HMA-Venetoclax Induction in Newly Diagnosed AML (NCT07748455) | Clinical Trial Compass
Not Yet RecruitingPhase 2
HMA-Venetoclax Induction in Newly Diagnosed AML
Pakistan60 participantsStarted 2026-08-01
Plain-language summary
Acute myeloid leukaemia (AML) is a clonal haematological malignancy characterised by arrested myeloid differentiation and uncontrolled proliferation of blast cells in the bone marrow and peripheral blood. It is the most common acute leukaemia in adults and carries a high disease-related mortality. Globally, the median age of diagnosis is approximately 68 years; however, a significant proportion of patients are diagnosed below the age of 50 years, constituting the 'young fit' population for whom aggressive curative-intent therapy is most appropriate.
The epidemiology of AML in South Asia and Pakistan remains incompletely characterised. Published single-centre and multi-centre data from Pakistan suggest that AML represents a major proportion of haematological malignancies presenting to tertiary centres. AFBMTC, as the largest haematology and transplant centre in Pakistan, has accumulated over 2,000 HSCT procedures since 2001 and has established the necessary infrastructure for prospective clinical research in this disease.
Cytogenetic and molecular classification of AML profoundly influences prognosis and treatment decisions. The European LeukemiaNet (ELN) 2022 risk stratification framework categorises AML into Favourable, Intermediate, and Adverse risk groups based on chromosomal abnormalities and somatic mutations including NPM1, FLT3-ITD, IDH1/2, RUNX1, ASXL1, TP53, and others. This framework underpins post-remission pathway assignment in the current protocol.
Who can participate
Age range
18 Years – 50 Years
Sex
ALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Age 18-50 years (inclusive) at the time of enrolment
. Confirmed diagnosis of AML (non-APL) per WHO 2022 Classification: ≥20% blasts in bone marrow or peripheral blood, or documented leukaemia-defining cytogenetic abnormality regardless of blast count (e.g. t(8;21), inv(16), t(15;17) is excluded as APL). Diagnosis must be confirmed by bone marrow aspirate and/or biopsy with morphology, flow cytometry, and cytogenetics.
. Newly diagnosed AML: no prior cytotoxic therapy for AML (excluding hydroxyurea for blast cytoreduction, which is permitted for ≤7 days prior to enrolment)
. ECOG Performance Status 0-2
. Adequate end-organ function at screening (within 7 days of first study drug administration):
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1This trial is testing azacitidine combined with venetoclax as an induction therapy for newly diagnosed AML — is this combination something my doctor would consider appropriate for my specific type of AML, given my mutation profile and overall health?
2Since this is a Phase 2 trial, the main goal is to measure how many patients go into remission — what do we know so far about the safety and side effects of this azacitidine-venetoclax combination from earlier research, and how does that compare to standard induction chemotherapy options for me?
3The trial isn't recruiting yet — do you know roughly when it might open, and should I start a standard treatment now rather than waiting to see if I might be eligible to discuss this study with you later?
4The primary outcome being measured is remission rate — if the treatment doesn't lead to remission, what would the next steps look like for me, and does enrolling in this trial affect my ability to pursue other treatments afterward?
5How does the intensity and logistics of this azacitidine plus venetoclax regimen compare to standard AML induction therapy in terms of hospital stays, infusion schedules, and what my day-to-day life might look like during treatment?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Remission rate after Aza-Ven Therapy
Timeframe: At the end of each cycle from Day 28-35 of Cycle (duration of each cycle is 28 days)
Trial details
NCT IDNCT07748455
SponsorPakistan Blood and Marrow Transplant (PBMT) Group
. ALT and AST ≤3 × ULN (≤5 × ULN if attributed to hepatic leukaemic infiltration)
. Total bilirubin ≤1.5 × ULN (≤3 × ULN for Gilbert syndrome or hepatic leukaemic infiltration)
Exclusion criteria
0. Acute promyelocytic leukaemia (APL) \[t(15;17); PML-RARA\]: patients with APL must be referred for ATRA-based therapy as per institutional standard
1. AML secondary to myeloproliferative neoplasm (MPN) in blast phase, where prior MPN was treated with ruxolitinib within 8 weeks of enrolment (due to drug interaction potential with venetoclax)
2. Prior exposure to a BCL-2 inhibitor (venetoclax, navitoclax, or other) at any time
3. Prior exposure to HMA therapy (azacitidine or decitabine) for a pre-existing MDS or MPN within 6 months of AML diagnosis
4. Active, uncontrolled systemic infection at the time of enrolment that in the investigator's judgement would preclude initiation of cytotoxic therapy (note: controlled infection with appropriate antimicrobial therapy is not an exclusion)
5. Known active hepatitis B virus (HBV) infection (HBsAg positive) without established antiviral prophylaxis; or active hepatitis C virus (HCV) infection with detectable viral load
6. Known HIV infection with CD4 count \<350 cells/μL or detectable viral load (HIV-positive patients with well-controlled disease on antiretroviral therapy may be enrolled after discussion with the Principal Investigator and Infectious Disease consultant)