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Investigation the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of RO7248824 in Participants with Angelman Syndrome
EU CTIS ID: 2024-514797-45-00
What this study is testing
To assess the safety and tolerability profile of RO7248824
- Human Pharmacology (Phase I)- Other
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- The participant has a parent, caregiver or legal representative (hereinafter “caregiver”) who is reliable, competent and at least 18 years of age. The caregiver is willing and able to accompany the participant to clinic visits and to be available to the Investigational Site by phone or email if needed and who (in the opinion of the investigator) is and will remain sufficiently knowledgeable of participant’s ongoing condition to respond to any inquiries about the participant from personnel from the Study Site
- Clinical diagnosis of AS confirmed by a molecular diagnosis with genotypic classification of either: ••Ubiquitin-protein ligase E3A (UBE3A) mutation of maternal allele Deletion on the maternally inherited chromosome 15q11q13 that includes the UBE3Agene and is less than 7 Mb in size
- Stable medical status for at least 4 weeks prior to Screening and at the time of enrollment
- Have adequate supportive psychosocial circumstances
- Able to tolerate blood draws
- Have adequate supportive psychosocial circumstances
You likely can't join if
- Clinically relevant hematological, hepatic, cardiac, renal disease event, or laboratory abnormality, in the judgment of the Investigator
- Any concomitant condition that might interfere with the clinical evaluation of AS and that is not related to AS
- Known history of human immunodeficiency virus (HIV) or hepatitis B virus (HBV) or hepatitis C virus (HCV)
- Any condition that increases risk of meningitis
- History of bleeding diathesis or coagulopathy
- History of clinically significant post-lumbar-puncture headache of moderate or severe intensity and/or blood patch
The study team makes the final eligibility decision.
Where it's taking place
- United States
- Canada
- United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; Canada; United Kingdom. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.