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NCT04428281 · ClinicalTrials.gov registry record · Phase 1
A Study to Investigate the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of RO7248824 in Participants With Angelman Syndrome (AS)
A Phase 1 study of Angelman Syndrome, sponsored by Hoffmann-La Roche.
- Completed
- Registry status
- Phase 1
- Development phase
- 74
- Enrollment target
- 12
- Study locations
NCT04428281: Completed Phase 1 study of Angelman Syndrome, sponsored by Hoffmann-La Roche.
NCT04428281 is a Phase 1 study of Angelman Syndrome that has completed, run by Hoffmann-La Roche. The registered enrollment target is 74 participants, below the 120-participant average among 11 other Angelman Syndrome trials with a reported enrollment target (38% lower). The trial reports 12 study locations across 9 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04428281, a Phase 1 study of Angelman Syndrome, has completed, sponsored by Hoffmann-La Roche.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 74 participants
- Enrollment target
- 12
- Study locations
Study Summary
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).
Primary Outcome
MAD part: From the baseline MAD visit to the final MAD visit (Day 365) or early withdrawal. LTE part: From the baseline LTE visit to final LTE visit (Day 1092) or early withdrawal. OOE part: From baseline OOE visit to final OOE visit (Day 420) or early termination.
Conditions Studied
Interventions
- DRUG RO7248824
Study Locations (12)
California
- UCLA Neuropsychiatric Institute - Los Angeles
- Rady Children's Hospital - San Diego - San Diego
Other
- Erasmus MC / location Sophia Kinderziekenhuis - Rotterdam
- Hospital Universitario Virgen del Rocío - Seville
Barcelona
- Hospital Sant Joan De Deu - Esplugues de Llobregas
- Corporacio Sanitaria Parc Tauli - Sabadell
Illinois
- Rush Medical Center - Chicago
Minnesota
- Mayo Clinic - Rochester - Rochester
New York
- Columbia University Medical Center - New York
North Carolina
- Carolina Institute for Development DisabilitiesUniversity of North Carolina/School of Medicine - Carrboro
Texas
- Baylor College of Med - Houston
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 74 participants |
| Start Date | 2020-08-19 |
| Est. Completion | 2025-07-31 |
| Phase | Phase 1 |
What the finished NCT04428281 record still lists
NCT04428281 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 74 participants, a relatively small participant target, below the 120-participant average among 11 other Angelman Syndrome trials with a reported enrollment target (38% lower).
The record links to 1 condition, with Angelman Syndrome appearing as the primary indexed condition, and to 1 intervention - of which RO7248824 is the first listed.
NCT04428281 lists 12 locations in 9 states (California, Other, Barcelona).
Frequently Asked Questions
What is clinical trial NCT04428281 about?
NCT04428281 is a clinical study titled "A Study to Investigate the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of RO7248824 in Participants With Angelman Syndrome (AS)". 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 o...
What is the current status of trial NCT04428281?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 74 participants. The study started on 2020-08-19. Estimated completion is 2025-07-31.
What conditions does trial NCT04428281 study?
This clinical trial studies the following conditions: Angelman Syndrome.
What interventions are being tested in trial NCT04428281?
The interventions under investigation include: RO7248824 (DRUG).
Who is sponsoring clinical trial NCT04428281?
This trial is sponsored by Hoffmann-La Roche, which has 909 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04428281 being conducted?
This trial has 12 study locations across California, Illinois, Minnesota, New York, North Carolina. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Angelman Syndrome
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT04428281's enrollment target sits among peer trials
74 8th of 11 higher than 4 of 11 other Angelman Syndrome trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Angelman Syndrome trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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