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

NCT06288230 | Recruiting Phase 1

• 1 other identifier: LT01-101
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Spinal Muscular Atrophy

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• Change from baseline in developmental gross motor milestones achieved according to WHO criteria

  Baseline up to 24 months

• Change from baseline in CHOP-INTEND in children <24 months of age

  Baseline up to 24 months

• Change from baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) in children ≥ 24 months of age

  Baseline up to 24 months

Study Arms (3)

Stage 1: Dose A in children < 24 months of age

EXPERIMENTAL

Dose escalting study: Administration Dose A of Vesemnogene Lantuparvovec in children \< 24 months of age

Biological: vesemnogene lantuparvovec

Stage 1: Dose B in children < 24 months of age

EXPERIMENTAL

Dose escalting study: Administration Dose B of Vesemnogene Lantuparvovec in children \< 24 months of age

Biological: vesemnogene lantuparvovec

Stage 2: The selected dose in children ≥ 24 months of age

EXPERIMENTAL

Administration the selected dose of Vesemnogene Lantuparvovec in children ≥ 24 months of age

Biological: vesemnogene lantuparvovec

Interventions

vesemnogene lantuparvovec BIOLOGICAL

Exploratory study evaluating the safety and efficacy of vesemnogene lantuparvovec in patients with SMA.

Also known as: AAV-hSMN1

Stage 1: Dose A in children < 24 months of age; Stage 1: Dose B in children < 24 months of age; Stage 2: The selected dose in children ≥ 24 months of age

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Lantu Biopharma: lead

Study Sites (1)

Kunming Hope of Health Hospital

Kunming, Yunnan, 650200, China

RECRUITING

MeSH Terms

Conditions

Muscular Atrophy, Spinal

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; Neuromuscular Diseases

Central Study Contacts

Austin Gao, PhD

CONTACT

+8617724360504; clinicaltrials@lantubiopharma.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 18, 2024
• First Posted: March 1, 2024
• Study Start: October 20, 2024
• Primary Completion (Estimated): October 30, 2027
• Study Completion (Estimated): October 30, 2027
• Last Updated: July 22, 2025

Record last verified: 2025-07

Locations

CN (1)