Status and phase
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About
This is a Phase I, multicenter, non-randomized, adaptive, open label, multiple ascending, intra-participant, dose-escalation study with an LTE part. The objective of the study is to investigate the safety, tolerability, PK and PD of RO7248824 in participants administered IT 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 A4 (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-A4 preceding and gating the linked cohort B1-B5 (e.g., A1 precedes B1).
Enrollment
Sex
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Volunteers
Inclusion criteria
Reproductive Status:
Some of the provisions that follow may have limited applicability based on the age range of study participants (i.e., up to the age of 12) and the nature of the disease understudy. These provisions are nonetheless included for purposes of completeness in order:
Female Participants
A female participant is eligible to participate if she is not pregnant, not breastfeeding, and at least one of the following conditions applies:
Male Participants
During the treatment period and for at least 6 months after the final dose of RO7248824 (RG6091), consent has to be provided to:
The reliability of sexual abstinence for male and/or female enrollment eligibility needs to be evaluated in relation to the duration of the clinical study and the preferred and usual lifestyle of the participant. Periodic abstinence (e.g., calendar, ovulation, symptothermal, or post-ovulation methods) and withdrawal are not acceptable methods of preventing drug exposure.
Exclusion criteria
Diagnostic Assessments
Type of Participants and Disease Characteristics
UBE3A missense mutation of maternal allele Paternal Uniparental Disomy (UPD) of 15q11-13 UBE3A Imprinting center defect (ID) A partial molecular diagnosis of AS, that cannot exclude UPD or ID despite appropriate genetic testing.
Medical history and concurrent disease
Prior Therapy
Other Exclusion Criteria: Prior/Concurrent Clinical Study Experience
Primary purpose
Allocation
Interventional model
Masking
74 participants in 18 patient groups
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Central trial contact
Reference Study ID Number: BP41674 https://forpatients.roche.com/
Data sourced from clinicaltrials.gov
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