NCT06308718

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

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: Terminated
• Phase: —
• Study type: Observational
• Enrollment: 2 (actual)
• Sponsor: Forge Biologics, Inc · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• Krabbe Disease

Interventions

Type	Name	Description
BIOLOGICAL	FBX-101	A replication-deficient adeno-associated virus gene transfer vector expressing the human galactocerebrosidase (hGALC) cDNA will be delivered one-time through a venous catheter inserted into a peripheral limb vein.

Timeline

• Start date: 2024-08-06
• Primary completion: 2025-04-10
• Completion: 2025-04-11
• First posted: 2024-03-13
• Last updated: 2025-09-05

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study