An observational study in Krabbe Disease, sponsored by Duke University. Terminated at 1 site in United States. Per ClinicalTrials.gov, last updated 2026-11-04.
Sponsored by Duke University · Observational
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.
31 studies on the registry are indexed under Leukodystrophy, Globoid Cell; 8 are open to participants now.
This study's enrollment of 2 is below the median of 55 across 14 observational studies indexed under Leukodystrophy, Globoid Cell.
Browse Leukodystrophy, Globoid Cell studies →Duke University is the lead sponsor of 2,063 studies on the registry; 273 are open to participants now.
Of its 204 completed or terminated interventional studies of FDA-regulated products, 167 (82%) have results posted.
Counted across the registry records on this site, refreshed daily.
This is an observational long-term follow-up (LTFU) study of participants from prior interventional trials involving the administration of FBX-101.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
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.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
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.
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.
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
Plan to share: No
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is terminated, as verified in Aug 2025. You cannot join it, but the record below documents what was studied.
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