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.
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.
Study Type
OBSERVATIONAL
Enrollment
2
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.
University of Michigan Hospitals - Michigan Medicine
Ann Arbor, Michigan, United States
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
Time frame: 36 months
Efficacy as assessed by change of gross motor function measured longitudinally by Peabody Developmental Motor Scale (PDMS-2)
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome.
Time frame: 36 months
Efficacy as assessed by change of gross motor function measured longitudinally by Bruininks-Oseretsky Test of Motor Proficiency (BOT-2)
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome.
Time frame: 36 months
Efficacy as assessed by change of gross motor function measured longitudinally by Gross Motor Function Measure 88 (GMFM-88)
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome.
Time frame: 36 months
Efficacy as assessed by change of fine motor function measured longitudinally by Mullen Scales of Early Learning (MSEL)
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome.
Time frame: 36 months
Efficacy as assessed by change of fine motor function measured longitudinally by Beery VMI Sixth Edition (VMI)
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome.
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Time frame: 36 months
Efficacy as assessed by change of fine motor function measured longitudinally by Bayley Scales of Infant Development (Bayley-III)
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome.
Time frame: 36 months
Efficacy as assessed by change of fine motor function measured longitudinally by Vineland Adaptive Behavior Scale (Vineland 3)
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome.
Time frame: 36 months
Efficacy as assessed by change of cognitive function measured longitudinally by Mullen Scales of Early Learning (MSEL)
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome.
Time frame: 36 months
Efficacy as assessed by change of cognitive function measured longitudinally by the Differential Ability Scale II (DAS-II)
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome.
Time frame: 36 months
Efficacy as assessed by change of cognitive function measured longitudinally by Bayley Scales of Infant Development (Bayley-III)
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome.
Time frame: 36 months
Efficacy as assessed by change of adaptive behaviour function measured longitudinally by Vineland Adaptive Behavior Scale (Vineland 3).
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome.
Time frame: 36 months
Efficacy as assessed by change of language function measured longitudinally by Mullen Scales of Early Learning (MSEL).
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome.
Time frame: 36 months
Efficacy as assessed by change of language function measured longitudinally by Clinical Evaluation of Language Fundamentals Fifth Edition (CELF-5)
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome.
Time frame: 36 months
Efficacy as assessed by change of language function measured longitudinally by Bayley Scales of Infant Development (Bayley-III)
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome.
Time frame: 36 months
Efficacy as assessed by change of language function measured longitudinally by Vineland Adaptive Behavior Scale (Vineland 3).
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome.
Time frame: 36 months
Efficacy as assessed by change of quality of life measured longitudinally by the Pediatric Quality of Life (PedsQL) Generic Core Scales with Family Impact Module and Multidimensional Fatigue Scale
Time frame: 36 months
Efficacy as assessed by change of developmental milestones such as language and motor skills as measured longitudinally by an specific Developmental Milestones questionnaire
Time frame: 36 months
Efficacy as assessed by change of hearing function measured longitudinally by Auditory Brainstem Responses (ABRs)
Absolute values for Waves I, III and V in milliseconds will be recorded along interpretation and waveform morphology; and also Auditory Brainstem Responses (corrected).
Time frame: 36 months
Efficacy as assessed by change of hearing function measured longitudinally by Behavior Audiometry (BAUD)
Time frame: 36 months
Efficacy as assessed by change of peripheral nerve conduction velocity measured longitudinally by Nerve Conduction Velocity (NCV) assessments
Time frame: 36 months
Efficacy as assessed by change of Fractional Anisotropy (FA) as measured longitudinally by brain Magnetic Resonance Imaging (MRI) with Diffusion Tensor Imaging (DTI)
Time frame: 36 months
Efficacy as assessed by change of visual function as measured longitudinally by visual evoked potentials (VEP)
Time frame: 36 months
Efficacy as assessed by change of visual function as measured longitudinally by visual acuity
Time frame: 36 months
Efficacy as assessed by change of whole blood donor chimerism
Time frame: 36 months
Efficacy as assessed by change of psychosine in whole blood and plasma
Time frame: 36 months
Efficacy as assessed by change of Galactosylceramidase (GALC) levels in plasma and Cerebrospinal Fluid (CSF)
Time frame: 36 months