This Research study is being done to characterize the safety, tolerability, and
preliminary antitumor activity of the NEXI-001 T cell product (a new experimental
therapy), which contains populations of CD8+ T cells targeting multiple leukemia
associated antigen peptides in patients with Acute Myeloid Leukemia (AML) or
Myelodysplastic Syndrome (MDS) who have relapsed disease after an allogeneic
hematopoietic cell transplant (HCT).
The study will enroll AML or MDS patients who have either Minimal Residual Disease (MRD)
or relapsed disease after a human leukocyte antigen (HLA)-matched allogeneic HCT.
Patients who have had an HLA-mismatched or haploidentical allogeneic HCT will not be
eligible to participate in this study. Eligible patients for this study must also have ≥
50% T-cell chimerism from the original donor at the time study entry.
The enrolled patients will undergo bridging therapy for the purposes of disease control
while the NEXI-001 T cell product is being manufactured. Choice of bridging therapy
administered will be per the Investigator's discretion, but is limited to acceptable
agents as specified in the protocol. Bridging therapy will be administered prior to
lymphodepleting (LD) therapy, with the last dose of the bridging therapy administered ≥
14 days prior to initiation of LD therapy. Within 72 hours after completing LD therapy,
patients will receive a single IV infusion of the NEXI-001 T cell product.
Additional locations may be listed on ClinicalTrials.gov for NCT04284228.
See trial information on ClinicalTrials.gov for a list of participating sites.
The NEXI-001 is an adoptive cellular therapy product which contains populations of
antigen-specific CD8+ T cells. The antigen-specific CD8+ T cells in the NEXI-001 T cell
product are derived from Peripheral Blood Mononuclear Cells (PBMC) obtained from the
original stem cell donor. During the manufacturing process, these cells are primed and
expanded ex vivo using nano-size artificial Antigen Presenting Cells (aAPC) loaded with
five leukemia associated antigen peptides in combination with a proprietary T cell
enrichment and expansion process.
The NEXI-001 T cell product is restricted to patients that are HLA-A2.01 allele positive
for this study.
There are two parts to this study, a Safety Evaluation Phase and a Dose Expansion Phase.
The Safety Evaluation Phase will determine the safety and tolerability of a single dose
of NEXI-001 T cell product, and will consist of Dose Escalation at two dose levels - each
with cohorts of three patients.
When all three patients at Dose Level 1 have dosed and cleared the DLT period, three
additional patients will be enrolled at Dose Level 2. When three patients have cleared
the DLT period at the highest dose level, that dose will be advanced to the Dose
Expansion Phase. The Dose Expansion Phase will enroll up to 16 additional patients to
further define the safety and evaluate the initial anti-tumor efficacy of the NEXI- 001 T
cell product at the dose established from the Safety Evaluation Phase.
All patients will enter a Post-Treatment Follow-Up period after infusion of the NEXI- 001
T cell product. During this phase, all patients will be monitored for AEs and followed
for anti-leukemia response until the end of study visit is complete (up to one year).
Additional assessments for safety, disease status, and other secondary and exploratory
endpoints will also be monitored during the follow-up period.
All patients will be followed for overall survival (OS) from time of disease progression
until the last visit of the last patient. During this time, patients will be followed via
telephone or other electronic contact at 12 week intervals for monitoring of OS.
Lead OrganizationNexImmune Inc.
