Clinical trial

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)

Name

FBX-101-LTFU

Description

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.

Trial arms

Trial start

2024-06-01

Estimated PCD

2029-12-01

Trial end

2029-12-01

Status

Not yet recruiting

Treatment

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.

Arms:

Patients enrolled in the FBX-101-LTFU study

Size

Primary endpoint

Long Term safety as assessed by incidence of Serious Adverse Events (SAEs) and Adverse Events of Special Interest (AESIs) that are attributed to FBX-101

36 months

Eligibility criteria

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.

Protocol

{'studyType': 'OBSERVATIONAL', 'patientRegistry': False, 'designInfo': {'observationalModel': 'OTHER', 'timePerspective': 'PROSPECTIVE'}, 'bioSpec': {'retention': 'SAMPLES_WITH_DNA', 'description': 'Whole Blood, Peripheral Blood Mononuclear Cells, Plasma, Urine, Faces, Saliva.'}, 'enrollmentInfo': {'count': 25, 'type': 'ESTIMATED'}}

Updated at

2024-04-26

1 organization

1 product

1 indication

Organization

Forge Biologics

Product

FBX-101

Indication

Krabbe Disease