AMX-883 for acute myeloid leukaemia and high-risk myelodysplastic syndrome

Trial details

Who runs it

Amphista Therapeutics Ltd

Trial code

NCT07723703

Record updated

2 November 2026

Original description

The purpose of the study is to assess the safety, pharmacokinetics, and preliminary efficacy of AMX-883 monotherapy in participants with acute myeloid leukaemia (AML) and high-risk myelodysplastic syndrome (MDS) and in combination with anticancer agents in participants with AML.Read full original descriptionShow less

A Phase 1, Open-Label, Multi-Centre Study to Assess the Safety, Pharmacokinetics, and Preliminary Efficacy of AMX-883 Monotherapy in Participants With Acute Myeloid Leukaemia and High-Risk Myelodysplastic Syndrome and in Combination With Anticancer Agents in Participants With Acute Myeloid Leukaemia

The purpose of the study is to assess the safety, pharmacokinetics, and preliminary efficacy of AMX-883 monotherapy in participants with acute myeloid leukaemia (AML) and high-risk myelodysplastic syndrome (MDS) and in combination with anticancer agents in participants with AML.

Inclusion: Participants with relapsed or refractory AML who have failed all available standard therapies or relapsed or refractory high-risk MDS with BM blasts 10-19%; Eastern Cooperative Oncology Group (ECOG) performance status of 0-2; Adequate washout from prior therapies; Adequate kidney and liver function; Female participants of childbearing potential must use highly effective contraception for at least 120 days, or 27 weeks (applicable to the United Kingdom [UK] only), after last dose, and male participants must agree to use barrier contraception and avoid sperm donation for at least 120 days after last dose; If enrolled in M1B: Participant must have no documented contraindication to treatment with posaconazole before start of treatment.

Exclusion: Receiving immunosuppressive therapy post HSCT; Presence of >Grade 1 active graft versus host disease within 4 weeks prior to C1D1; Major surgery within 4 weeks prior to C1D1 or inadequate recovery from prior surgery; Requirement for medications with a known risk of Torsades de Pointes that cannot be discontinued prior to study treatment; History of another malignancy that is active, progressing, or has required systemic treatment within the past 2 years; Significant cardiovascular disease; Uncontrolled intercurrent illness; Inability to fast, swallow, ingest, or absorb oral medication due to a pre-existing condition; History of interstitial lung disease or pneumonitis requiring systemic corticosteroid treatment; Detectable human immunodeficiency virus (HIV) viral load (applicable to rest of the world); Known serologic status reflecting active hepatitis B or C infection; Active uncontrolled systemic fungal, bacterial, viral, or other infection or a condition predisposing to severe infection; Family history of sudden cardiac death before 40 years of age or a family history of long QT syndrome; Clinically significant electrolyte imbalances (e.g., hypokalaemia, hypomagnesaemia, hypocalcaemia) that may contribute to QT interval prolongation; Known history of human immunodeficiency virus (HIV)-1/2 antibodies with an HIV ribonucleic acid (RNA) viral load ≥50 copies/mL, cluster of differentiation (CD)4+ lymphocyte count <350 cells/µL, a history of an acquired immunodeficiency syndrome-defining opportunistic infection within the past 12 months, on the same anti-HIV medications for <12 weeks or further changes in the number or type of antiretroviral drugs in the regimen could be anticipated in the next 12 weeks (applicable to UK only); Diagnosis of acute promyelocytic leukaemia or chronic myelogenous leukaemia in blast crisis; Clinically active central nervous system (CNS) leukaemia; Clinically significant bradycardia (<50 beats per minute) that is symptomatic or causes haemodynamic instability.

ClinicalTrials.gov NCT07723703