Obe-cel in Refractory Progressive Forms of Multiple Sclerosis
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Obecabtagene autoleucel (obe-cel).
- Who it may be relevant to
- Registry conditions: Progressive Multiple Sclerosis. Basic parameters: 18 years — 60 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Spain, United Kingdom
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
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
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 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.
Detailed 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 - 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.
Interventions
- Biological Obecabtagene autoleucel (obe-cel)
Obecabtagene autoleucel (obe-cel) given as a single infusion.
Primary outcome measures
- Percentage of participants receiving obe-cel who experience dose-limiting toxicities (DLTs)) [Time frame: Up to Day 28]
- To evaluate the safety of obe-cel in participants with refractory forms of progressive multiple sclerosis (PMS) [Time frame: Up to end of study]
Secondary outcome measures (4)
- 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 [Time frame: 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). [Time frame: 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) [Time frame: 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) [Time frame: Up to Month 24]
Eligibility criteria
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United Kingdom · 4 centers
- Addenbrooke's Hospital — Cambridge
- Western General Hospital Edinburgh - PPDSE Edinburgh — Edinburgh
- The National Hospital for Neurology & Neurosurgery — London
- Royal Hallamshire Hospital — Sheffield
Spain · 2 centers
- Hospital Universitario Vall d'Hebron - PPDS — Barcelona
- Hospital Universitari i Politecnic La Fe de Valencia — Valencia
United States · 1 center
- Stanford University — Redwood City
Identifiers
NCT: NCT07139743 · AUTO1-MS1 · 2024-519552-93-00 · 1011413