Clinical Trial Feasibility – Acute Myeloid Leukemia (AML)

Clinical Trial Summary

Phase 1 & II, Interventional, multi-center, randomized, single-arm, multiple ascending dose study to evaluate the safety, pharmacokinetics & pharmacodynamics in patients diagnosed with AML

The purpose of this study is to determine the safety, tolerability, dose-limiting toxicities (DLT) and maximum tolerated dose (MTD) of CXD in patients with relapsed/refractory acute myeloid leukemia (AML).

Key Inclusion Criteria:

  • Age ≥ 18 years at the time of screening.
  • Subjects must have a documented diagnosis of acute myeloid leukemia (AML) (WHO 2016)
  • Subjects non-eligible for intensive chemotherapy as first-line, second-line, or third-line treatment.
  • Written informed consent and any locally required authorization
  • Liver Function Tests: AST and ALT ≤ 3 × ULN, and serum TBL ≤ 1.5 × ULN, unless consistent with Gilbert’s syndrome for which TBL ≤ 2.5 × ULN is allowed.
  • CrCL ≥ 40 mL/min 6. Female patients of childbearing potential who are sexually active with a non sterilized male partner must use at least one highly effective method of contraception from 7 days post-screening and must agree to continue using such precautions for 90 days after the last dose of investigational product.
  • Non Sterilized male patients who are sexually active with a female partner of childbearing potential must use a male condom plus spermicide from 7 days post-screening and for 90 days after receipt of the last dose of investigational product

Key Exclusion Criteria:

  • Active human immunodeficiency virus (HIV), hepatitis B virus (HBV) or hepatitis C virus (HCV) infections at the time of screening.
  • Patients at risk of non-disease related major bleeding (eg, recent GI hemorrhage or neurosurgery, within the previous 21 days).
  • Current severe active systemic disease including active concurrent malignancy
  • Received cytotoxic chemotherapy within 21 days (or 42 days for nitrosoureas or mitomycin C) prior to the first scheduled dose of CXD.
  • Received an investigational drug within 14 days of the first scheduled dose of CXD or not recovered from associated toxicities.
  • Received any major surgery (as defined by the Investigator), radiotherapy, or immunotherapy (including immune checkpoint inhibitors and adoptive cellular therapy such as autologous or donor NK cell or T lymphocyte infusions (e.g. CAR -T cells)) within 28 days of the first scheduled dose of CXD.
  • Patients who have previously received an autologous SCT, are excluded if less than 120 days have elapsed from the time of transplant or the patient has not recovered from transplant-associated toxicities prior to the first scheduled dose of CXD.
  • History of liver cirrhosis, liver fibrosis or prior liver irradiation regardless of the time interval (not including total body irradiation administered during allogeneic SCT).
  • Failure to recover from all prior treatment-related non-hematological toxicities to ≤ Grade 1 prior to the first scheduled dose of CXD (except for alopecia and neuropathy).
  • Central nervous system (CNS) disease that is untreated, symptomatic, or requires therapy to control symptoms.

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