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

Launched by LANTU BIOPHARMA · Feb 24, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a new gene therapy called Vesemnogene Lantuparvovec for patients with Spinal Muscular Atrophy (SMA). The goal is to see how safe and effective this treatment is for people diagnosed with SMA, which is caused by specific genetic mutations. The trial is currently looking for participants of all ages who have been diagnosed with SMA through genetic testing showing two mutations in the SMN1 gene. Patients, or their caregivers, need to be willing to learn about the study and follow its procedures.

To be eligible, participants should not have any active viral infections, should not require invasive breathing support, and should not have other serious health issues that could complicate the treatment. Additionally, those who have recently participated in another SMA trial might not qualify. If someone joins this study, they can expect to receive the gene therapy and will be monitored closely by doctors to assess how well it works and if there are any side effects. This trial represents an important step in exploring new treatments for SMA, aiming to improve the lives of those affected by this condition.

Gender

ALL

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:
• • 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.