A Study to Investigate the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of RO7248824 in Participants With Angelman Syndrome (AS)
Public ClinicalTrials.gov record NCT04428281. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
An Open-label, Multicenter Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of RO7248824 in Participants With Angelman Syndrome
Study identification
- NCT ID
- NCT04428281
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 1
- Lead sponsor
- Hoffmann-La Roche
- Industry
- Enrollment
- 74 participants
Conditions and interventions
Conditions
Interventions
- RO7248824 Drug
Drug
Eligibility (public fields only)
- Age range
- 1 Year to 12 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Aug 18, 2020
- Primary completion
- Jul 30, 2025
- Completion
- Jul 30, 2025
- Last update posted
- Aug 23, 2025
2020 – 2025
United States locations
- U.S. sites
- 7
- U.S. states
- 6
- U.S. cities
- 7
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| UCLA Neuropsychiatric Institute | Los Angeles | California | 90024 | — |
| Rady Children's Hospital - San Diego | San Diego | California | 92123 | — |
| Rush Medical Center | Chicago | Illinois | 60612 | — |
| Mayo Clinic - Rochester | Rochester | Minnesota | 55905 | — |
| Columbia University Medical Center | New York | New York | 10032 | — |
| Carolina Institute for Development DisabilitiesUniversity of North Carolina/School of Medicine | Carrboro | North Carolina | 27510 | — |
| Baylor College of Med | Houston | Texas | 77030 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 5 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04428281, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Aug 23, 2025 · Synced Jul 23, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04428281 live on ClinicalTrials.gov.