Study of AAV-hSMN1 (Vesemnogene Lantuparvovec) Gene Therapy in Subjects With Progressive Spinal Muscular Atrophy
Status: Recruiting
Location: See location...
Intervention Type: Biological
Study Type: Interventional
Study Phase: Phase 1/Phase 2
SUMMARY
This is an interventional study to evaluate safety and efficacy of AAV-hSMN1 in spinal muscular atrophy patients.
Eligibility
Participation Requirements
Sex: All
Healthy Volunteers: f
View:
• 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.
Locations
Other Locations
China
Kunming Hope of Health Hospital
RECRUITING
Kunming
Contact Information
Primary
Austin Gao, PhD
clinicaltrials@lantubiopharma.com
+8617724360504
Time Frame
Start Date: 2024-10-20
Estimated Completion Date: 2027-10-30
Participants
Target number of participants: 20
Treatments
Experimental: Stage 1: Dose A in children < 24 months of age
Dose escalting study: Administration Dose A of Vesemnogene Lantuparvovec in children \< 24 months of age
Experimental: Stage 1: Dose B in children < 24 months of age
Dose escalting study: Administration Dose B of Vesemnogene Lantuparvovec in children \< 24 months of age
Experimental: Stage 2: The selected dose in children ≥ 24 months of age
Administration the selected dose of Vesemnogene Lantuparvovec in children ≥ 24 months of age
Related Therapeutic Areas
Sponsors
Leads: Lantu Biopharma