An Adaptive, Multicentre, Phase IIa, Multi-disease Trial Investigating the Safety & Activity of a Single Infusion of Selected Mesenchymal Stromal Cells in the Treatment of Patients With Primary Sclerosing Cholangitis & Autoimmune Hepatitis
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 18
- 试验地点
- 2
- 主要终点
- Activity and Safety at the Highest Safe Dose (HSD) of ORBCEL-C in PSC patients, by measure of change in Alkaline Phosphatase (ALP)
研究概览
简要总结
MERLIN is an adaptive, single arm, multi-centre, phase IIa multi-disease clinical trial. It is designed to:
i) Determine dose safety of ORBCEL-C™ (selected Mesenchymal stromal cells derived from human umbilical cord) ii) Evaluate treatment activity through assessment of biomarkers (for patients treated at the highest safe dose only (HSD))
This trial will determine the Highest Safe Dose (HSD) that can be administered by observing for occurrence of dose limiting toxicity (DLT).
Upon completion of this trial we hope to be able to justify and conduct separate, larger scale trials using ORBCEL-C™.
详细描述
MERLIN is an adaptive, single arm, multi-centre, phase IIa multi-disease clinical trial. It is designed to:
i) Determine dose safety of ORBCEL-C™ (selected Mesenchymal stromal cells derived from human umbilical cord) ii) Evaluate treatment activity through assessment of biomarkers
This trial will determine the HSD* that can be administered by observing for occurrence of dose limiting toxicity (DLT).
* An investigated dose level is determined to be safe if we see 0 DLTs in 3 patients or ((in the instance where a cohort is expanded to 6 patients due to occurrence of a DLT in the first 3 patients treated at a cohort) < 2 DLTs in 6 patients. The HSD is the highest such dose which fulfils these criteria and will be ascertained via dose-escalation using 3+3 methodology.
Further safety and activity outcomes will be determined on patients treated at the HSD only. Upon completion of this trial we hope to be able to justify and conduct separate, larger scale trials using ORBCEL-C™.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
结局指标
主要结局
Activity and Safety at the Highest Safe Dose (HSD) of ORBCEL-C in PSC patients, by measure of change in Alkaline Phosphatase (ALP)
时间窗: Baseline to Visit 8 - Approximately 80 days
Change in Alkaline Phosphatase (ALP) after ORBCEL-C infusion - Examination of change in ALP at Day 28 from Baseline and changes over multiple time-points before and after infusion (Visit 1 to Visit 8)
Dose finding and incidence of treatment emergent adverse events (safety and tolerability) in all PSC and AIH Patients
时间窗: Visit 3 to Visit 5 -14 days
Occurrence of Dose Limiting Toxicity (DLT) over 14 day (Visit 3 to Visit 5) reporting period after ORBCEL-C infusion
Incidence of treatment emergent adverse events (safety and tolerability) for PSC and AIH patients treated at the Highest Safe Dose (HSD) only
时间窗: Visit 3 to Visit 8 - 56 days
Determine safety and tolerability by occurrence of Dose Limiting Toxicity (DLT) (Visit 3 to Visit 5 only), Serious Adverse Events (SAEs) and Adverse Events (AEs) throughout trial period (up to Visit 8)
Activity at the Highest Safe Dose (HSD) of ORBCEL-C in AIH patients, by measure of change in Alanine Aminotransferase (ALT)
时间窗: Baseline to Visit 8 - Approximately 80 days
Change in Alanine Aminotransferase (ALT) trend after ORBCEL-C infusion. Measurements of ALT will be taken at multiple time points from Visit 1 to Visit 8.
次要结局
- All patients with Primary Sclerosing Cholangitis (PSC) - Secondary Outcome Measure 3(Baseline to Visit 8 - Approximately 80 days)
- All patients with Autoimmune Hepatitis (AIH) - Secondary Outcome Measure 2(Baseline to Visit 8 - Approximately 80 days)
- All patients with Autoimmune Hepatitis (AIH) - Secondary Outcome Measure 4(Baseline to Visit 8 - Approximately 80 days)
- All patients with Autoimmune Hepatitis (AIH) - Secondary Outcome Measure 5(Baseline to Visit 8 - Approximately 80 days)
- All patients with Primary Sclerosing Cholangitis (PSC) - Secondary Outcome Measure 1(Baseline to Visit 8 - Approximately 80 days)
- All patients with Autoimmune Hepatitis (AIH) - Secondary Outcome Measure 1(Baseline to Visit 8 - Approximately 80 days)
- All patients with Autoimmune Hepatitis (AIH) - Secondary Outcome Measure 3(Baseline to Visit 8 - Approximately 80 days)
- All patients with Primary Sclerosing Cholangitis (PSC) - Secondary Outcome Measure 2(Baseline to Visit 8 - Approximately 80 days)
- All patients with Primary Sclerosing Cholangitis (PSC) - Secondary Outcome Measure 4(Baseline to Visit 8 - Approximately 80 days)