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Clinical trial

A Long-term Follow-up Study of an Open-label, Single-arm, Single-dose Escalation Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Intracerebroventricular Injection of RB001 in Participants With SHANK3-related Phelan-McDermid Syndrome.

NCT07690527 is registered in ClinicalTrials.gov with Peking University First Hospital as lead sponsor.

NCT NCT07690527
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Registry record

As filed in ClinicalTrials.gov.

Title
A Long-term Follow-up Study of an Open-label, Single-arm, Single-dose Escalation Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Intracerebroventricular Injection of RB001 in Participants With SHANK3-related Phelan-McDermid Syndrome.
Lead sponsor
Peking University First Hospital
Phase
Phase 1
Overall status
Enrolling by invitation
Start date
August 1, 2026
Completion date
December 1, 2031
Enrollment
8 (estimated)
Sites
1

Conditions

As the registry lists them; a condition with its own page is linked.

phelan-mcdermid syndrome, shank3 haploinsufficiency