Emily’s Entourage Officially Launches Global ‘CF Clinical Trial Connect’ to Drive Breakthrough Therapies for the Underserved CF Community

New, upgraded platform connects people with cystic fibrosis worldwide with new clinical trial opportunities, accelerating development of therapies for those who remain unserved by current CFTR modulators

Lower Merion, PA – September 12, 2024 — Emily’s Entourage (EE), an innovative 501(c)3 accelerating research for new treatments and a cure for the final 10% of people with cystic fibrosis (CF), is thrilled to announce the launch of CF Clinical Trial Connect (CTC). Initially launched as the EE Patient Registry, the CTC is a newly upgraded global database designed exclusively for people with CF who do not benefit from current CFTR modulator therapies due to ineligible genetic mutations, side effects, or suboptimal responses to modulators. The CTC offers a unique opportunity to accelerate the development of lifesaving treatments for this ultra-rare, underserved group.

Expanding on the foundation of the previous EE Registry, the CF Clinical Trial Connect database has been upgraded to deliver even greater impact. Supported by a global network of partner organizations, the CTC provides essential information and resources to help people who do not benefit from CFTR modulators identify and pursue clinical trial opportunities. By streamlining recruitment and enrollment, the CTC accelerates the discovery of treatments and helps address the unique challenges of rare disease drug development.

By signing up for CF Clinical Trial Connect, people with CF will receive early access to relevant information about new clinical trial opportunities based on their profile, including their CF mutations, microbiology, age, and location.

The process is simple:

1. Individuals register by providing basic demographic and genetic information.
2. They receive personalized notifications about relevant clinical trials via email and can explore active trial opportunities supported by EE on the CTC portal at any time.
3. Registrants can learn more about relevant clinical trials and consider participating in a trial, helping to accelerate cutting-edge research and drug development.

“At Emily’s Entourage, we believe that people with CF have the greatest vested interest in their own health and should be equipped with the resources to make informed choices about clinical research and care,” said Emily Kramer-Golinkoff, MBE, co-founder of Emily’s Entourage. “We also believe that cultivating a well-coordinated, activated community of people within the final 10% through the CTC will be critical to getting lifesaving treatments for this ultra rare population to the finish line fast — and that is our singular goal.”

Those who register for CTC gain early access to new clinical trial information, enabling them to learn about trials directly, without relying solely on care centers or clinicians. This approach accelerates clinical trial recruitment and drives faster progress toward the development of treatments for those that do not benefit from current CFTR modulators.

“The launch of CF Clinical Trial Connect is a critical moment in the field of CF research. By connecting people with CF that do not benefit from current CFTR modulators to the latest clinical trials, we are accelerating the development of new therapies for a group with critical, unmet needs,” said Chandra Ghose, PhD, Chief Scientific Officer at Emily’s Entourage.

CF Clinical Trial Connect is open to people with CF from around the world who are unable to benefit from CFTR modulators, including those with ineligible genetic mutations, side effects, or suboptimal responses to modulators. Parents or guardians can register on behalf of individuals under the age of 18.

For more information and to register for CF Clinical Trial Connect, visit www.emilysentourage.org/ctc.

 

About Emily’s Entourage

Emily’s Entourage is an innovative 501(c)3 that accelerates research for new treatments and a cure for individuals in the final 10% of the cystic fibrosis (CF) population that does not benefit from currently available mutation-targeted therapies. Since 2011, Emily’s Entourage has awarded millions of dollars in research grants, launched a now-acquired CF gene therapy company, developed a patient database and clinical trial matchmaking program to accelerate clinical trial recruitment, and led worldwide efforts to drive high-impact research and drug development. The organization has been featured in national media, including the New York Times, STAT, CNN, People, and more. Learn more at emilysentourage.org.

 

Media Contact

Hannah Kleckner

Director of Marketing and Communications

hannah@emilysentourage.org