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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 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.

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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).

Conditions Studied

Angelman Syndrome

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

FieldValue
Enrollment Target 74 participants
Start Date 2020-08-19
Est. Completion 2025-07-31
Phase Phase 1

Sponsor

Hoffmann-La Roche

909 total trials

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What the Registry Record Tells You About NCT04428281

The ClinicalTrials.gov registry entry for NCT04428281 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 74 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 120-participant average among 11 other Angelman Syndrome trials with a reported enrollment target (38% lower). The listed sponsor is Hoffmann-La Roche, which has 909 total studies on file at ClinicalTrials.gov.

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 reports 12 study locations spanning 9 distinct geographic areas - top geographies include 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.

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Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.