Status:
ACTIVE_NOT_RECRUITING
Gene Transfer Clinical Trial for Krabbe Disease
Lead Sponsor:
Forge Biologics, Inc
Conditions:
Krabbe Disease
Eligibility:
All Genders
1-12 years
Phase:
PHASE1
PHASE2
Brief Summary
This is a nonblinded, non-randomized dose escalation study of intravenous AAVrh10 after hematopoietic stem cell transplantation (HSCT) in which subjects will receive standard of care hematopoietic cel...
Eligibility Criteria
Inclusion
- Diagnosis of infantile Krabbe disease, characterized by the following criteria outlined below:
- Galactocerebrosidase (GALC) activity levels in leukocytes compatible with the diagnosis of Krabbe disease; AND AT LEAST ONE OF THE FOLLOWING:
- Elevated psychosine levels predictive of infantile disease onset by DBS; OR
- Imaging or neurophysiological findings consistent with Krabbe disease (CSF, MRI, NCV, ABR); OR
- Two GALC mutations predictive to result in infantile onset phenotype.
- Age at the time of screening: 1 day to 12 months
- Participant has been deemed eligible for treatment with HSCT (standard of care) and a fully myeloablative reduced intensity/toxicity conditioning regimen (RIC/RTC) is/has been used
- Participant's parents or legal guardian consents to participate in the study and provides informed consent according to IRB guidelines prior to any study procedures being performed
- Parent(s) and/or legal guardian able to comply with the clinical protocol
- Participant must have adequate organ function at time of screening as measured by:
- Creatinine ≤ 1.5x upper limit of age appropriate normal and creatinine clearance ≥ 60 mL/min/1.73 m2
- Hepatic transaminases (ALT/AST) ≤ 2x age related upper limit of normal
- Ejection fraction of \> 50% by echocardiogram or other appropriate study without evidence of pulmonary hypertension
- Pulmonary evaluation testing demonstrating resting pulse oximeter \> 95% on room air
- Coagulation tests within 110% of normal ranges for age. (PT/INR and PTT)
Exclusion
- History of prior treatment with a gene therapy product
- Presence of major congenital anomaly or any other condition that affects neurodevelopmental function
- Presence of any neurocognitive deficit or brain damage not attributable to Krabbe disease
- Active aspiration
- Signs of active infection or disease from cytomegalovirus, adenovirus or other viruses
- HIV positive
- Uncontrolled and progressive bacterial or fungal infection
- Presence of any contraindication for MRI
- Use of any investigational product prior to study enrollment or current enrollment in another study that involves clinical interventions
- Any other medical condition, serious intercurrent illness, or extenuating circumstance that, in the opinion of the PI, would preclude participation in the study
- Ongoing veno-occlusive disease (VOD) as determined by liver ultrasound (moderate ascites and static or retrograde portal vein flow) the day before FBX-101 infusion.
Key Trial Info
Start Date :
November 5 2021
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
November 1 2026
Estimated Enrollment :
6 Patients enrolled
Trial Details
Trial ID
NCT04693598
Start Date
November 5 2021
End Date
November 1 2026
Last Update
January 30 2025
Active Locations (1)
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1
University of Michigan Hospitals - Michigan Medicine
Ann Arbor, Michigan, United States, 48109