Last updated on May 2020

Phase 1 Study to Evaluate the Safety and Efficacy of ATA188 in Subjects With Progressive Multiple Sclerosis


Brief description of study

The purpose of this study is to evaluate the safety and tolerability of ATA188 as a monotherapy in Parts 1 and 2, to determine the recommended Part 2 dose (RP2D) of ATA188 as monotherapy in Part 1, and to evaluate the effect of ATA188 treatment on biological markers of disease activity in cerebral spinal fluid in Part 2 in participants with progressive forms of multiple sclerosis (MS) (primary progressive multiple sclerosis [PPMS] and secondary progressive multiple sclerosis [SPMS]).

Detailed Study Description

This is a multicenter, 2 part study in adult participants with progressive forms of MS (PPMS/SPMS) with an open-label, single-arm, sequential dose-escalation period (Part 1) and a double-blind, randomized, placebo-controlled dose-expansion period (Part 2) and open-label extension (OLE) period, both of which may be initiated at the sponsor's discretion, based on a review of data from the dose-escalation cohorts.

This study will evaluate the safety and efficacy of ATA188 administered by intravenous (IV) infusion. ATA188 will be selected for each participant based on matching at least 2 human leukocyte antigen (HLA) alleles shared between ATA188 and the participant including at least 1 HLA-restricting allele.

In Part 1, participants will receive 2 cycles of ATA188 and will enter 12 months follow-up period after the last dose of ATA188. Participants who have completed at least the first year of the dose-escalation period and are still active in the study may be allow to enter in "Open-label Extension" period and will receive the same RP2D dose being assigned to Part 2 participants. In "Open-label Extension" period, participants will receive 1 cycle of ATA188 treatment every 12 months (Q12M) for up to 4 years (ie, Years 2 to 5). Otherwise, end of study (EOS) visit will be conducted at 24 months after Cycle 1 Day 1.

In Part 2, participants will be randomized in 5:4 ratio to receive ATA188 at the RP2D or matching placebo. Based on a review of available data, up to 36 additional participants may be randomized 2:1 to ATA188 or placebo. Participants will receive 2 cycles of ATA188 at the RP2D or matching placebo and will be followed for at least 12 months after the first dose of study drug (ie, Year 1). In second year (Year 2), participants who received placebo in first year will receive 2 cycles of ATA188 at the RP2D assigned at randomization and participants who received ATA188 at the RP2D in first year will receive 1 cycle of ATA188 and 1 cycle of placebo. Participants who complete Year 2 will enter in OLE period to receive ATA188 Q12M for up to 3 years (ie, Years 3 to 5) at the RP2D received in the double-blind period (Year 1 and Year 2). The end of study visit will be scheduled at 5 years (60 months) after the first dose of study drug (ie, Cycle 1 Day 1).

Clinical Study Identifier: NCT03283826

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