Clinical Trial 23226

• Overview

  Study Title:

  A Prospective Randomized, Double-Blind, Placebo-Controlled, Multi-Center Phase III Study 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)

  Objective:

  Primary: To compare the efficacy of mocravimod to that of placebo. Secondary: To compare mocravimod s effect on OS to that of placebo. To assess the efficacy of the second dose of mocravimod in comparison to placebo, if applicable. To assess the sustained efficacy of mocravimod in comparison to placebo. To assess mocravimod s effect on relapse, as compared to placebo. To assess mocravimod s effect on the occurrence of non-relapse mortality in comparison to placebo. To compare the effect of the second dose mocravimod on OS, if applicable. To assess mocravimod s effect on the occurrence of GvHD in comparison to placebo. To assess the safety and tolerability of mocravimod. To assess quality of life (QoL).

• Treatments

  Therapies:

  Bone Marrow Transplant; Immunomodulators; Therapy (NOS)

  Medications:

  Mocravimod (); Placebo ()

• Inclusion Criteria

  Key Inclusion Criteria:
  • Subjects with a diagnosis of AML (excluding acute promyelocytic leukemia) according to the World Health Organization (WHO) 2022 classification of AML and related precursor neoplasms, including AML with myelodysplasia-related gene mutations.
  • Subjects with European LeukemiaNet (ELN) high-risk or intermediate-risk AML in CR1, or AML of any risk in CR2. (Complete remission with incomplete count recovery [CRi] is also allowable).
  • Subjects planned to undergo allogeneic HCT.
  • Life expectancy >= 6 months at screening.
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.
  • Male or female, age >= 18 years and > Body weight of >= 50 kg.
  • Able and willing to provide written informed consent and comply with the trial protocol and procedures.
  • For persons of childbearing potential who are sexually active: willingness to use highly effective contraception methods (with a failure rate of > Sexually active males (especially who have sexual contact with a person of childbearing potential) must use a condom during intercourse from the Screening visit until at least 6 months after the last dose of IMP and should not father a child in this period. A condom is required to be used also by vasectomized men in order to prevent delivery of the IMP via seminal fluid. Male subjects must refrain from donating fresh unwashed semen.
  • Affiliation to a national health insurance scheme (only applicable if required by local regulations).
  • Other inclusion criteria may apply.

• Exclusion Criteria

  Key Exclusion Criteria:
  • Planned use of anti-thymocyte globulin (ATG), alemtuzumab, abatacept, for GvHD prophylaxis.
  • Planned use of serotherapy during conditioning, including ATG and alemtuzumab.
  • Planned ex vivo major graft manipulation, including T-cell depletion or CD34+ selection.
  • Subjects having received prior allogeneic HCT or recipients of a solid organ transplant.
  • Immunosuppressive drugs for concomitant disease. Subjects must be able to be off prednisone (> 10 mg/day) or other immunosuppressive medications for at least 3 days prior to the start of treatment of the study. Physiologic replacement dosing of hydrocortisone is permissible.
  • Major surgery within 4 weeks prior to randomization or a major wound that has not fully healed.
  • Require any of the following treatments for cardiac dysfunction without option of treatment pause at the start of IMP treatment: Treatment with medication that impairs cardiac conduction (e.g. beta blockers, verapamil-type and diltiazem-type calcium channel blockers, or cardiac glycosides), or treatment with quinidine.
  • Subjects with acute promyelocytic leukemia.
  • Diagnosis of any previous or concomitant malignancy, except subjects diagnosed with localized basal cell carcinoma of the skin or in situ cervical cancer, or subjects who have completed treatment (chemotherapy and/or surgery and/or radiotherapy) with curative intent for the malignancy at least 3 years prior to enrollment.
  • Blast crisis of chronic myeloid leukemia.
  • Concurrent severe and/or uncontrolled medical condition.
  • Cardiac dysfunction.
  • Pulmonary dysfunction as defined by oxygen saturation > Significant liver disease or liver injury or known history of alcohol abuse, chronic liver or biliary disease. 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 x ULN.
  • Renal dysfunction with creatinine clearance > History of stroke or intracranial hemorrhage within 1 year prior to screening.
  • Active clinically significant infection (viral, bacterial, or fungal) that requires ongoing antimicrobial therapy and in the judgment of the investigator represents a risk to proceeding with HCT.
  • History of human immunodeficiency virus (HIV) or active infection with hepatitis B virus (HBV) or hepatitis C virus (HCV) defined as a positive HIV antibody, hepatitis B surface antigen (HBsAg) or hepatitis C antigen.
  • Subjects who are breastfeeding or have positive pregnancy test (serum pregnancy test is mandatory at screening and prior to the IMP administration on Cycle 1 Day 1 for women of childbearing potential).
  • Known allergy to any of the components of mocravimod (e.g. excipient) and any other medications that are not study related but will be used according to local SoC, such as conditioning regimen agents, GvHD prophylaxis, and concomitant medications and therapies.
  • Diagnosis of macular edema during screening. Subjects with a history of macular edema will be allowed to enter the study provided they do not have macular edema at the ophthalmic examination at screening.
  • Participation in another interventional clinical trial within 4 weeks prior to randomization or participation in a concomitant interventional clinical trial.
  • Other Exclusion criteria may apply.

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