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

Lantu Biopharma

Status and phase

Enrolling

Phase 2

Phase 1

Conditions

Spinal Muscular Atrophy

Treatments

Drug: vesemnogene lantuparvovec

Study type

Interventional

Funder types

Industry

Identifiers

NCT06288230

LT01-101

Details and patient eligibility

About

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

Full 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 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.

Enrollment

6 estimated patients

Sex

All

Volunteers

No Healthy Volunteers

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

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.