A Single-arm, Open-label, Phase I Study to Determine the Safety, Tolerability, and Preliminary Efficacy of Obe-cel in Participants With Refractory Progressive Forms of Multiple Sclerosis
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 18
- 试验地点
- 8
- 主要终点
- To evaluate the safety of obe-cel in participants with refractory forms of progressive multiple sclerosis (PMS)
研究概览
简要总结
The main purpose of this study is to evaluate if obe-cel is safe or causes any side effects in adults with refractory progressive MS. The study also plans to assess if obe-cel can show early signs of efficacy in MS. The trial includes only 1 group of participants (single-arm). The study population comprises participants with progressive forms of MS, not responsive to highly effective therapies.
Upon confirmation of study eligibility, participants will receive chemotherapy (used here for lymphodepletion) over 1 to 3 days in preparation for receiving a single obe-cel infusion.
Participants will be checked closely in the 28 days following obe-cel treatment. After this, participants will be monitored to evaluate safety and efficacy up to 24 months.
详细描述
This is a Phase 1 study to determine the safety, tolerability, and preliminary efficacy of obe-cel in participants with refractory progressive forms of multiple sclerosis.
The study comprises 3 periods:
Screening Period: From Day -30 to enrolment Treatment Period: From day of enrolment to End of Treatment (EOT) or Day 1 - Participants will be evaluated to confirm eligibility for lymphodepletion, and thereafter receive lymphodepletion to enhance treatment efficacy and cluster of differentiation (CD)19 chimeric antigen receptor (CAR) T cell survival.
Obe-cel Administration: If the participant's eligibility for receiving obe-cel infusion is confirmed, the participant is to receive a single obe-cel infusion on Day 1.
Post treatment Period: From Day 1 to End of Study all participants will be followed up for efficacy and safety evaluation.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 60 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Willing and able to give written informed consent for participation in the study.
- •Ability and willingness to adhere to the protocol's Schedule of Activities and other requirements.
- •Participants must be 18 to 60 years of age inclusive at the time of signing the informed consent form.
- •A female participant is eligible to participate if she is not pregnant or breastfeeding.
- •Current diagnosis of PMS.
- •Must have been treated previously with 2 disease-modifying therapies.
排除标准
- •Any medications prohibited by the protocol.
- •Highly active multiple sclerosis.
- •Diagnosis of another autoimmune central nervous system condition.
- •Active or uncontrolled fungal, bacterial, viral infection.
- •History of malignant neoplasms unless disease-free for at least 24 months.
- •History of heart, lung, kidney, liver transplant or hematopoietic stem cell transplant.
研究组 & 干预措施
Obecabtagene autoleucel (obe-cel)
干预措施: Obecabtagene autoleucel (obe-cel) (Biological)
结局指标
主要结局
To evaluate the safety of obe-cel in participants with refractory forms of progressive multiple sclerosis (PMS)
时间窗: Up to end of study
To evaluate the safety of obe-cel in participants with refractory forms of progressive multiple sclerosis (PMS)
Percentage of participants receiving obe-cel who experience dose-limiting toxicities (DLTs))
时间窗: Up to Day 28
次要结局
- To evaluate the preliminary efficacy of obe-cel in participants with refractory forms of PMS using Expanded Disability Status Scale (EDSS) - Change from baseline in EDSS(Up to Month 24)
- To evaluate the preliminary efficacy of obe-cel in participants with refractory forms of PMS using Timed 25-foot Walk Test (T25FWT).(Up to Month 24)
- To evaluate the preliminary efficacy of obe-cel in participants with refractory forms of PMS using 9-hole Peg Test (9-HPT)(Up to Month 24)
- To evaluate the preliminary efficacy of obe-cel in participants with refractory forms of PMS using Symbol Digit Modalities Test (SDMT)(Up to Month 24)