nctId: NCT06308718 id: FBX-101-LTFU briefTitle: Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients overallStatus: NOT_YET_RECRUITING date: 2024-06 date: 2029-12 date: 2029-12 date: 2024-03-13 date: 2024-04-26 name: Forge Biologics, Inc class: INDUSTRY briefSummary: 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. conditions: Krabbe Disease studyType: OBSERVATIONAL observationalModel: OTHER timePerspective: PROSPECTIVE count: 25 type: ESTIMATED name: FBX-101 measure: 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 measure: Efficacy as assessed by change of gross motor function measured longitudinally by Peabody Developmental Motor Scale (PDMS-2) measure: Efficacy as assessed by change of gross motor function measured longitudinally by Bruininks-Oseretsky Test of Motor Proficiency (BOT-2) measure: Efficacy as assessed by change of gross motor function measured longitudinally by Gross Motor Function Measure 88 (GMFM-88) measure: Efficacy as assessed by change of fine motor function measured longitudinally by Mullen Scales of Early Learning (MSEL) measure: Efficacy as assessed by change of fine motor function measured longitudinally by Beery VMI Sixth Edition (VMI) measure: Efficacy as assessed by change of fine motor function measured longitudinally by Bayley Scales of Infant Development (Bayley-III) measure: Efficacy as assessed by change of fine motor function measured longitudinally by Vineland Adaptive Behavior Scale (Vineland 3) measure: Efficacy as assessed by change of cognitive function measured longitudinally by Mullen Scales of Early Learning (MSEL) measure: Efficacy as assessed by change of cognitive function measured longitudinally by the Differential Ability Scale II (DAS-II) measure: Efficacy as assessed by change of cognitive function measured longitudinally by Bayley Scales of Infant Development (Bayley-III) measure: Efficacy as assessed by change of adaptive behaviour function measured longitudinally by Vineland Adaptive Behavior Scale (Vineland 3). measure: Efficacy as assessed by change of language function measured longitudinally by Mullen Scales of Early Learning (MSEL). measure: Efficacy as assessed by change of language function measured longitudinally by Clinical Evaluation of Language Fundamentals Fifth Edition (CELF-5) measure: Efficacy as assessed by change of language function measured longitudinally by Bayley Scales of Infant Development (Bayley-III) measure: Efficacy as assessed by change of language function measured longitudinally by Vineland Adaptive Behavior Scale (Vineland 3). measure: 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 measure: Efficacy as assessed by change of developmental milestones such as language and motor skills as measured longitudinally by an specific Developmental Milestones questionnaire measure: Efficacy as assessed by change of hearing function measured longitudinally by Auditory Brainstem Responses (ABRs) measure: Efficacy as assessed by change of hearing function measured longitudinally by Behavior Audiometry (BAUD) measure: Efficacy as assessed by change of peripheral nerve conduction velocity measured longitudinally by Nerve Conduction Velocity (NCV) assessments measure: Efficacy as assessed by change of Fractional Anisotropy (FA) as measured longitudinally by brain Magnetic Resonance Imaging (MRI) with Diffusion Tensor Imaging (DTI) measure: Efficacy as assessed by change of visual function as measured longitudinally by visual evoked potentials (VEP) measure: Efficacy as assessed by change of visual function as measured longitudinally by visual acuity measure: Efficacy as assessed by change of whole blood donor chimerism measure: Efficacy as assessed by change of psychosine in whole blood and plasma measure: Efficacy as assessed by change of Galactosylceramidase (GALC) levels in plasma and Cerebrospinal Fluid (CSF) sex: ALL stdAges: CHILD stdAges: ADULT stdAges: OLDER_ADULT hasResults: False