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

Study of AAV-hSMN1 (Vesemnogene Lantuparvovec) Gene Therapy in Subjects With Progressive Spinal Muscular Atrophy

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

Study Overview

• Status: Recruiting
• Conditions: Spinal Muscular Atrophy
• Intervention / Treatment: Biological: vesemnogene lantuparvovec

Detailed Description

Study duration per participant is approximately 25 months including an approximately 30-day screening/baseline period, an approximately 24-month study observation period including 1 treatment day, and an approximately 24-month follow-up period. Patients will be stratified in two groups, those < 24 months of age at time of dosing and those ≥ 24 months of age at time of dosing. This study will be conducted in 2 stages:

Stage 1: dose escalting study in children <24 months of age. Stage 2: the selecetd dose from Stage 1 in children ≥ 24 months of age.

Patients will be tested at baseline and return for follow-up visits twice a week through the first month post dose, and followed by visits at months 2, 3, 6 12, 18 and 24 post infusion. Unscheduled visits may occur if the investigator determines that they are necessary.

For patients enrolled from overseas, follow-up visits with patient's own paediatrician together with remote virtual visits are allowed.

• Study Type: Interventional
• Enrollment (Estimated): 20
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Austin Gao, PhD; Phone Number: +8617724360504; Email: clinicaltrials@lantubiopharma.com

Study Locations

• China
  • Yunnan
    • Kunming, Yunnan, China, 650200
      • Recruiting
      • Kunming Hope of Health Hospital
      • Contact: Austin Gao, PhD; Email: clinicaltrials@lantubiopharma.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
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	Biological: vesemnogene lantuparvovec; Exploratory study evaluating the safety and efficacy of vesemnogene lantuparvovec in patients with SMA.; Other Names: AAV-hSMN1
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	Biological: vesemnogene lantuparvovec; Exploratory study evaluating the safety and efficacy of vesemnogene lantuparvovec in patients with SMA.; Other Names: AAV-hSMN1
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	Biological: vesemnogene lantuparvovec; Exploratory study evaluating the safety and efficacy of vesemnogene lantuparvovec in patients with SMA.; Other Names: AAV-hSMN1

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Numbers of participants with adverse events (AEs), serious adverse events (SAEs)	Participants are monitored for safety from baseline up to the end of the follow-up period.	Baseline up to 24 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from baseline in developmental gross motor milestones achieved according to WHO criteria	For patients with SMA, the percentage of participants who are able to preserve ambulatory function.	Baseline up to 24 months
Change from baseline in CHOP-INTEND in children <24 months of age	The CHOP-INTEND contains 16 items rated from 0 to 4. Total scores range from 0-64. Higher scores indicate higher levels of motor ability.	Baseline up to 24 months
Change from baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) in children ≥ 24 months of age	The HFMSE contains 33 items rated from 0 (unable to perform) to 2 (performs without modification/adaptation/compensation). Total scores range from 0-66. Higher scores indicate higher levels of motor ability.	Baseline up to 24 months

Collaborators and Investigators

• Sponsor: Lantu Biopharma

Study record dates

Study Major Dates

• Study Start (Actual): October 20, 2024
• Primary Completion (Estimated): October 30, 2027
• Study Completion (Estimated): October 30, 2027

Study Registration Dates

• First Submitted: February 18, 2024
• First Submitted That Met QC Criteria: February 24, 2024
• First Posted (Actual): March 1, 2024

Study Record Updates

• Last Update Posted (Actual): July 22, 2025
• Last Update Submitted That Met QC Criteria: July 17, 2025
• Last Verified: July 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neurologic Manifestations; Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Manifestations; Pathological Conditions, Anatomical; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; Motor Neuron Disease; Atrophy; Muscular Atrophy; Muscular Atrophy, Spinal
• Other Study ID Numbers: LT01-101

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No