Study to Find a Safe and Effective Dose of SKI-G-801 in the Treatment of Patients With Acute Myeloid Leukemia (AML)

A Phase 1 Dose Escalation Trial of SKI-G-801 in Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML)

This Phase I study is designed to assess the safety, tolerability, pharmacokinetics and anti-tumor effect of increasing doses of study drug SKI-G-801 in patients with relapsed or refractory Acute Myeloid Leukemia (AML) who are unresponsive to currently available therapies. Eligible participants will receive cycles of treatment involving IV infusion of SKI-G-801 daily for 14 days followed by 14 days off. Treatment cycles will be repeated until progressive disease or unacceptable toxicity.

Inclusion Criteria:

• Willing and able to provide written informed consent for participation, prior to completing any study-related procedures.
• Diagnosis of Acute Myeloid Leukemia (AML)
• Patients must have been off previous antileukemia therapy for at least 2 weeks or 5 half-lives, whichever is longer if the immediate prior regimen included only weekly chemotherapy; or 4 weeks or 5 half-lives, whichever is longer, from any therapy with therapeutic biologics and from any type of investigational therapy. Daily hydroxyurea for up to 2 weeks to keep the absolute blast count below 50 x 10⁹/L will be allowed, but must be discontinued 24 hours prior to administration of study drug. Hydroxyurea will be permitted during the first cycle of treatment if necessary.
• At least one prior induction regimen (with or without consolidation) which may have included hematopoietic stem cell transplantation (HSCT).
• Have adequate liver function.
• Have adequate renal (kidney) function.
• Female patients must either be of non-childbearing potential, or, if of childbearing potential, have a negative urine pregnancy test at screening and agree not to try to become pregnant during the study and for 45 days after the final study drug administration. Women of childbearing potential, if heterosexually active, must agree to use 2 forms of highly effective birth control as determined by the protocol, starting at screening, throughout the study period and for 45 days after the final study drug administration.
• Female patients must agree not to breastfeed at screening, throughout the study period and for 45 days after the final study drug administration.
• Male patients with female spouse/partner of childbearing potential, must agree to use 2 forms of highly effective birth control as determined by the protocol, starting at screening, throughout the study period and for 45 days after the final study drug administration.

Exclusion Criteria:

• Patient has a diagnosis of Acute Promyelocytic Leukemia (APL) or chronic myelogenous leukemia in blast crisis.
• If patient is post allogenic transplant and requires therapy for graft vs host disease (GVHD) within 14 days prior to date of screening.
• Requires treatment with concomitant drugs that prolong QT/QTc interval.
• Recent history of cardiac ischemic disease (acute myocardial infarction within 6 months; uncontrolled angina); severe uncontrolled ventricular arrhythmia; recent transient ischemic attack or stroke within 6 months of screening; poorly controlled hypertension (systolic blood pressure >140 mm Hg or diastolic blood pressure >90 mm Hg).
• Patient has active, untreated central nervous system (CNS) disease.

Other protocol defined inclusion/exclusion criteria could apply.