuropathy or cardiac disease outside of FRDA
• Have a history of an uncontrolled arrhythmia

Additional requirements apply and will be discussed with you by the study doctor.

If you agree to take part, your participation will require a phone pre-screening, as well as up to 6 in-person visits over the course of 1 year. As a subject in the research study you will:

• Complete a phone pre-screening questionnaire to review eligibility criteria
• Receive a study drug (Elamipretide) as a daily shot (injection)
• Perform various vision testing
• Undergo heart function evaluations
• Have research blood tests at each study visit
• Complete questionnaires and speech testing
• Keep a subject dosing diary

The study drug and all study-related assessments will be provided at no cost. You may be reimbursed for study-related expenses, such as parking and meals up to $500 per visit.

Contact one of the CHOP study coordinators to learn more about what study procedures are involved and if you may be eligible to participate.

Courtney Cheek Park: Phone: (267) 426-9667; Email: This email address is being protected from spambots. You need JavaScript enabled to view it.
McKenzie Wells: Phone: (267) 426-9608; Email: This email address is being protected from spambots. You need JavaScript enabled to view it.
Medina Keita: Phone: (267) 426-7584; Email: This email address is being protected from spambots. You need JavaScript enabled to view it.
Kellie McIntyre: Phone: (215) 590-2975; Email: This email address is being protected from spambots. You need JavaScript enabled to view it.

View the Study flyer

 

 

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: This email address is being protected from spambots. You need JavaScript enabled to view it.
Phone: 267-426-9567

University of South Florida
Investigator: Dr. Teresa Zesiewicz
Study coordinator: Mary Freeman
Email: This email address is being protected from spambots. You need JavaScript enabled to view it.
Phone: 813-974-4685

UCLA
Investigator: Dr. Susan Perlman
Study coordinator: Aaron Fisher
Email: This email address is being protected from spambots. You need JavaScript enabled to view it.
Phone: 310-206-8153

University of Iowa
Investigator: Dr. Kathy Mathews
Study Coordinator: Evgenia Folts
Email: This email address is being protected from spambots. You need JavaScript enabled to view it.
Phone: 319-335-7617

University of Florida, Gainesville
Investigator: Dr. Sub Subramony
Study Coordinator: Amanda Cowsert
Email: This email address is being protected from spambots. You need JavaScript enabled to view it.
Phone: 352-733-2435

Murdoch Children's Research Institute, Victoria, Australia
Investigator: Dr. Martin Delatycki
Study coordinator: Geneieve Tai
Email: This email address is being protected from spambots. You need JavaScript enabled to view it.
Phone: +61 3 8341 6374

Centre Hospitalier de l'Universite de Montreal, Canada
Investigator: Dr. Antoine Duquette
Study coordinator: Martine Comeau
Email: This email address is being protected from spambots. You need JavaScript enabled to view it.
Phone: 514-890-8000 post 30154

CHU Sainte-Justine, Canada
Enrolling children <14 years old
Study Coordinator: Fabioloa Breault
Email: This email address is being protected from spambots. You need JavaScript enabled to view it.
Phone: 514-345-4931 x5407

University of Campinas (UNICAMP) , Brazil
Investigator: Dr. Marcondes Cavalcante Franca Junior
Study coordinator: Maria Fernanda Ribeiro Bittar
Email: This email address is being protected from spambots. You need JavaScript enabled to view it.
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: This email address is being protected from spambots. You need JavaScript enabled to view it.
Phone: +33 (0) 1 57 27 46 91

Auckland City Hospital, New Zealand
Investigator: Dr. Richard Roxburgh
Study coordinator: Kay Yeoman
Email: This email address is being protected from spambots. You need JavaScript enabled to view it.
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: This email address is being protected from spambots. You need JavaScript enabled to view it.
Phone: 673 135168

Ospedale Pediatrico Bambino Gesu’ IRCCS, Italy
Investigator: Dr. Enrico Bertini
Study Coordinator: Dr. Antonella Longo
Email: This email address is being protected from spambots. You need JavaScript enabled to view it.
Phone: 39 06 68592105

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.
 

 

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.