Mocravimod as Adjunctive and Maintenance Treatment in AML Patients Undergoing Allo-HCT

Study Overview
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Brief Summary
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This is a multi-center, randomized, double-blinded, placebo controlled trial.

Detailed Description
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The purpose of this study is to evaluate the efficacy and safety of mocravimod as an adjunctive and maintenance treatment in adult acute myeloid leukemia (AML) patients undergoing allogeneic hematopoietic cell transplantation (HCT).

Official Title
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Randomized, Double-blind, Placebo-controlled, Multi-center Phase III Study to Evaluate the Efficacy and Safety of Mocravimod as Adjunctive and Maintenance Treatment in Adult AML Patients Undergoing Allogeneic HCT

Conditions
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Adult Acute Myeloid Leukemia

Intervention / Treatment
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* Drug: mocravimod


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of AML according to the WHO 2022 classification (excluding acute promyelocytic leukemia) and European Leukemia Net (ELN) high risk AML in CR1, intermediate risk AML in CR1, or AML of any risk in CR2. Planned allogeneic HCT from fully matched sibling donor or unrelated donor with no more than 1 antigen mismatch using PBSC graft Planned use of myeloablative conditioning regimen that is expected to result in donor chimerism of > 90% Planned use of CsA-based or TAC-based GvHD prophylaxis age ≥ 18 years and ≤ 75 years Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1 Exclusion Criteria: Use of anti-thymocyte globulin (ATG), alemtuzumab, sirolimus, abatacept for GvHD prophylaxis Diagnosis of macular edema during screening Cardiac/pulmonary/hepatic/renal dysfunction Hepatic dysfunction as defined by aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) > 2.5 x upper limit of normal (ULN); or total bilirubin > 1.5 mg/dL Renal dysfunction with estimated creatinine clearance < 60 mL/min by the Cockcroft-Gault formula

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Interventional Model Description: prospective, multicenter, randomized, double-blind, placebo-controlled, and 3-arm parallel group study
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 3mg mocravimod arm<br>3 mg of mocravimod orally once per day for 12 months | Drug: mocravimod<br>* S1PR modulator<br>|
| Experimental: 1mg mocravimod arm<br>1 mg of mocravimod orally once per day for 12 months | Drug: mocravimod<br>* S1PR modulator<br>|
| Placebo Comparator: Placebo arm<br>placebo orally once per day for 12 months | Drug: mocravimod<br>* S1PR modulator<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Relapse-free survival (RFS) | To compare the efficacy of mocravimod to that of placebo | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall survival (OS) | To compare mocravimod's effect on overall survival (OS) to that of placebo | 24 months |