Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients
- Registration Number
- NCT06308718
- Lead Sponsor
- Forge Biologics, Inc
- 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.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 25
- 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.
•Planned or current participation in any other interventional clinical study that may confound the safety or efficacy evaluation of FBX-101 during this study.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Patients enrolled in the FBX-101-LTFU study FBX-101 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.
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 hearing function measured longitudinally by Behavior Audiometry (BAUD) 36 months 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 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
Trial Locations
- Locations (1)
University of Michigan Hospitals - Michigan Medicine
🇺🇸Ann Arbor, Michigan, United States