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Characterization of the Cardiac Phenotype of Friedreich’s Ataxia

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

Clinical Outcome Measures in Friedreich’s Ataxia: a natural history study

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.

Idebenone 2008 Phase 3 Follow-Up Study through CHOP and UCLA

The Children’s Hospital of Philadelphia (CHOP) is recruiting patients with Friedreich ataxia (FRDA) who previously participated in the 2008 phase 3 clinical trial of Idebenone for a new follow-up study. The purpose of the new study is to better understand how this specific group of patients have changed since the trial. There are no interventions or research visits in this study. The research team will mainly analyze previous medical records. If you (or your child) participated in the 2008 clinical trial of Idebenone, you are invited to contact Dr. Lynch or his study coordinator to learn more about this research project. This study is funded by the Friedreich Ataxia Research Alliance (FARA) and is taking place only at CHOP and the University of California Los Angeles (UCLA).

Please Click Here to learn more.

Idebenone 2005-06 Phase 2 Follow-Up Study through CHOP

The Children’s Hospital of Philadelphia (CHOP) is recruiting patients with Friedreich ataxia (FRDA) who previously participated in the 2005-06 phase 2 clinical trial of Idebenone for a new follow-up study. The purpose of the new study is to better understand how the cardiac phenotype of this specific group of patients has changed since the trial. There are no interventions or research visits in this study. The research team will mainly analyze previous medical records. If you (or your child) participated in the 2005-06 NIH clinical trial of Idebenone, you are invited to contact Dr. Lin and her study team to learn more about this research project. This study is funded by the Friedreich Ataxia Research Alliance (FARA) and is taking place only at CHOP.

Please Click Here to learn more.

Call for Patients with Friedreich ataxia with upcoming surgical procedure or existing tissue

The Children's Hospital of Philadelphia (CHOP) is recruiting patients with Friedreich ataxia (FA) who are planning to have a surgical procedure performed. These surgical procedures can include things like scoliosis surgery and tendon transfer surgery. We are also looking for patients with FA who have had biopsies performed in the past at other hospitals.

Please Click Here to learn more.

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