GEN3018 for relapsed or treatment‑resistant acute myeloid leukemia and higher‑risk MDS
An Open-Label, Multicenter, First-in-Human Trial of GEN3018 in Participants With Relapsed or Refractory Acute Myeloid Leukemia or Higher-Risk Myelodysplastic Syndrome
This trial will test a new antibody drug called GEN3018 in adults whose AML or higher‑risk MDS has relapsed or not responded to prior treatments.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 78 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Genmab Industry-sponsored |
| Drugs / interventions | chemotherapy, radiation |
| Locations | 9 sites (Aarhus and 8 other locations) |
| Trial ID | NCT07384715 on ClinicalTrials.gov |
What this trial studies
GEN3018 is a novel antibody given to adults with relapsed or refractory acute myeloid leukemia (AML) or higher‑risk myelodysplastic syndrome (HR‑MDS) in a first‑in‑human, open‑label Phase 1 trial. Part 1 uses dose escalation to find a safe dose and characterize safety, pharmacokinetics, immunogenicity, and tolerability, while Part 2 refines the recommended dose(s) for further testing. Up to 78 participants across multiple centers will receive GEN3018 and be monitored with clinical exams, laboratory tests, and bone marrow sampling for preliminary anti‑tumor activity and adverse events. Collected data will guide dose selection and design of later studies.
Who should consider this trial
Good fit: Adults (≥18 years) with relapsed or refractory AML or higher‑risk MDS, bone marrow blasts ≥5%, ECOG performance status 0–2, acceptable laboratory values, and prior therapy failure who can undergo required bone marrow sampling are the intended candidates.
Not a fit: Patients with newly diagnosed disease, bone marrow blasts below 5%, ECOG performance status >2, significant uncontrolled comorbidities, or inability to undergo bone marrow sampling are unlikely to be eligible or to benefit from this early‑phase trial.
Why it matters
Potential benefit: If GEN3018 is safe and active, it could produce remissions or longer disease control for people with relapsed or refractory AML or HR‑MDS who have limited treatment options.
How similar studies have performed: Antibody‑based therapies in AML/MDS (for example gemtuzumab ozogamicin) have produced benefit in some patient subsets, but many novel antibodies are untested in humans and outcomes remain uncertain.
Eligibility criteria
Show full inclusion / exclusion criteria
Key Inclusion Criteria: All Participants: * Be at least 18 years of age at the time of signing informed consent form (ICF). * Participant's life expectancy at screening is judged to be at least 3 months. * Must have fresh bone marrow samples collected at screening. * Bone marrow (BM) blasts ≥ 5% at screening. * Eastern Cooperative Oncology Group (ECOG) performance status (PS) score of ≤ 2. * Has acceptable laboratory test results during the screening period Participants with R/R AML: * Relapsed or refractory AML, either de novo or secondary, and must have failed all conventional therapies. * Relapsed or refractory to at least one prior line of therapy. Participants with R/R HR-MDS: * Diagnosed with high- or very-high risk MDS according to International Prognostic Scoring System (IPSS-R) (score of \> 4.5 ie, high or very high) or World Health Organization (WHO) 2022 classification (ie, MDS-IB1 or MDS-IB2). * Refractory or relapsed after hypomethylating agents (HMAs) (such as azacitidine or decitabine). Key Exclusion Criteria: All Participants: * Diagnosis of acute promyelocytic leukemia (APL). * Presence of extramedullary AML at screening. * Prior autologous or allogenic hematopoietic stem cell transplant (HSCT) within 3 months prior to initiation of trial treatment. * Active graft-versus-host disease. * History of severe immune-related adverse events. * Treatment with anti-cancer agent (eg, small molecule, antibody, chemotherapy, radiation therapy), or major surgery within 2 weeks prior to the first dose of GEN3018. Other protocol-defined Inclusion and Exclusion criteria may apply.
Where this trial is running
Aarhus and 8 other locations
- Aarhus Universitetshospital - Skejby — Aarhus, Denmark (Recruiting)
- Copenhagen Rigshospitalet — Copenhagen, Denmark (Recruiting)
- Charite Campus Virchow-Klinikum — Berlin, Germany (Not_yet_recruiting)
- Universitätsklinikum Carl Gustav Carus Dresden — Dresden, Germany (Not_yet_recruiting)
- Klinikum der Universität München Großhadern — Munich, Germany (Not_yet_recruiting)
- Hospital Universitari Vall d'Hebron — Barcelona, Spain (Not_yet_recruiting)
- Hospital Clínic de Barcelona — Barcelona, Spain (Not_yet_recruiting)
- Hospital Universitario 12 de Octubre — Madrid, Spain (Not_yet_recruiting)
- Hospital Universitari i Politècnic La Fe — Valencia, Spain (Not_yet_recruiting)
Study contacts
- Study coordinator: Genmab Trial Information
- Email: clinicaltrials@genmab.com
- Phone: +4570202728
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.