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Gene Therapy for IGHMBP2-Related Diseases

NCT05152823 · Status: ENROLLING_BY_INVITATION · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2025-09-30

No results posted yet for this study

Summary

Open-label, single intrathecal injection study of a AAV9 vector carrying the IGHMBP2 gene for IGHMBP2-related diseases.

Conditions

  • SMARD1
  • CMT2S

Interventions

BIOLOGICAL

Gene Therapy

AAV9 carrying the IGHMBP2 gene.

Sponsors & Collaborators

  • Megan Waldrop

    lead OTHER

Principal Investigators

  • Megan Waldrop, MD · Nationwide Children's Hospital

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Months
Max Age
14 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-11-04
Primary Completion
2028-07-31
Completion
2030-07-31
FDA Drug
Yes

Countries

  • United States

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 NCT05152823 on ClinicalTrials.gov