RB001: The study enrolled up to 2 cohorts, evaluating a higher or lower dose
Study summary
This is a long-term follow-up study of participants in the RB001-101 gene replacement therapy clinical trial for Children With SHANK3-related Phelan-McDermid Syndrome (PMS). Participants will roll over from the parent study into this long-term study for continuous safety monitoring and preliminary efficacy assessment for up to 5 years.
Eligibility
Sex
ALL
Min age
—
Max age
—
Healthy volunteers
No
Inclusion Criteria:
* Patients with SHANK3-related Phelan-McDermid Syndrome who have completed the last follow-up visit (Week 52 post-dosing) in the RB001-101 study.
Exclusion Criteria:
* Some conditions deemed unsuitable for participation in this study by the investigator
Primary outcome measure(s)
To evaluate the safety and tolerability of a single intracerebroventricular injection of RB001 — 1 to 5 years Types, severity, and incidence of adverse events (AEs) and serious adverse events (SAEs)
Trial sites (1)
Facility
City
Region
Status
Peking University First Hospital
Beijing
China
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This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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