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