Gene Therapy for IGHMBP2-Related Diseases

Phase I/IIa Intrathecal Gene Delivery Clinical Trial for IGHMBP2-Related Diseases

NCT05152823•Megan Waldrop

View on ClinicalTrials.gov

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Conditions

SMARD1CMT2S

Interventions

Gene Therapy

Locations

United States

Locations (1)

Nationwide Children's Hospital

Columbus, Ohio, United States

Key Dates

Start Date

November 4, 2021

Primary Completion Date

July 1, 2028

Completion Date

July 1, 2030

Last Updated

September 30, 2025

Sponsors

Lead Sponsor

Megan Waldrop

Full Eligibility Criteria

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: September 30, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

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