临床试验/NCT04655911

NCT04655911终止不适用

A Long-term Follow-up Study of Patients With MPS IIIB From Gene Therapy Clinical Trials Involving the Administration of ABO-101 (rAAV9.CMV.hNAGLU)

Abeona Therapeutics, Inc3 个研究点分布在 3 个国家目标入组 1 人开始时间: 2020年10月28日最近更新: 2022年5月31日

适应症

试验速览

阶段: 不适用
状态: 终止
发起方: Abeona Therapeutics, Inc
入组人数: 1
试验地点: 3
主要终点: Adverse Events

研究概览研究设计入排标准结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

This is a multicenter, non-interventional, long-term follow-up (LTFU) study in participants who have been treated with ABO-101 in a prior trial. Eligible participants will undergo clinical evaluations at prespecified intervals for 3 years from the last visit in the prior clinical trial (up to 5 years post-treatment).

详细描述

This is a multicenter, noninterventional, long-term follow-up study of patients with (Mucopolysaccharidosis IIIB) MPS IIIB who have completed a prior clinical trial involving the administration of ABO-101. This study is designed to provide LTFU in accordance with the FDA and European Medicines Agency (EMA) guidelines for patients treated with gene therapy products. The duration of the current study is 3 years for a total of up to 5 years post-treatment for these participants who rollover from a prior clinical trial of ABO-101. Participants will have a maximum of 5 annual scheduled visits with assessments as specified in the schedule of assessments.

研究设计

研究类型: Observational
观察模型: Cohort
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 ABO-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

排除标准

•Planned or current participation in another clinical trial that may confound the safety and efficacy evaluation of ABO-101 during the duration of this study
•Any other situation that precludes the participant from undergoing procedures required in this study

结局指标

主要结局

Adverse Events

时间窗: 60 months

Long-term product safety as defined by the incidence, type, and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)

次要结局

Age Equivalent Compared to Natural History Study Data(AII study visits (30, 36, 42, 48, and 60 Months))
Parenting Stress lndex, 4th Edition (PSl-4)(AII study visits (30, 36, 42, 48, and 60 Months))
Developmental Quotient Compared to Natural History Study Data calculated by the Mullen Scales of Early Learning or the Kaufman Assessment Battery for Children(AII study visits (30, 36, 42, 48, and 60 Months))
Pediatric Quality of Life lnventory (PedsQL ™)(AII study visits (30, 36, 42, 48, and 60 Months))
Cognitive Age Equivalent Compared to Natural History Study Data(AII study visits (30, 36, 42, 48, and 60 Months))
Developmental Quotient Compared to Natural History Study Data calculated using the Bayley Scales of lnfant and Toddler Development or the Kaufman Assessment Battery for Children(AII study visits (30, 36, 42, 48, and 60 Months))
Adaptive Age Equivalent Compared to Natural History Study Data(AII study visits (30, 36, 42, 48, and 60 Months))
Parent Symptom Scale Questionnaire(AII study visits (30, 36, 42, 48, and 60 Months))
Parental Global lmpression Scale(AII study visits (30, 36, 42, 48, and 60 Months))
T-cell responses against the AAV9 capsid(60 Months)
Antibody formation (humoral) against the AAV9 capsid(60 Months)
Viral Load(AII study visits (30, 36, 42, 48, and 60 Months))
Clinical Global lmpression lmprovement Scale(AII study visits (30, 36, 42, 48, and 60 Months))
T-cell responses against the α-N- acetylglucosaminidase (NAGLU) transgene product(60 Months)
Antibody formation (humoral) against the α-N- acetylglucosaminidase (NAGLU) transgene product(60 Months)

研究者

发起方

Abeona Therapeutics, Inc

申办方类型

Industry

责任方

Sponsor

研究点 (3)

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