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Observer-Reported Communication Ability (ORCA)

The ORCA study will adapt a communications-assessment tool for use in clinical trials to determine the efficacy of Rett treatments. The study has successfully completed Phase 1 and is now entering Phase 2. In this second phase we need to enroll 250 Rett syndrome families from English-speaking countries, including the US, Canada, United Kingdom, and Australia. Your participation is critical. To participate please complete the survey linked here. Please note the survey will take approximately 45 minutes to complete. Families of boys with Rett syndrome with confirmed MECP2 mutation are encouraged to participate.

ORCA Study Addresses Communication, a Top Concern of Rett Parents

Most parents of non-verbal children would agree that communication is at the top of the symptoms list they would like addressed. Currently a communication-focused assessment tool does not exist for Rett. However, one does exist for Angelman syndrome (a disorder with similarities to Rett), called the Observer-Reported Communication Ability (ORCA). We are leveraging the efforts already invested in ORCA by the Angelman community to adapt ORCA for Rett.

The ORCA was developed by the Center for Health Measurement (CHM) at Duke University School of Medicine. It includes 72 questions that capture various types of expressive, receptive, and pragmatic forms of communication and places individuals on a continuum of communication ability that detects changes over time. The ORCA does not rely on speech, but allows gestures, vocalizations, and use of aids to capture communication ability. It takes about 45 minutes for a parent or caregiver to complete the measure independently, and it does not require the help of a clinician or speech language pathologist. The ORCA is now being used in clinical trials and natural history studies for individuals with Angelman syndrome.

RSRT has provided funding to Dr. Reeve, the director of CHM, and his team to develop the ORCA for Rett. During Phase 1 a number of one-on-one interviews with parents of individuals with Rett took place. We are now ready for Phase 2 which will require enrolling 250 Rett syndrome families to complete the newly revised ORCA. Our hope is that the ORCA will become the standard tool to assess communication abilities in Rett syndrome and an important addition to assessing efficacy in therapeutic development.