Overview
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 patients (single-arm). The study population comprises patients with progressive forms of MS, not responsive to highly effective therapies.
Upon confirmation of study eligibility, patients will receive fludarabine and cyclophosphamide (types of chemotherapy, used here for lymphodepletion) over 1 to 3 days in preparation for receiving a single obe-cel infusion.
Patients will be checked closely in the 28 days following obe-cel treatment. After this, patients will be monitored to evaluate safety and efficacy up to 24 months.
Description
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 - Patients will be evaluated to confirm eligibility for lymphodepletion, and thereafter receive lymphodepletion to enhance treatment efficacy and CD19 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.
Eligibility
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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.