Clinical Network & Trials
Collaborative Clinical Research Network
in Friedreich's Ataxia
The Collaborative Clinical Research Network in Friedreich's Ataxia (CCRN in FA) is an international network of clinical research centers that work together to advance treatments and clinical care for individuals with Friedreich's ataxia. The network collaborates with pharmaceutical companies, government agencies and other research centers and the patient community to facilitate clinical research and trials needed to identify new therapies.
The Core Objectives of the Network
- Identifying and validating clinical outcome measures and biomarkers in FA that are necessary for clinical trials
- Facilitating the implementation and delivery of clinical trials
- Sharing data and resources to advance treatments for FA
- Defining best clinical practices for FA and providing the highest level of clinical care for patients
At each clinical research center there is a team of researchers, physicians and health care providers dedicated to FA.
The CCRN in FA is funded by the Friedreich's Ataxia Research Alliance.
|Network Principal Investigator|
|Dr. David R. Lynch
Children's Hospital of Philadelphia
Jennifer Farmer, MS, CGC
firstname.lastname@example.org (484) 879-6160
|Data & Clinical Trial Coordination|
Dr. Cindy Casaceli
University of Rochester
|Biorepository (DNA, RNA and Plasma)|
Giovanni Coppola, UCLA
and David Lynch, CHOP
Principal Investigator - Dr. David Lynch
Coordinators – Karlla Brigatti & Lauren Seyer, Brigatti@email.chop.edu & SeyerL@email.chop.edu
Telephone, (267) 426-7538
Principal Investigator - Dr. Susan Perlman
Coordinator - Brian Clemente email@example.com
Telephone: (310) 206-8153
Principal Investigator, Dr. George Wilmot
Coordinator - Rebecca S. McMurray, RN, MSN firstname.lastname@example.org
Telephone: (404) 728-6427
Principal Investigator - Dr. Kathy Matthews
Coordinator - Carrie Stephan, RN, email@example.com
Telephone: (319) 356-2673
Principal Investigator – Dr. Cindy Casaceli
Information Analyst – Lisa Rumfola
Principal Investigators - Drs. Tetsuo Ashizawa and Sub Subramony
Coordinator - Phuong Deleyrolle, RN, firstname.lastname@example.org
Telephone: (352) 273-7732
University of South Florida - Tampa, FL
Principal Investigator - Dr. Theresa Zesiewicz
Coordinators – Kevin Allison & Jessica Shaw
email@example.com & firstname.lastname@example.org
Telephone: (813) 974-5909
The Bruce Lefroy Centre for Genetic Health Research
Murdoch Children's Research Institute - Melbourne, Australia
Principal Investigator - Dr. Martin Delatycki
Coordinator - Geneieve Tai & Louise Corben, email@example.com & firstname.lastname@example.org
Telephone: (613) 8341-6374
The Ohio State University - Columbus, OH
Principal Investigator - Dr. J. Chad Hoyle
Coordinator – Colleen Pineda, Colleen.Pineda@osumc.edu
Telephone: (614) 685-5661
Sick Kids - Toronto ON, Canada
Principal Investigator – Dr. Grace Yoon
Telephone: (416) 813-6389
Each of the CCRN in FA sites is a place where FA patients can go to participate in research studies and to receive clinical care. At every site researchers are conducting natural history and clinical outcome measure studies. These are studies that document the clinical symptoms and progression of FA and evaluate specific tests or measures, such as a neurological scale, timed walk, speech, vision, and quality of life measures, that can be used in future clinical trials. All of the research data collected at each site is gathered into a combined database. Some sites have additional studies, such as hearing evaluations, biomarker studies, and clinical trials.
The investigators and coordinators at each of these sites are very familiar with medical issues related to FA and can provide clinical services as well.
We recommend that you contact the site located close to you to learn about the studies and clinical services being offered.
The CCRN in FA can assist you in offering your patients access to clinical research studies in FA. The investigators and coordinators at each of the sites can work with you to provide guidance on updates in clinical care and management for your patients.
The CCRN in FA wants to work with researchers (academic, pharmaceutical, biotechnology and government) to facilitate investigations of novel interventions and bring improved clinical outcomes to FA patients. Specifically, the CCRN in FA has an established infrastructure that can support the following:
- Facilitation in recruitment and planning of clinical trials - The CCRN in FA has a dedicated Data and Clinical Trial Core that is experienced in the planning and conduct of clinical trials for neurodegenerative diseases. Services utilized can range from planning consultation to full implementation and coordination of a study.
- Natural history and outcome measures in FA - The CCRN in FA was formed from a group of investigators who were previously collaborating to develop clinical outcome measures in FA. Longitudinal data is available and continues to be collected and shared publically with the FA research community. A list of such publications is accessible in the right hand column.
- Partnership for new clinical studies to support the advancement of novel interventions, such as biomarker development, new outcome measure development, and genetic studies. The CCRN in FA can function as the clinical site for a new study providing access to patients and their relevant clinical data, handling the procurement of biological tissues and fluids, and assisting with preparation and filing of the necessary regulatory approvals.
Researchers who would like to learn more about the CCRN in FA or discuss a study should contact the network PI: David Lynch, MD, PhD, Lynchd@mail.med.upenn.edu, 215-590-2242 or Coordinator: Jennifer Farmer, MS, email@example.com, (484) 879-6160