Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients

Krabbe Disease Clinical Trial

Official title: A Long-term Follow-up Study to Evaluate Safety and Efficacy of Krabbe Patients From Gene Therapy Clinical Trials Involving the Administration of FBX-101 (AAVrh.10-hGALC)

Status Not yet recruiting

Clinical Trial Summary

This is an observational study that will enroll any patients with Krabbe disease that have participated in prior interventional clinical trials involving the administration of FBX-101.

Clinical Trial Description

FBX-101-LTFU is a multicenter, non-interventional, Long-Term Follow-Up (LTFU) study of participants from prior interventional trials involving the administration of FBX-101. Eligible participants will undergo clinical evaluations at prespecified intervals for at least 3 years from the last visit in the prior clinical trial (up to 5 years post-FBX-101 treatment). Overall safety and additional signs of efficacy will be collected with a series of laboratory tests, diagnostic tests, and performance surveys. Additionally, children participating in interventional trials that are terminated early will be transferred to this LTFU study and will complete any pending visits from the interventional trial before starting the clinical evaluations included in this protocol. ;

Related Conditions & MeSH terms

• Krabbe Disease
• Leukodystrophy, Globoid Cell

• NCT number: NCT06308718
• Study type: Observational
• Source: Forge Biologics, Inc
• Contact: Kelly Bossola
• Phone: +1.380.239.2052
• Email: advocacy@forgebiologics.com
• Status: Not yet recruiting
• Start date: June 2024
• Completion date: December 2029