Gene therapy for spinal muscular atrophy

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

This study is testing a new gene therapy to see if it can improve symptoms and health for people with spinal muscular atrophy.

Quick facts

• Phase: Phase1; Phase2
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sex: All
• Sponsor: Lantu Biopharma (Industry-sponsored)
• Locations: 1 site (Kunming, Yunnan)
• Trial ID: NCT06288230 on ClinicalTrials.gov

What this trial studies

This interventional study evaluates the safety and efficacy of the gene therapy product vesemnogene lantuparvovec in patients diagnosed with spinal muscular atrophy (SMA). Participants will undergo a screening period followed by a treatment day and a 24-month observation period with regular follow-up visits. The study aims to assess the impact of the therapy on SMA symptoms and overall patient health. Patients will be monitored closely for any adverse effects and treatment outcomes throughout the study duration.

Who should consider this trial

Good fit: Ideal candidates are patients diagnosed with spinal muscular atrophy who have bi-allelic survival motor neuron (SMN1) mutations.

Not a fit: Patients with active viral infections, those requiring invasive ventilatory support, or with significant concurrent illnesses may not benefit from this study.

Why it matters

Potential benefit: If successful, this gene therapy could significantly improve the quality of life and functional abilities of patients with spinal muscular atrophy.

How similar studies have performed: Other studies involving gene therapy for spinal muscular atrophy have shown promising results, indicating potential for success in this approach.

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.

Where this trial is running

Kunming, Yunnan

• Kunming Hope of Health Hospital — Kunming, Yunnan, China (Recruiting)

Study contacts

• Study coordinator: Austin Gao, PhD

Conditions Spinal Muscular Atrophy