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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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline to last visit or early withdrawal
Awards & highlights

Study 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.Check my eligibility
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.See study design
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.

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Frequency And Severity Of Adverse Events
Frequency And Severity Of Serious Adverse Events
Frequency Of Abnormal ECG Values
+8 more
Secondary outcome measures
AUC From Time 0 To Infinity (AUCinf) for RO7248824
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

Trial Design

18Treatment groups
Experimental Treatment
Group I: Cohort EB4 RO7248824Experimental Treatment1 Intervention
Participants continuing from MAD Cohort B5
Group II: Cohort EB3 RO7248824Experimental Treatment1 Intervention
Participants continuing from MAD cohorts B3 and B4
Group III: Cohort EB2 RO7248824Experimental Treatment1 Intervention
Participants continuing from MAD cohorts B1 and B2
Group IV: Cohort EB1 RO7248824Experimental Treatment1 Intervention
New participants (age 1-4) enrolling directly into the LTE
Group V: Cohort EA4 RO7248824Experimental Treatment1 Intervention
Participants continuing from MAD Cohort A5
Group VI: Cohort EA3 RO7248824Experimental Treatment1 Intervention
Participants continuing from MAD cohorts A3 and A4
Group VII: Cohort EA2 RO7248824Experimental Treatment1 Intervention
Participants continuing from MAD cohorts A1 and A2
Group VIII: Cohort EA1 RO7248824Experimental Treatment1 Intervention
New participants (age 5-12) enrolling directly in the LTE part
Group IX: Cohort B5 RO7248824Experimental Treatment1 Intervention
Participants 1-4 Years
Group X: Cohort B4 RO7248824Experimental Treatment1 Intervention
Participants 1-4 Years
Group XI: Cohort B3 RO7248824Experimental Treatment1 Intervention
Participants 1-4 Years
Group XII: Cohort B2 RO7248824Experimental Treatment1 Intervention
Participants 1-4 Years
Group XIII: Cohort B1 RO7248824Experimental Treatment1 Intervention
Participants 1-4 Years
Group XIV: Cohort A5 RO7248824Experimental Treatment1 Intervention
Participants 5-12 Years
Group XV: Cohort A4 RO7248824Experimental Treatment1 Intervention
Participants 5-12 Years
Group XVI: Cohort A3 RO7248824Experimental Treatment1 Intervention
Participants 5-12 Years
Group XVII: Cohort A2 RO7248824Experimental Treatment1 Intervention
Participants 5-12 Years
Group XVIII: 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
2,428 Previous Clinical Trials
1,088,980 Total Patients Enrolled
3 Trials studying Angelman Syndrome
117 Patients Enrolled for Angelman Syndrome
Clinical TrialsStudy DirectorHoffmann-La Roche
2,199 Previous Clinical Trials
888,428 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: Cohort B2 RO7248824, Cohort EB3 RO7248824, Cohort EB4 RO7248824, Cohort B3 RO7248824, Cohort B4 RO7248824, Cohort B5 RO7248824, Cohort EA1 RO7248824, Cohort EA2 RO7248824, Cohort EA3 RO7248824, Cohort EA4 RO7248824, Cohort EB1 RO7248824, Cohort EB2 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is this research initiative allowing elderly individuals to participate?

"The parameters for enrolling in this clinical trial dictate that applicants should be between one and twelve years old. This particular study has a total of 375 participants aged below 18, while there are 983 participants over 65 taking part."

Answered by AI

Are there any possible risks associated with RO7248824 treatment?

"RO7248824 has a safety rating of 1 due to it being in the first stage of clinical trials and thus having limited evidence showing its efficacy or security."

Answered by AI

Is there currently any opportunity to join this research study?

"Affirmative. Clinicaltrials.gov data displays that this trial, initial posted on August 19th 2020, is presently seeking participants. 74 people need to be recruited from 6 distinct medical centres."

Answered by AI

What is the major goal of this research endeavor?

"The primary endpoint of the investigation will be to gauge any changes in temperature over a Baseline-to-last visit or early withdrawal timeline. Secondary objectives include measuring Maximum Plasma Concentration Observed (Cmax) for RO7248824, Area Under Curve From Time 0 To Infinity (AUCinf) and Area Under Curve From Time 0 To Last Sampling Point Or Last Quantifiable Sample, Whichever Comes First (AUC last). These outcomes need to be evaluated from baseline MAD/LTE visits until D365 or earlier withdrawals."

Answered by AI

How many healthcare facilities are taking part in this research experiment?

"This clinical trial is currently taking place in 6 different medical centres, located in Rochester, Chicago and Houston as well as other areas. It would be wise to select the clinic that best suits your geographical needs should you choose to partake in this study."

Answered by AI

Do I meet the criteria to be included in this experiment?

"This clinical trial seeks 74 Angelman syndrome patients between the ages of one and twelve. To qualify, they must be able to withstand blood draws and LP or IT injections if needed; have a competent caregiver aged 18 or older who is contactable by phone or e-mail; consent in accordance with international regulations; comply with all study requirements; possess adequate psychosocial circumstances; have maintained stable medical status for four weeks prior to screening, weigh at least seven kilograms, and present with either UBE3A mutation on the maternal allele or deletion within 7 Mb size on maternally inherited chromosome 15q11q13."

Answered by AI

How many subjects are enrolled in this clinical trial?

"This clinical trial necessitates 74 eligible participants, and can be accessed from various locations such as Rochester's Mayo Clinic or Chicago's Rush Medical Centre."

Answered by AI

Who else is applying?

What state do they live in?
California
What site did they apply to?
UCLA Neuropsychiatric Institute
What portion of applicants met pre-screening criteria?
Met criteria

How responsive is this trial?

Average response time
  • < 1 Day
Typically responds via
Phone Call
Most responsive sites:
  1. UCLA Neuropsychiatric Institute: < 24 hours
Recent research and studies
clinicaltrials.govStudy
A Study To Investigate The Safety, Tolerability, Pharmacokinetics And Pharmacodynamics Of RO7248824 In Participants With Angelman Syndrome
2020. "A Study To Investigate The Safety, Tolerability, Pharmacokinetics And Pharmacodynamics Of RO7248824 In Participants With Angelman Syndrome". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04428281.
All > Angelman Syndrome
~5 spots leftby Aug 2024
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