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Clinical trial

An Open-label, Single Arm, Dose-Escalation Clinical Study to Evaluating the Safety, Tolerability and Preliminary Efficacy of a Single Intracerebroventricular Injection of RB001 for the Treatment of SHANK3-related Phelan-McDermid Syndrome.

NCT07014020 is registered in ClinicalTrials.gov with Peking University First Hospital as lead sponsor.

NCT NCT07014020
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Registry record

As filed in ClinicalTrials.gov.

Title
An Open-label, Single Arm, Dose-Escalation Clinical Study to Evaluating the Safety, Tolerability and Preliminary Efficacy of a Single Intracerebroventricular Injection of RB001 for the Treatment of SHANK3-related Phelan-McDermid Syndrome.
Lead sponsor
Peking University First Hospital
Phase
Phase 1
Overall status
Active, not recruiting
Start date
June 16, 2025
Completion date
December 31, 2027
Enrollment
8 (actual)
Sites
1

Conditions

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

phelan-mcdermid syndrome, shank3 haploinsufficiency