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

L

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.

Trial design

Primary purpose

Treatment

Allocation

N/A

Interventional model

Sequential Assignment

Masking

None (Open label)

6 participants in 1 patient group

vesemnogene lantuparvovec
Experimental group
Description:
One-time Intravenous administration of vesemnogene lantuparvovec.
Treatment:
Drug: vesemnogene lantuparvovec

Trial contacts and locations

1

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Central trial contact

Austin Gao, PhD

Data sourced from clinicaltrials.gov

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