The drug that will be investigated in the trial is an antibody, GEN3018. Since this is the first trial of GEN3018 in humans, the main purpose is to evaluate safety. In addition to safety, the trial will determine the recommended GEN3018 dose(s) to be tested in a larger group of participants and assess preliminary anti-tumor activity of GEN3018. GEN3018 will be studied in refractory (resistant to treatment) or relapsed (disease has returned) acute myeloid leukemia (also known as R/R AML) and refractory or relapsed higher-risk myelodysplastic syndrome (also known as R/R HR-MDS). The trial consists of 2 parts:
1. Part 1 Dose Escalation will test increasing doses of GEN3018 to identify a safe dose level to be tested in the next part
2. Part 2 Dose Refinement will further test the GEN3018 dose(s) determined from the Dose Escalation.
Up to 78 participants may be treated in this trial (up to 60 participants in Part 1; up to 18 participants in Part 2).
For an individual participant in the trial, the estimated treatment duration will be up to 1 year. Participation in the trial will require regular scheduled visits to the site. At site visits, there will be various tests (such as blood draws) to monitor whether the treatment is safe and effective. Participants will also be contacted every 3 months after treatment ends to monitor how they are doing.
All participants in the trial will receive active drug (ie, GEN3018); no one will be given placebo.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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 …
Questions worth asking your doctor
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.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
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
Part 1: Number of Participants with Dose-limiting Toxicities (DLTs)
Timeframe: 28 days
2
Parts 1 and 2: Number of Participants with Adverse Events (AEs)