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Enrolling By Invitation
NCT07690527
Long-Term Follow-Up Study for RB001 Gene Therapy Study in Children With SHANK3-related Phelan McDermid Syndrome (PMS)
Conditions: SHANK3 Haploinsufficiency, Phelan-McDermid Syndrome
Sex: All
Healthy volunteers: No
Phase: PHASE1
Enrollment: 8
Sponsor: Peking University First Hospital
Location: Peking University First Hospital Beijing China
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 Criteria
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
Source: ClinicalTrials.gov (NCT07690527). StuddyBuddy aggregates publicly available trial information.
