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FARAFARA Cure FA

Clinical Network & Trials

Collaborative Clinical Research Network in Friedreich's Ataxia

The investigators and coordinators in the FA Collaborative Clinical Research Network are incredibly responsive and professional. With their partnership and FARA leadership, we were able to identify interested investigators and sites, develop a phase 2/3 protocol, obtain regulatory and ethics committee approvals, and launch a study in approximately six months. Once the study opened for screening, the first cohort of patients was identified, screened and enrolled in less than two months. –Colin J. Meyer, PhD, Vice President Product Development & Chief Medical Office, Reata Pharmaceuticals

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.

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, jen.farmer@curefa.org, (484) 879-6160

CCRN
Contacts

Network Principal Investigator
Dr. David R. Lynch
Children's Hospital of Philadelphia

Network Coordinator
Jennifer Farmer, MS, CGC
(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

CCRN
Sites

Children's Hospital of Philadelphia - Philadelphia, PA
Principal Investigator - Dr. David Lynch
Coordinator –
Lauren Hauser (267) 426-7538
Info for travel to CHOP
Airport: Philadelphia International (PHL)

University of California Los Angeles - Los Angeles, CA
Principal Investigator - Dr. Susan Perlman
UCLA Ataxia Center
For clinic appointments: (310) 794-1195
For research questions: (310) 206-8153
Airport: Los Angeles International (LAX)

Emory University - Atlanta, GA
Principal Investigator, Dr. George Wilmot
Coordinator - Rebecca S. McMurray, RN, MSN (404) 712-7013
Airport: Hartsfield Jackson Atlanta International (ATL)

Hotels near the clinic site:
MARRIOTT COURTYARD
at Executive Park
1236 Executive Park Dr NE
Atlanta, GA 30329
(404) 728-0708
DOUBLETREE by HILTON
2061 N Druid Hills Rd. NE
Atlanta, GA.  30329
(404) 321-4174
HAMPTON INN
ATLANTA NORTH
1975 North Druid Hills
Atlanta, GA.  30329
(800) 862-1796
(404) 320-6600


University of Iowa - Iowa City, IA
Principal Investigator - Dr. Kathy Matthews
Coordinator - Carrie Stephan, RN (319) 356-2673
Travel Information

Data Coordination:
University of Rochester - Rochester, NY

Principal Investigator – Dr. Cindy Casaceli
Information Analyst – Lisa Rumfola
PhD Project Manager – Alice Rudolph (585) 275-7311

University of Florida - Gainesville, FL
Principal Investigator - Dr. Sub Subramony
Coordinator - Blake Meyer (352) 294-8754
Airports:
Gainesville Regional (GNV)
Orlando International (MCO)
Tampa International (TPA)

Hotels near the clinic site:
Hilton University of Florida Conference Center
1714 SW 34th St
Gainesville, FL 32607
Home2Suites by Hilton
2115 SW 13th St
Gainesville, FL 32608
Holiday Inn University Center
1250 W University Ave
Gainesville, FL 32601
DoubleTree by Hilton
3726 Southwest 40 Boulevard
Gainesville, FL 32608


University of South Florida - Tampa, FL
Principal Investigator - Dr. Theresa Zesiewicz
Coordinator – Kevin Allison(813) 974-5909
Airport: Tampa International (TPA)

The Bruce Lefroy Centre for Genetic Health Research
Murdoch Children's Research Institute - Melbourne, Australia

Principal Investigator - Dr. Martin Delatycki
Coordinators - Geneieve Tai & Louise Corben (613) 8341-6374

Ohio State University - Columbus, OH
Principal Investigator - Dr. J. Chad Hoyle
Coordinator – Tabi Alexander
(614) 293-6953
Info for travel to Ohio State
Airport: John Glenn Columbus International (CMH)

Sick Kids - Toronto ON, Canada
Principal Investigator – Dr. Grace Yoon
(416) 813-6389

Clinical Data Science GmbH - Switzerland
Lead Statistician/Data Scientist - Christian Rummey christian@clindata.science

Case Report Forms

Friedreich Ataxia Rating Scale (FARS) - The FARS is comprised of a neurological exam, functional staging and activities of daily living. CRFs for all three components are available below (in PDF format):
  
Neuro Exam 
 
Functional Staging 
 
Activities of Daily Living

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Clinical Trials



Clinical Trials - Active - Enrolling

Clinical Trials - Active - Enrollment Closed

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