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).
Age range
1 Year – 12 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Frequency and Severity of Adverse Events (AEs)
Timeframe: Baseline to last visit or early withdrawal
Frequency and Severity of Serious Adverse Events (SAEs)
Timeframe: Baseline to last visit or early withdrawal
Number of Participants Discontinued Treatment due to AEs
Timeframe: Baseline to last visit or early withdrawal
Frequency of Abnormal Laboratory Findings (Blood, Cerebrospinal Fluid [CSF] and Urinalysis)
Timeframe: Baseline to last visit or early withdrawal
Frequency of Abnormal Vital Signs
Timeframe: Baseline to last visit or early withdrawal
Frequency of Abnormal Electrocardiography (ECG) Values
Timeframe: Baseline to last visit or early withdrawal
Mean Changes From Baseline in Temperature Over Time
Timeframe: Baseline to last visit or early withdrawal
Mean Changes From Baseline in Systolic Blood Pressure Over Time
Timeframe: Baseline to last visit or early withdrawal
Mean Changes From Baseline in Diastolic Blood Pressure Over Time
Timeframe: Baseline to last visit or early withdrawal
Mean Changes From Baseline in Heartrate Over Time
Timeframe: Baseline to last visit or early withdrawal
Mean Changes From Baseline in Respiratory Rate Over Time
Timeframe: Baseline to last visit or early withdrawal