RO7248824 for Angelman Syndrome
Trial Summary
Will I have to stop taking my current medications?
The trial requires that certain medications be stable for a period before joining. Sleep medications must be stable for 4 weeks, epilepsy medications for 12 weeks, and psychotropic medications for 4 weeks before screening. Antiplatelet or anticoagulant therapy should not be used for 2 weeks prior to screening.
What is the purpose of this trial?
This is a phase I, multicenter, non-randomized, adaptive, open-label, multiple ascending, intra-participant, dose-escalation study with a long-term extension (LTE) part and an optional open-label extension (OOE) part. The objective of the study is to investigate the safety, tolerability, PK and PD of RO7248824 administered intrathecally (IT) in participants with AS. Two linked sets of dose escalation cohorts are planned based on two different age groups, namely participants with AS aged ≥ 5 to ≤ 12 years in cohorts A1 to A5 (with at least 2 participants ≤ 8 years old in each cohort) and AS participants aged ≥ 1 to ≤ 4 years in cohorts B1 to B5. The two sets of cohorts will be run in parallel, with each cohort A1 to A5 preceding and gating the linked cohort B1 to B5 (e.g., A1 precedes B1).
Research Team
Clinical Trials
Principal Investigator
Hoffmann-La Roche
Eligibility Criteria
This trial is for children with Angelman Syndrome (AS) aged 1 to 12 years who can undergo procedures under sedation or anesthesia. They must have a confirmed clinical diagnosis of AS, be able to tolerate blood draws, and have a caregiver willing to comply with study requirements. Participants should not be pregnant or breastfeeding and must meet certain contraceptive requirements.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Multiple ascending dose (MAD) phase where participants receive RO7248824 intrathecally
Long-term extension (LTE)
Participants continue to receive RO7248824 to assess long-term safety and efficacy
Open-label extension (OOE)
Participants in the LTE part of the study will be given the opportunity to participate in the OOE part
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- RO7248824
Find a Clinic Near You
Who Is Running the Clinical Trial?
Hoffmann-La Roche
Lead Sponsor
Dr. Levi Garraway
Hoffmann-La Roche
Chief Medical Officer since 2019
MD from the University of Basel
Dr. Thomas Schinecker
Hoffmann-La Roche
Chief Executive Officer since 2023
PhD in Molecular Biology from New York University