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

Forge Biologics

Status: Terminated

Conditions: Krabbe Disease

Treatments: Biological: FBX-101

Study type: Observational

Funder types: Industry

Identifiers

NCT06308718

FBX-101-LTFU

Details and patient eligibility

About

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.

Full 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.

Enrollment: 2 patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion criteria

• Participants that have completed a prior clinical trial involving the administration of FBX-101.
• Parent(s)/legal guardian(s) of participant willing and able to complete the informed consent process and comply with study procedures and visit schedule.

Exclusion criteria

•Planned or current participation in any other interventional clinical study that may confound the safety or efficacy evaluation of FBX-101 during this study.

Trial design

2 participants in 1 patient group

Patients enrolled in the FBX-101-LTFU study

Description:

The participants will be followed for 36 months after they have concluded their participation in the interventional trial. They will complete 5 scheduled visits with assessments as specified in the schedule of assessments, to collect data for safety and additional signs of efficacy for FBX-101. Those patients enrolled from any other early terminated trial, will first complete pending evaluations from that trial.

Treatment:

Biological: FBX-101