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

NCT07690527 · Status: ENROLLING_BY_INVITATION · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 8

Last updated 2026-07-13

No results posted yet for this study

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.

Conditions

  • SHANK3 Haploinsufficiency
  • Phelan-McDermid Syndrome

Interventions

BIOLOGICAL

RB001

The study enrolled up to 2 cohorts, evaluating a higher or lower dose

Sponsors & Collaborators

  • Shenzhen Reborngene Therapeutics Co., Ltd.

    collaborator UNKNOWN

  • Peking University First Hospital

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-08-01
Primary Completion
2031-07-01
Completion
2031-12-01

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07690527 on ClinicalTrials.gov