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

Last updated: October 7, 2024
Sponsor: Lantu Biopharma
Overall Status: Active - Recruiting

Phase

1/2

Condition

Muscular Dystrophy

Myasthenia Gravis (Chronic Weakness)

Spinal Muscular Atrophy

Treatment

vesemnogene lantuparvovec

Clinical Study ID

NCT06288230
LT01-101
  • All Genders

Study Summary

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

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Diagnosis of SMA based on gene mutation analysis with bi-allelic survival motorneuron (SMN1) mutations (deletion or point mutations).

  • Patients or Parent(s)/legal guardian(s) willing and able to complete the informedconsent process and comply with study procedures and visit schedule.

Exclusion

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 pulseoximetry <95% saturation.

  • Concomitant illness and any drug that in the opinion of the investigator createsunnecessary risks for gene transfer.

  • Clinically significant abnormal laboratory values.

  • Participation in a recent SMA treatment clinical trial that in the opinion of the PIcreates unnecessary risks for gene transfer.

  • Patient with signs of aspiration based on a swallowing test and unwilling to use analternative method to oral feeding.

Study Design

Total Participants: 6
Treatment Group(s): 1
Primary Treatment: vesemnogene lantuparvovec
Phase: 1/2
Study Start date:
October 20, 2024
Estimated Completion Date:
October 30, 2027

Study 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.

Connect with a study center

  • Kunming Hope of Health Hospital

    Kunming, Yunnan 650200
    China

    Active - Recruiting

Not the study for you?

Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.