A Long-term Follow-up Study to Evaluate Safety and Efficacy of Krabbe Patients From Gene Therapy Clinical Trials Involving the Administration of FBX-101 (AAVrh.10-hGALC)
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 入组人数
- 2
- 试验地点
- 1
- 主要终点
- 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
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants that have completed a prior clinical trial involving the administration of FBX-
- •Parent(s)/legal guardian(s) of participant willing and able to complete the informed consent process and comply with study procedures and visit schedule.
排除标准
- •Planned or current participation in any other interventional clinical study that may confound the safety or efficacy evaluation of FBX-101 during this study.
研究组 & 干预措施
Patients enrolled in the FBX-101-LTFU study
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.
干预措施: FBX-101 (Biological)
结局指标
主要结局
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
时间窗: 36 months
次要结局
- Efficacy as assessed by change of fine motor function measured longitudinally by Vineland Adaptive Behavior Scale (Vineland 3)(36 months)
- Efficacy as assessed by change of fine motor function measured longitudinally by Mullen Scales of Early Learning (MSEL)(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 gross motor function measured longitudinally by Peabody Developmental Motor Scale (PDMS-2)(36 months)
- Efficacy as assessed by change of cognitive function measured longitudinally by Bayley Scales of Infant Development (Bayley-III)(36 months)
- Efficacy as assessed by change of language function measured longitudinally by Mullen Scales of Early Learning (MSEL).(36 months)
- Efficacy as assessed by change of language function measured longitudinally by Clinical Evaluation of Language Fundamentals Fifth Edition (CELF-5)(36 months)
- Efficacy as assessed by change of hearing function measured longitudinally by Auditory Brainstem Responses (ABRs)(36 months)
- Efficacy as assessed by change of gross motor function measured longitudinally by Bruininks-Oseretsky Test of Motor Proficiency (BOT-2)(36 months)
- Efficacy as assessed by change of gross motor function measured longitudinally by Gross Motor Function Measure 88 (GMFM-88)(36 months)
- Efficacy as assessed by change of fine motor function measured longitudinally by Beery VMI Sixth Edition (VMI)(36 months)
- Efficacy as assessed by change of fine motor function measured longitudinally by Bayley Scales of Infant Development (Bayley-III)(36 months)
- Efficacy as assessed by change of cognitive function measured longitudinally by Mullen Scales of Early Learning (MSEL)(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 cognitive function measured longitudinally by the Differential Ability Scale II (DAS-II)(36 months)
- Efficacy as assessed by change of adaptive behaviour function measured longitudinally by Vineland Adaptive Behavior Scale (Vineland 3).(36 months)
- Efficacy as assessed by change of language function measured longitudinally by Bayley Scales of Infant Development (Bayley-III)(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(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)
- 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 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)