A Long-term Follow-up Study of Patients With MPS IIIA From Gene Therapy Clinical Trials Involving the Administration of ABO-102 (scAAV9.U1a.hSGSH)
试验速览
- 阶段
- 3 期
- 状态
- Enrolling By Invitation
- 入组人数
- 41
- 试验地点
- 7
- 主要终点
- Number of Participants with Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (SAEs)
研究概览
简要总结
The main objective of this study is to evaluate the safety/tolerability and efficacy of UX111 (previously known as ABO-102) in participants with Mucopolysaccharidosis IIIA (MPS IIIA).
详细描述
This is a multicenter, long-term follow-up study of patients with MPS IIIA who have participated in a prior clinical trial involving the administration of UX111 (NCT02716246 and NCT04088734). No investigational product will be provided. At approved sites adjuvant immunomodulatory (IM) therapy may be administered to selected participants. The Principal Investigator and/or caregiver, in consultation with the medical monitor, will determine whether to initiate adjuvant IM therapy. Not all participants may receive IM therapy.
This study was previously posted by Abeona Therapeutics, Inc and was transferred to Ultragenyx in August 2022.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Other
- 盲法
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants that have participated in a prior clinical trial in which they received UX111
- •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 or efficacy evaluation of UX111 during this study
- •Any other situation or medical condition that precludes the participant from undergoing procedures required in this study
研究组 & 干预措施
Cohort A
Participants who have participated in a prior clinical trial involving the administration of UX111 (rebisufligene etisparvovec) and are able to comply with onsite scheduled visits and assessments. Select participants may receive adjuvant IM therapy.
干预措施: Adjuvant Immunomodulatory (IM) Therapy (Drug)
Cohort B
Participants who have participated in a prior clinical trial involving the administration of UX111 (rebisufligene etisparvovec) and who cannot participate in Cohort A. Participants will partake in a reduced number of assessments, performed either onsite or at home via a combination of telehealth and home healthcare visits.
干预措施: No Investigational Product (Other)
Cohort A
Participants who have participated in a prior clinical trial involving the administration of UX111 (rebisufligene etisparvovec) and are able to comply with onsite scheduled visits and assessments. Select participants may receive adjuvant IM therapy.
干预措施: No Investigational Product (Other)
结局指标
主要结局
Number of Participants with Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (SAEs)
时间窗: Up to Year 5
Bayley Scales of Infant and Toddler Development - Third Edition (BSITD-III) Cognitive Raw Score Over Time
时间窗: Up to Year 5
次要结局
- BSITD-III Receptive Communication Raw Score Over Time(Up to Year 5)
- BSITD-III Expressive Communication Raw Score Over Time(Up to Year 5)
- Annualized Percentage Change from Baseline in Prior Trial in Total Cortical Volume(Baseline, Up to Month 36)
- Survival(Up to Year 5)
- Percent Change From Baseline in Prior Trial in CSF HS(Baseline, Up to Month 36)
- Cerebrospinal Fluid (CSF) Heparin Sulfate (HS) (Disaccharide) Exposure(Baseline, Up to Month 36)