Phase 1 Open-label Study of AMX-883 Alone in Participants With AML and High-risk MDS and in Combination in Participants With AML
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
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- 18 Years and older
- Sex
- All sexes
- Healthy volunteers
- Not accepted
- Compensation
- Not stated
Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.
What to expect
No one can promise a trial will help you personally — that is what the research is trying to find out.
What participation involves
Not stated in the study description — ask the study team about visits and procedures.
- Compensation
- Not stated
- Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
- Participants are monitored closely by the research team throughout the study.
- Some participants may receive access to an investigational treatment, which is still being studied.
- Your participation helps researchers learn whether this approach could help future patients.
About this study
In plain language
This study tests the safety of AMX-883, how the body handles it, and whether it may help treat cancer. It tests AMX-883 alone for people with acute myeloid leukemia (AML), a blood cancer, or high-risk myelodysplastic syndrome (MDS), a bone marrow disease. It also tests AMX-883 with other cancer drugs for people with AML.
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.
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- Have AML that came back or did not respond after all available standard treatments, or high-risk MDS that came back or did not respond, with 10–19% immature blood cells in the bone marrow.
- Be able to care for yourself and be out of bed for at least half of your waking hours.
- Have enough time pass since your last treatment before starting this study.
- Have kidneys and a liver that work well enough.
- Women who could become pregnant must use highly effective birth control for at least 120 days after the last dose, or 27 weeks in the UK. Men must use barrier birth control and not donate sperm for at least 120 days after the last dose.
- For the M1B study group, have no known medical reason that prevents treatment with posaconazole, an antifungal drug.
Who can't join
- Have acute promyelocytic leukemia, chronic myelogenous leukemia in its fast-growing stage, or active leukemia in the brain or spinal cord.
- Take drugs that weaken the immune system after a stem cell transplant, or have more than mild active graft-versus-host disease within 4 weeks before treatment. This is when donor cells attack your body.
- Have another cancer that is active, getting worse, or needed treatment that reaches the whole body within the past 2 years.
- Have serious heart disease, certain low blood mineral levels, or a heart rate below 50 that causes symptoms or poor blood flow. Certain family histories of sudden heart death before age 40 or long QT syndrome, a heart rhythm problem, also rule you out, as do needed medicines that can cause a dangerous heart rhythm.
- Have major surgery within 4 weeks before treatment, have not recovered enough from surgery, or have another uncontrolled illness. A condition that prevents fasting, swallowing, taking, or absorbing medicine by mouth also rules you out.
- Have had lung inflammation or scarring that needed steroid treatment that affects the whole body.
- Have active hepatitis B or C, an uncontrolled infection, or a condition that raises the risk of severe infection.
- Outside the UK, have a detectable amount of HIV in the blood. In the UK, people with HIV are excluded for HIV levels of 50 copies/mL or more, CD4 immune cell counts below 350 cells/µL, certain AIDS-related infections in the past year, less than 12 weeks on the same HIV medicines, or expected medicine changes in the next 12 weeks.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
Inclusion Criteria: * 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 Criteria: * Diagnosis of acute promyelocytic leukaemia or chronic myelogenous leukaemia in blast crisis * Clinically active central nervous system (CNS) leukaemia * Receiving immunosuppressive therapy post HSCT * History of another malignancy that is active, progressing, or has required systemic treatment within the past 2 years * Presence of \>Grade 1 active graft versus host disease within 4 weeks prior to C1D1 * Significant cardiovascular disease * 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 * Clinically significant bradycardia (\<50 beats per minute) that is symptomatic or causes haemodynamic instability * Major surgery within 4 weeks prior to C1D1 or inadequate recovery from prior surgery * 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 * Requirement for medications with a known risk of Torsades de Pointes that cannot be discontinued prior to study treatment * Detectable human immunodeficiency virus (HIV) viral load (applicable to rest of the world) * 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) * 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
Study sites (1)
VA
- NEXT Oncology Virginia · Fairfax, VA 22031More trials in Fairfax