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

U.S., July 9 -- ClinicalTrials.gov registry received information related to the study (NCT07690527) titled 'Long-Term Follow-up Study for RB001 Gene Therapy Study in Children With SHANK3-related Phelan McDermid Syndrome (PMS)' on July 01.

Brief 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.

Study Start Date: Aug. 01

Study Type: INTERVENTIONAL

Condition: SHANK3 Haploinsufficiency Phelan-McDermid Syndrome

Intervention: BIOLOGICAL: RB001

The...