FDA Provides Positive Feedback About Rett Syndrome Global Registry

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by Patricia Inácio, PhD |

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The U.S. Food and Drug Administration (FDA) has provided positive feedback about the Rett Syndrome Global Registry — a database of clinical and caregiver-provided information on Rett syndrome patients — whose ultimate goal is to help develop novel therapeutics and a potential cure.

The Rett Syndrome Research Trust (RSRT) presented the registry in a non-regulatory meeting with the FDA.

“Meeting with FDA to discuss our vision for the Rett Syndrome Global Registry, outline how this database meets the high standards needed for clinical trial datasets, and how we plan to support companies was an opportunity to receive guidance from the agency,” Jana von Hehn, PhD, senior director of research and clinical strategy, and the RSRT’s registry director, said in a press release. “The FDA guidance will help to ensure our database can support clinical development and approval of novel therapeutics as we intend.”

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The design of the registry, done in collaboration with DSG — a clinical trial database provider — combines clinical data of Rett patients from several sources and caregiver information in a single, clinical trial-grade format.

Data from caregivers, medical records, clinical trials, lab studies, and wearable and non-wearable biosensors potentially — devices that measure biomarkers — are among the registry’s sources.

The summary of all this information will provide the most unified picture to date of Rett syndrome to caregivers, researchers, biopharmaceutical companies, and regulators, according to the RSRT.

The meeting with the FDA follows the publication of the agency’s draft guidance for registries, in November 2021, including the potential of a registry to inform the design of and support clinical trials. One of the goals, according to the RSRT, is to leverage the registry so that it serves as a placebo control group in future trials.

“Leveraging trial-matched registry participants in treatment trials would lead to reduced enrollment numbers, shortened trial timelines, reduced trial costs, and accelerated completion of development programs,” said von Hehn. “We believe these domino effects would lead to earlier patient access to therapeutics.”

Moreover, the RSRT hopes the registry will enable the development of novel outcome measures in trials, including parent-reported questionnaires, and provide high-quality clinical research infrastructure.

“By nimbly gathering additional data and linking registry and medical record datasets to RSRT’s biorepository, biosensor data, and external studies, we will generate integrated datasets that will more powerfully accelerate therapeutic development,” said Randall Carpenter, MD, chief medical officer at the RSRT.

Added Tony Varano, DSG’s CEO: “The registry design is in full compliance with FDA’s recent guidance document on Assessing Registries for Regulatory Decision Making.”

It also includes prior unused resources: medical records of children with Rett and feedback from caregivers.

“The impetus for this new registry approach was to utilize untapped resources to facilitate therapeutic development,” said Monica Coenraads, CEO of the RSRT. “Two invaluable but essentially unused sources of information about Rett syndrome are the child’s medical records, which, for the most part, sit inaccessible and unaggregated in hospitals and doctors’ offices, and Rett caregivers, who manage their child’s symptoms every day.”

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The clinical component of the registry will be managed by Ciitizen, a health technology platform now part of Invitae.

Ciitizen collects medical information across all medical facilities where patients receive care, soon providing the most comprehensive digital resource of Rett’s natural course. Companies can access the data once it has been consolidated and de-identified.

The caregiver-driven component of the registry allows for parents to provide information about the patient, including symptoms and treatments. This is facilitated by tools for management of day-to-day care, including customizable symptom tracking, data summaries, and the option to connect with other families.

“By providing regular data summaries back to parents, we hope to give parents transparency into how powerful their collective information can be, not only for themselves, but for everyone,” said Coenraads.

The registry was developed with the input of three advisory committees — consisting of parents, clinicians, and biopharmaceutical representatives — to ensure that the needs of all parties are met.

The registry will become available soon, after ongoing testing is completed.