Clinical Trials

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 attached flyer to contact the study coordinator at CHOP.

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

For enrollment at other sites -
Please see this attached 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.

The Collaborative Clinical Research Network in Friedreich's Ataxia (CCRN in FA) is an international network of clinical research centers, where physicians, researchers and patients work together to advance treatments and best practices for management and treatment of Friedreich's ataxia. The Children’s Hospital of Philadelphia (CHOP) is one of the sites participating in this network. The CCRN in FA is funded by the Friedreich’s Ataxia Research Alliance (FARA). The research centers involved in this network, including CHOP, run a study called Clinical Outcome Measures in Friedreich’s Ataxia (FACOMS). This is a natural history study with the following objectives:

• Collect data every year to understand progression of FA and be able to quantify change in progression over time
• Develop and validate outcome measures (timed walk tests, vision tests) that can be used in future clinical trials
• Expand the network of clinical research centers working on Friedreich ataxia
• Make design and implementation of clinical trials more efficient and effective
• Collect biological samples such as cheek swabs and blood to identify and study biomarkers involved with the disease process and quantify the change in these markers over time

You may be eligible to take part if:

• You have a confirmed diagnosis of Friedreich’s ataxia
• A parent/guardian gives permission for you to participate if you are under 18 years of age

The sites in this network provide opportunities for individuals to participate in the FACOMS study annually.

If you would like to participate in this study at the Children’s Hospital of Philadelphia, please call the CHOP Friedreich Ataxia Program number at 267-426-7538. The Principal Investigator at this site is Dr. David Lynch.

For a list of additional FACOMS Study Sites, please refer to the FARA website: http://www.curefa.org/network

Thank you for your ongoing support of clinical research in Friedreich ataxia.