Clinical Trials

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

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.

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.

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 attached flyer for additional details about the study and to contact the study coordinator.