Your family can be the key that researchers and industry need to create a world without Rett.
And the International Rett Syndrome Foundation (IRSF) is making it easier than ever for families to help unlock treatments and cures through the new Rett Syndrome Registry™.
“Everything IRSF does is designed to accelerate the creation of treatments for all of our loved ones with Rett syndrome,” says IRSF CSO Dr. Dominique Pichard, MD, “Because Rett is a rare disorder, every family can be an essential partner in achieving this goal. By broadly participating in the Registry, our families will help create a dataset that captures the diversity within Rett that scientists need to guide treatment development efforts that reach everyone.”
Clinical & Caregiver Data
To facilitate such wide participation in the Registry, IRSF and its Center of Excellence network are making it as simple as possible for families to get involved. The Registry has two components: clinical data collection and caregiver reported information. The caregiver reports can be filled out online at each family’s convenience. Meanwhile, the IRSF Center of Excellence physicians will collect the clinical data for the Registry as part of normal clinic visits. There are no examinations or tests individuals with Rett must undergo beyond the practices of current best-in-class clinical care.
“Having both physician and caregiver data is ideal for clinical trial researchers. It’s much easier to make sound clinical trial design decisions when there’s data that indicates how these two very different stakeholders in the treatment development process are viewing Rett syndrome symptoms” explains Dr. Sasha Djukic, the director of the Montefiore Medical Center IRSF Center of Excellence.
Families can also take encouragement in the knowledge that their participation in the Registry makes a big difference for Rett drug development efforts. In general, limited available data on rare diseases like Rett makes it more difficult for regulatory agencies such as the Food and Drug Administration (FDA) to approve clinical trial designs and treatments. The agencies simply do not have sufficient information to evaluate the designs or determine if a new treatment substantially improves symptoms. Datasets like the one the Rett Syndrome RegistryTM will create are one of the best tools that rare disease patient advocacy groups like IRSF can deploy to address this situation.
Increasing Clinical Trial Success
The Rett Syndrome RegistryTM was designed with guidance from the FDA and has been optimized to increase the chances of clinical trial success and help bring curative treatment options to families. The combination of clinical data with caregiver-reported information is one of the keys to this design. The FDA and pharmaceutical companies view this combination as essential for clinical trial design, treatment approval, and efforts to include the patient voice in drug development. Essentially, clinical researchers and the FDA will be able to use the Registry to see how both expert physicians who are positioned to evaluate Rett syndrome symptoms as objectively as possible and those who care for individuals with Rett everyday view Rett. They can then use this information to select clinical trial measurement and evaluation tools that make sense to both parties. It will also help them assess a new treatment’s effect on patients to determine if it merits approval.
The Registry will also build upon the clinical trial readiness foundation laid by the Rett Syndrome Natural History Study (NHS) that is ending on July 31, 2021. Careful planning has been undertaken with key stakeholders so that the Rett Syndrome Registry™ benefits from the 15 years of data collected through the Rett Syndrome NHS and fills the gaps that exist in clinical trial readiness.
“To those of you who participated in the NHS, thank you,” shares IRSF CEO Melissa Kennedy. “Our Foundation has assumed stewardship of that database, ensuring that your time and efforts will continue to support research and drug development. Whether you and your family participated in the Rett Syndrome NHS or not, you can still help create a world without Rett syndrome.”
Sign-up to learn more about taking part in the Rett Syndrome Registry™ and get connected with your nearest IRSF Center of Excellence.
We won’t stop until we have treatments and a cure for all with Rett syndrome, but we can’t do it without you. Please sign-up today.