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

Conditions: Spinal Muscular Atrophy

Sex: All

Healthy volunteers: No

Phase: PHASE1, PHASE2

Enrollment: 20

Sponsor: Lantu Biopharma

Location: Kunming Hope of Health Hospital Kunming Yunnan

Summary

This is an interventional study to evaluate safety and efficacy of AAV-hSMN1 in spinal muscular atrophy patients.

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 \

Interested in this study? View the official listing for contact and enrollment details.

View on ClinicalTrials.gov

Source: ClinicalTrials.gov (NCT06288230). StuddyBuddy aggregates publicly available trial information.