A Phase 1b Extension Trial To Allow Repeat Intracerebroventricular Injections of Ex Vivo Expanded, Autologous Adipose-Derived Stem Cells (RB-ADSCs) in Subjects Previously Treated in RBI Protocol RB-ADSC-02
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 入组人数
- 9
- 主要终点
- The safety of repeated RB-ADSC treatment in study participants with AD
研究概览
简要总结
This is a Phase 1b Extension Trial to allow repeat intracerebroventricular injections of RB-ADSCs in subjects previously treated in and successfully completed RBI Protocol RB-ADSC-02. In the previous Phase 1 clinical trial, RB-ADSC-02, subjects with mild to moderate Alzheimer's disease (AD) received a single intraventricular injection of RB-ADSC. RB-ADSC will be delivered intracerebroventricularly every 2 months via the previously implanted Ommaya reservoir for up to 6 injections in total. The primary objective of safety is performed 2 months after the last dose administration at the month 12 follow-up visit. The secondary objective endpoint evaluations of efficacy are performed at the month 6 and 12 visits.
详细描述
This is a Phase 1b Extension Trial to allow repeat intracerebroventricular injections of RB-ADSCs in subjects previously treated in and successfully completed RBI Protocol RB-ADSC-02. In the previous Phase 1 clinical trial, RB-ADSC-02, subjects with mild to moderate Alzheimer's disease (AD) received a single intraventricular injection of RB-ADSC.
RB-ADSC will be delivered intracerebroventricularly every 2 months via the previously implanted Ommaya reservoir for up to 6 injections in total. Participants will be followed for 2 months after the last administration. The primary objective is safety and tolerability of repeated dosing of RB-ADSC. Adverse events (AEs) and serious adverse events (SAEs) will be assessed by the incidence and severity of dose-limiting toxicity (DLT) and other AEs, incidence and severity of cytokine response syndrome, vital sign measurements, clinical laboratory tests and physical examination. Preliminary efficacy of repeated dosing will be evaluated with clinical assessments (MMSE, FAST, ADAS-Cog), volumetric MRI (NeuroQuant), CSF biomarkers (phosphor-Tau, total-Tau,AB-42), and diagnostic imaging comparison (Amyloid-PET).
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 45 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Successful completion of the Phase 1 single dose administration study of RB-ADSC, Protocol RB-ADSC-
- •Written informed consent to participate in this Phase 1b Extension Study either provided by the participant or a legal representative
- •The participant must have a relative/caregiver who is alert for possible side effects.
- •The caregiver must separately meet the specified inclusion/
排除标准
- •for caregivers for this Phase 1b Extension Study.
- •Exclusion Criteria:
- •Participant experienced treatment related SAE or DLT in Protocol RB-ADSC-02
- •Participants who, in the opinion of the Investigator, are unsuitable for this Phase 1 Extension Study
研究组 & 干预措施
RB-ADSC low dose
Participants will receive 2x10^6 RB-ADSC infused in the previously implanted Ommaya reservoir every 2 months for up to a total of 6 doses
干预措施: RB-ADSC (Biological)
RB-ADSC medium dose
Participants will receive 5x10^6 RB-ADSC infused in the previously implanted Ommaya reservoir every 2 months for up to a total of 6 doses
干预措施: RB-ADSC (Biological)
RB-ADSC high dose
Participants will receive 10x10^6 RB-ADSC infused in the previously implanted Ommaya reservoir every 2 months for up to a total of 6 doses
干预措施: RB-ADSC (Biological)
结局指标
主要结局
The safety of repeated RB-ADSC treatment in study participants with AD
时间窗: up to 12 months
Safety will be determined by incidence, type and severity of adverse events (AE) and serious adverse events (SAE) graded according to CTCAE v5.0 and CRS revised grading system and defined by clinical relevant findings at every visit and 2 month following last dose in physical examination, vital signs and laboratory data
次要结局
- Change from Baseline in cerebrospinal fluid (CSF) biomarker pTau(Baseline, Month 6, Month 12)
- Change from Baseline in cerebrospinal fluid (CSF) biomarker Total-Tau(Baseline, Month 6, Month 12)
- Change from Baseline in cerebrospinal fluid (CSF) biomarker Ab42(Baseline, Month 6, Month 12)
- Change from Baseline in cerebrospinal fluid (CSF) biomarker ratios reported as amyloid-tau index (ATI)(Baseline, Month 6, Month 12)
- Change from Baseline in Amyloid Positron Emission Tomography (Amyloid-PET)(Baseline, Month 6, Month 12)
- Change from Baseline in MMSE(Baseline, Month 6, Month 12)
- Change from Baseline in ADAS-cog13(Baseline, Month 6, Month 12)
- Change from Baseline in FAST(Baseline, Month 6, Month 12)
- Change from Baseline in MoCA(Baseline, Month 6, Month 12)
- Change from Baseline in Volumetric MRI with Contrast(Baseline, Month 6 and Month 12)
- Change from Baseline in NeuroQuant MRI(Baseline, Month 6 and Month 12)