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Clinical Trial Report

Gene Therapy Trial Report

Summary

Gene Therapy for IGHMBP2-Related Diseases

NCTID NCT05152823 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Spinal Muscular Atrophy with Respiratory Distress
Disease Ontology Term DOID:0111064
Compound Name ACTX-401
Compound Alias AAV9-IGHMBP2
Sponsor Megan Waldrop
Funder Type Other
Recruitment Status
Enrolling by invitation
Enrollment Count 10 (ESTIMATED)
Results Posted Not Available

Therapy Information

Target Gene/Variant IGHMBP2
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intrathecal
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV9
Editor Type
Dose 1 Undisclosed
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates

Current Stage Phase1, Phase2
Submit Date 2021-11-25
Completion Date 2030-07
Last Update 2025-09-30

Participation Criteria

Eligible Age 2 Months - 14 Years
Standard Ages Child
Sexes Eligible for Study ALL

Locations

No.of Trial Sites 1
Locations United States

Regulatory Information

Has US IND True
FDA Designations
Recent Updates Alcyone Therapeutics partnered with Abigail Wexner Research Institute (Nationwide Children's Hospital)

Resources/Links

Preclinical Publications