Long-Term Follow-Up Study for RB001 Gene Therapy Study in Children With SHANK3-related Phelan McDermid Syndrome (PMS)

Enrolling by invitation · Phase 1

Conditions studied: SHANK3 Haploinsufficiency, Phelan-McDermid Syndrome

In brief

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.

Key facts

Study ID

NCT07690527

Run by

Peking University First Hospital

People needed

8

Starts

2026-08-01

Expected to finish

2031-12-01

Last updated by the study team

2026-07-13

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Patients with SHANK3-related Phelan-McDermid Syndrome who have completed the last follow-up visit (Week 52 post-dosing) in the RB001-101 study.

You may not qualify if…

• Some conditions deemed unsuitable for participation in this study by the investigator

Where it is running

• Peking University First Hospital — Beijing, China, China

Full record on ClinicalTrials.gov

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