RSRT Launches Rett Syndrome Clinical Trial Consortium with $1.8 Million Award

TRUMBULL, CT — The Rett Syndrome Research Trust (RSRT) announced today the launch of the Rett Syndrome Clinical Trial Consortium. The primary objective of this Consortium is to provide expert Rett Syndrome physicians with the personnel and resources necessary to execute high-quality clinical trials in a timely and efficient manner.

During the last decade, the Rett Syndrome basic science community, fueled by millions of dollars in research investments, has made phenomenal progress in understanding the disease. Coupled with technological advances in a number of fields, including gene therapy, we are now on the brink of advancing potentially life-changing therapeutics into human clinical trials.

These human clinical trials will generally be undertaken at Rett Syndrome Clinics, many of which are facing a number of key challenges due to lack of funding.

Firstly, there are a limited number of Rett physicians who have sufficient expertise and resources to participate in clinical research that meets rigorous FDA standards. Secondly, most Rett Clinics are resource constrained and constantly challenged to support and retain the highly trained personnel necessary to conduct rigorous clinical research. Lastly, lack of funding and trained personnel impacts the Rett clinician’s ability to analyze and publish data in a timely fashion, potentially delaying the sharing of important information.

For all these reasons, the time is right to invest in a Clinical Trial Consortium to support rigorous testing of the therapeutics we have worked so hard to develop.

The Clinical Trial Consortium will launch with an initial investment of $1.8 million to support four Rett Clinics. As our funding allows, we hope to grow the number of Clinics in the Consortium. This initial grant of $1.8 million will support full-time research support staff (research coordinators, nurses and/or physicians in training) for three years at the following Clinics:

1. Center for Rare Neurological Diseases (CRND, Atlanta); Dr. Daniel Tarquinio

2. Montefiore Medical Center (MMC, Bronx NY); Dr. Sasha Djukic

3. Children’s Hospital of Philadelphia (CHOP); Dr. Eric Marsh

4. Boston Children’s Hospital (BCH); Drs. David Lieberman & Mustafa Sahin

While this funding will allow the v to focus their efforts on clinical trials that provide the most benefit to the Rett community, a complementary outcome will be to leverage the resources and infrastructure developed in our Outcome Measures and Biomarker Development Consortium (OMBD) to improve clinical care for individuals with Rett Syndrome. Specifically, the outcome measures developed in the OMBD will be used to track and evaluate whether changes in clinical care (e.g., drugs or biologicals, diet, therapy) result in improved health for individual patients.

“As the principal investigator or sponsor of over 75 clinical trials during my academic and biotechnology career, I intimately understand the challenge of performing high quality clinical trials in a busy medical practice. I look forward to working closely with these investigators, openly sharing our insights and results with the many other expert investigators that we were regrettably unable to fund in this initial effort, and hopefully, ultimately recruiting these investigators into the Clinical Trial Consortium,” said Randall Carpenter, MD, Chief Scientific Officer of RSRT.

“It’s a very exciting time for Rett research as we’ve reached the point where clinical trials of potentially curative approaches are imminent. I could never thank our generous donors and the families that fundraise for RSRT enough. We’re at this stage because of you,” said Monica Coenraads, Executive Director of RSRT and mother to a young woman with Rett Syndrome.