Krabbe Disease
Conditions
Keywords
Lysosomal Storage Disorder, Globoid Cell Leukodystrophy, Leukodystrophy, GALC
Brief summary
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.
Detailed 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.
Interventions
BIOLOGICALFBX-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.
Sponsors
Forge Biologics, Inc
Study design
Observational model
OTHER
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Efficacy as assessed by change of gross motor function measured longitudinally by Bruininks-Oseretsky Test of Motor Proficiency (BOT-2) | 36 months | The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome. |
| Efficacy as assessed by change of gross motor function measured longitudinally by Gross Motor Function Measure 88 (GMFM-88) | 36 months | The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome. |
| Efficacy as assessed by change of fine motor function measured longitudinally by Mullen Scales of Early Learning (MSEL) | 36 months | The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome. |
| Efficacy as assessed by change of fine motor function measured longitudinally by Beery VMI Sixth Edition (VMI) | 36 months | The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome. |
| Efficacy as assessed by change of fine motor function measured longitudinally by Bayley Scales of Infant Development (Bayley-III) | 36 months | The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome. |
| Efficacy as assessed by change of fine motor function measured longitudinally by Vineland Adaptive Behavior Scale (Vineland 3) | 36 months | The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome. |
| Efficacy as assessed by change of cognitive function measured longitudinally by Mullen Scales of Early Learning (MSEL) | 36 months | The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome. |
| Efficacy as assessed by change of cognitive function measured longitudinally by the Differential Ability Scale II (DAS-II) | 36 months | The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome. |
| Efficacy as assessed by change of cognitive function measured longitudinally by Bayley Scales of Infant Development (Bayley-III) | 36 months | The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome. |
| Efficacy as assessed by change of adaptive behaviour function measured longitudinally by Vineland Adaptive Behavior Scale (Vineland 3). | 36 months | The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome. |
| Efficacy as assessed by change of language function measured longitudinally by Mullen Scales of Early Learning (MSEL). | 36 months | The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome. |
| Efficacy as assessed by change of language function measured longitudinally by Clinical Evaluation of Language Fundamentals Fifth Edition (CELF-5) | 36 months | The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome. |
| Efficacy as assessed by change of gross motor function measured longitudinally by Peabody Developmental Motor Scale (PDMS-2) | 36 months | The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome. |
| Efficacy as assessed by change of language function measured longitudinally by Vineland Adaptive Behavior Scale (Vineland 3). | 36 months | The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome. |
| 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 | 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 | 36 months | — |
| Efficacy as assessed by change of hearing function measured longitudinally by Auditory Brainstem Responses (ABRs) | 36 months | Absolute values for Waves I, III and V in milliseconds will be recorded along interpretation and waveform morphology; and also Auditory Brainstem Responses (corrected). |
| Efficacy as assessed by change of hearing function measured longitudinally by Behavior Audiometry (BAUD) | 36 months | — |
| Efficacy as assessed by change of peripheral nerve conduction velocity measured longitudinally by Nerve Conduction Velocity (NCV) assessments | 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) | 36 months | — |
| Efficacy as assessed by change of visual function as measured longitudinally by visual evoked potentials (VEP) | 36 months | — |
| Efficacy as assessed by change of visual function as measured longitudinally by visual acuity | 36 months | — |
| Efficacy as assessed by change of whole blood donor chimerism | 36 months | — |
| Efficacy as assessed by change of psychosine in whole blood and plasma | 36 months | — |
| Efficacy as assessed by change of Galactosylceramidase (GALC) levels in plasma and Cerebrospinal Fluid (CSF) | 36 months | — |
| Efficacy as assessed by change of language function measured longitudinally by Bayley Scales of Infant Development (Bayley-III) | 36 months | The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome. |
Countries
United States
Outcome results
None listed