NCT07723703 · Amphista Therapeutics Ltd
Phase 1 Open-label Study of AMX-883 Alone in Participants With AML and High-risk MDS and in Combination in Participants With AML
(BRAMLIE)
What this study is about
The purpose of the study is to assess the safety, how the drug moves through the body, and preliminary effectiveness of AMX-883 treatment given alone in participants with acute myeloid leukaemia (AML) and high-risk myelodysplastic syndrome (MDS) and in combination with anticancer agents in participants with AML.
View original scientific 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.
Interventions
DRUG
AMX-883
AMX-883 will be administered orally.
DRUG
Posaconazole
Posaconazole tablets will be administered orally.
Primary outcome measures
Number of participants with adverse events (AEs), treatment-emergent adverse events (TEAEs), adverse events of special interests (AESIs) and serious adverse events (SAEs)
Time frame: Until 30 days after last dose (Approximately 2 years 8 months)
To determine the safety and tolerability of AMX-883 in participants with relapsed or refractory AML and high-risk MDS when administered as monotherapy and in combination with posaconazole.
Number of participants with dose limiting toxicities (DLTs)
Time frame: During Cycle 1 (each cycle will be 28 days)
To determine the maximum tolerated dose (MTD)/optimal biological dose (OBD) and the recommended dose for expansion (RDE) of AMX-883 in participants with relapsed or refractory AML and high-risk MDS when administered as monotherapy and in combination with posaconazole.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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, 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
- 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
Where
- Fairfax, Virginia
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Sep 15, 2026 · Source of record for eligibility and locations