An Open Label Study of Gene Therapy Product in Spinal Muscular Atrophy Patients

Spinal Muscular Atrophy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06288230
• Other study ID #: LT01-101
• Status: Not yet recruiting
• Phase: Phase 1/Phase 2
• Start date: March 1, 2024
• Est. completion date: March 1, 2027

Study information

• Verified date: February 2024
• Source: Lantu Biopharma
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 6
• Est. completion date: March 1, 2027
• Est. primary completion date: March 1, 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Atrophy
• Muscular Atrophy
• Muscular Atrophy, Spinal
• Spinal Muscular Atrophy

Intervention

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

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Lantu Biopharma

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Change from baseline in hours of daily ventilation support and motor function	For patients with spinal muscular atrophy (SMA) Type 1 or non-sitters/sitters, the percentage of participants who (a) reduce the number of hours of non-invasive ventilation support required per day, (b) achieve the ability to stand without support, (c) achieve the ability to walk without assistance.	Baseline up to 24 months
Secondary	Change from baseline in ambulatory function	For patients with SMA Type 2/3 or walkers, the percentage of participants who are able to preserve ambulatory function.	Baseline up to 24 months
Secondary	Change from baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE)	The HFMSE contains 33 items rated from 0 (unable to perform) to 2 (performs without modification/adaptation/compensation). Total scores range from 0-66. Higher scores indicate higher levels of motor ability.	Baseline up to 24 months