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Gene Therapy Trial Report

Summary

An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy


NCTID NCT06288230 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Spinal Muscular Atrophy
Disease Ontology Term DOID:13137
Compound Name Vesemnogene lantuparvovec
Compound Description AAV-hSMN1
Sponsor Lantu Biopharma
Funder Type Industry
Recruitment Status
Enrollment Count 20 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant SMN1
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV
Dose 1 1.5E14 vg
Dose 2 3.0E14 vg

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2024-02-18
Completion Date 2027-10-30
Last Update 2025-07-22

Participation Criteria


Eligible Age
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: * Diagnosis of SMA based on gene mutation analysis with bi-allelic survival motor neuron (SMN1) mutations (deletion or point mutations). * Patients or Parent(s)/legal guardian(s) willing and able to complete the informed consent process and comply with study procedures and visit schedule. Exclusion Criteria: * Anti-AAV9 antibody titers \>1:20 as determined by Enzyme-linked Immunosorbent Assay (ELISA) binding immunoassay. * Active viral infection (includes HIV or serology positive for hepatitis B or C). * Use of invasive ventilatory support (tracheotomy with positive pressure) or pulse oximetry \<95% saturation. * Concomitant illness and any drug that in the opinion of the investigator creates unnecessary risks for gene transfer. * Clinically significant abnormal laboratory values. * Participation in a recent SMA treatment clinical trial that in the opinion of the PI creates unnecessary risks for gene transfer. * Patient with signs of aspiration based on a swallowing test and unwilling to use an alternative method to oral feeding. * For children ≥ 24 months of age, contraindications for spinal tap procedure or administration of intrathecal therapy or presence of an implanted shunt for the drainage of CSF or an implanted central venous (CNS) catheter. * For children ≥ 24 months of age, severe contractures as determined by Physical Therapist(s) at screening that interfere with either the ability to attain/demonstrate functional measures or interferes with ability to receive dosing. * For children ≥ 24 months of age, severe scoliosis (defined as ≥ 50° curvature of spine) evident on X-ray examination. * For children ≥ 24 months of age, previous, planned or expected scoliosis repair surgery/procedure within 1 year of dose administration.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations China

Regulatory Information


Has US IND False
FDA Designations
Recent Updates

Resources/Links