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RO7248824 for Angelman Syndrome

Phase 1
Waitlist Available
Research Sponsored by Hoffmann-La Roche
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Stable medical status for at least 4 weeks prior to Screening and at the time of enrollment
Female participants must not be pregnant or breastfeeding and must meet specific contraceptive requirements
Must not have
Hospitalization for major medical or surgical procedure involving general anesthesia within 12 weeks of Screening or planned during the study
Use of antiplatelet or anticoagulant therapy
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline to last visit or early withdrawal
Awards & highlights
No Placebo-Only Group

Summary

This trial is a study to test a new drug for safety, tolerability, and how well it works. The study will enroll two groups of children, those aged 5-12 and those aged 1-4, and will test different doses of the drug in each group.

Who is the study for?
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.
What is being tested?
The trial tests the safety and effects of RO7248824 in children with AS. It's an open-label study where doses increase within participants based on age groups: ≥5 to ≤12 years for one set of cohorts, and ≥1 to ≤4 years for another. Each older cohort precedes a younger one, ensuring safety before advancing.
What are the potential side effects?
Potential side effects are not explicitly listed but may include reactions related to intrathecal therapy (administered into the spinal canal), such as headaches or discomfort at the injection site. As this is an early-phase trial assessing safety, close monitoring will identify any adverse effects.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My health has been stable for the last 4 weeks.
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I am not pregnant or breastfeeding and follow the required contraceptive guidelines.
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I agree to use contraception as required.
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I am between 1 and 12 years old.
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I have Angelman Syndrome confirmed by genetic testing.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I haven't been hospitalized for a major procedure under general anesthesia in the last 3 months.
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I am taking medication to prevent blood clots.
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I have a history of bleeding or clotting disorders.
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I have a condition that raises my risk of getting meningitis.
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I do not have brain or spinal conditions affecting spinal procedures.
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My condition is genetically confirmed with specific mutations.
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I have a history of HIV, hepatitis B, or hepatitis C.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline to last visit or early withdrawal
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to last visit or early withdrawal for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Frequency and Severity of Adverse Events (AEs)
Frequency and Severity of Serious Adverse Events (SAEs)
Frequency of Abnormal Electrocardiography (ECG) Values
+8 more
Secondary study objectives
AUC From Time 0 to Infinity (AUCinf) for RO7248824
Area Under Curve (AUC) From Time 0 to Time of Last Sampling Point or Last Quantifiable Sample, Whichever Comes First (AUC last) for RO7248824
Maximum Plasma Concentration Observed (Cmax) for RO7248824
+1 more

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

19Treatment groups
Experimental Treatment
Group I: OOE: RO7248824Experimental Treatment1 Intervention
Participants in the LTE part of the study will be given the opportunity to participate in the OOE part.
Group II: LTE: Cohort EB4 RO7248824Experimental Treatment1 Intervention
Participants continuing from MAD Cohort B5
Group III: LTE: Cohort EB3 RO7248824Experimental Treatment1 Intervention
Participants continuing from MAD cohorts B3 and B4
Group IV: LTE: Cohort EB2 RO7248824Experimental Treatment1 Intervention
Participants continuing from MAD cohorts B1 and B2
Group V: LTE: Cohort EB1 RO7248824Experimental Treatment1 Intervention
New participants (age 1-4) enrolling directly into the LTE
Group VI: LTE: Cohort EA4 RO7248824Experimental Treatment1 Intervention
Participants continuing from MAD Cohort A5
Group VII: LTE: Cohort EA3 RO7248824Experimental Treatment1 Intervention
Participants continuing from MAD cohorts A3 and A4
Group VIII: LTE: Cohort EA2 RO7248824Experimental Treatment1 Intervention
Participants continuing from multiple ascending dose (MAD) cohorts A1 and A2
Group IX: LTE: Cohort EA1 RO7248824Experimental Treatment1 Intervention
New participants (age 5-12) enrolling directly in the LTE part
Group X: LTE: Cohort B5 RO7248824Experimental Treatment1 Intervention
Participants 1-4 years
Group XI: Cohort B4 RO7248824Experimental Treatment1 Intervention
Participants 1-4 years
Group XII: Cohort B3 RO7248824Experimental Treatment1 Intervention
Participants 1-4 years
Group XIII: Cohort B2 RO7248824Experimental Treatment1 Intervention
Participants 1-4 years
Group XIV: Cohort B1 RO7248824Experimental Treatment1 Intervention
Participants 1-4 years
Group XV: Cohort A5 RO7248824Experimental Treatment1 Intervention
Participants 5-12 years
Group XVI: Cohort A4 RO7248824Experimental Treatment1 Intervention
Participants 5-12 years
Group XVII: Cohort A3 RO7248824Experimental Treatment1 Intervention
Participants 5-12 years
Group XVIII: Cohort A2 RO7248824Experimental Treatment1 Intervention
Participants 5-12 years
Group XIX: Cohort A1 RO7248824Experimental Treatment1 Intervention
Participants 5-12 years
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
RO7248824
2021
Completed Phase 1
~30

Find a Location

Who is running the clinical trial?

Hoffmann-La RocheLead Sponsor
2,456 Previous Clinical Trials
1,097,568 Total Patients Enrolled
3 Trials studying Angelman Syndrome
117 Patients Enrolled for Angelman Syndrome
Clinical TrialsStudy DirectorHoffmann-La Roche
2,225 Previous Clinical Trials
896,316 Total Patients Enrolled
1 Trials studying Angelman Syndrome
21 Patients Enrolled for Angelman Syndrome

Media Library

RO7248824 (Other) Clinical Trial Eligibility Overview. Trial Name: NCT04428281 — Phase 1
Angelman Syndrome Research Study Groups: LTE: Cohort B5 RO7248824, LTE: Cohort EB1 RO7248824, OOE: RO7248824, LTE: Cohort EA1 RO7248824, LTE: Cohort EA2 RO7248824, LTE: Cohort EA3 RO7248824, LTE: Cohort EA4 RO7248824, LTE: Cohort EB3 RO7248824, LTE: Cohort EB2 RO7248824, LTE: Cohort EB4 RO7248824, Cohort B2 RO7248824, Cohort B3 RO7248824, Cohort B4 RO7248824, Cohort A1 RO7248824, Cohort A2 RO7248824, Cohort A3 RO7248824, Cohort A4 RO7248824, Cohort A5 RO7248824, Cohort B1 RO7248824
Angelman Syndrome Clinical Trial 2023: RO7248824 Highlights & Side Effects. Trial Name: NCT04428281 — Phase 1
RO7248824 (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04428281 — Phase 1
~10 spots leftby Jul 2025
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