New Platform Enables Collaborative Data Sharing to Develop Therapies for Friedreich’s Ataxia

A new data-sharing platform recently launched by the Critical Path Institute’s (C-Path) Data Collaboration Center (DCC) and the Friedreich’s Ataxia Research Alliance (FARA) is designed to ultimately lead to the development of effective treatments for Friedriech’s ataxia (FA).

Called the Friedreich’s Ataxia Integrated Clinical Database (FAICD), the platform will enable collaborative research and data sharing to promote the understanding of natural history, prospective biomarkers and clinical objectives. It also will foster research into novel clinical trial design in FA, the organizations said in a press release.

The hope is that by making data accessible to scientists, tools that will help design and interpret efficient clinical trials will be developed.

“FA is a rare, progressive and fatal disease that affects multiple organ systems, and those living with the disease are in urgent need of effective treatments,” said Jennifer Farmer, FARA’s executive director.

“FAICD addresses this need by providing a platform to share data and making it available to qualified researchers to expedite the drug development process. We believe this resource will inform and give future clinical trials of potential therapies the best chance of success.”

Launched last month, the platform includes data from partnering companies that have conducted clinical trials in FA. It also contains natural history and information from the Friedreich’s Ataxia Clinical Outcomes Measure Study (FACOMS) gathered by the FARA-funded Collaborative Clinical Research Network (CCRN), an international network of clinical research centers that work collaboratively to advance treatments and clinical care in FA.

Join the Friedreich’s Ataxia News forums: an online community for people with FA and their caregivers.

FACOMS (NCT03090789) is a multicenter natural history study aimed at providing a framework for facilitating therapeutic interventions, including the development of clinical measures crucial to outcome assessment of FA patients. The trial is still recruiting participants.

Before being included in the FAICD, contributed data are de-identified to preserve participants’ privacy, mapped to standards defined by the Clinical Data Interchange Standards Consortium (CDISC), and curated by the DCC. The nonprofit CDISC works through collaborative teams to develop global standards and innovations to streamline medical research data.

“C-Path has a long history of expertise in data standards development, curation and oversight of multiple data-sharing initiatives,” said Richard Liwski, DCC director and C-Path’s chief technology officer. “We are proud to be part of the effort to coordinate collaborative contributions from data owners, and integrate that data into a single database for this rare, degenerative and life-shortening neuromuscular disorder.”

Researchers who wish to contribute data to FAICD or use the database can get more information here.

C-Path is an independent, nonprofit organization that seeks to support the development of new approaches that advance medical innovation and regulatory science.

FA is a genetic disease caused by an expanded GAA trinucleotide repeat in the FXN gene, which affects the nervous system and muscles. About 1 in 50,000 people in the United States have the degenerative neuromuscular disorder, according to FARA.