R/R AMLPhase 1NCT07384715
First-in-human (FIH) Trial of GEN3018 in Relapsed or Refractory (R/R) Acute Myeloid Leukemia (AML) or Higher-risk Myelodysplastic Syndrome (HR-MDS)
A Phase 1 study testing GEN3018 in people with R/R AML and R/R HR-MDS, run by Genmab at 6 sites in 2 countries.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- R/R AML, R/R HR-MDS, Acute Myeloid Leukemia
- What is being tested?
- GEN3018
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 78 participants
- Where
- 6 sitesin 2 countries
- Study dates
- 16 Feb 2026to 20 Apr 2030
- Phase
- Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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).
Who cannot join
- 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.
Trial locations and contacts
6 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
6 sites in 2 countries
Spain4 sites, 4 recruiting
- Recruiting
Hospital Universitari Vall d'Hebron
Barcelona, Barcelona, 08035
- Recruiting
Hospital Clínic de Barcelona
Barcelona, Barcelona, 08036
- Recruiting
Hospital Universitario 12 de Octubre
Madrid, Madrid, 28041
- Recruiting
Hospital Universitari i Politècnic La Fe
Valencia, Valencia, 46026
Denmark2 sites, 2 recruiting
- Recruiting
Aarhus Universitetshospital - Skejby
Aarhus, Denmark, 8200
- Recruiting
Copenhagen Rigshospitalet
Copenhagen, Denmark, 2100
About this study
Official title: An Open-Label, Multicenter, First-in-Human Trial of GEN3018 in Participants With Relapsed or Refractory Acute Myeloid Leukemia or Higher-Risk Myelodysplastic Syndrome
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:
- Part 1 Dose Escalation will test increasing doses of GEN3018 to identify a safe dose level to be tested in the next part
- Part 2 Dose Refinement will further test the GEN3018 dose(s) determined from the Dose Escalation.
Read more
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.
Read the detailed description
This is a FIH, Phase 1, open-label, multicenter trial in participants with R/R AML or R/R HR-MDS, to evaluate the safety, tolerability, pharmacokinetics (PK), immunogenicity, pharmacodynamics, and preliminary anti-tumor activity of GEN3018. The trial will be conducted in 2 parts: Dose Escalation (Part 1) and Dose Refinement (Part 2).
- Being tested
- GEN3018
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GEN3018Biological
Intravenous (IV) infusion.
| Study group | Role | What they receive |
|---|---|---|
| GEN3018 | Experimental | Participants will receive escalating doses of GEN3018 in Part 1 (Dose Escalation). In Part 2 (Dose Refinement), additional participants may be enrolled to evaluate the safety and preliminary efficacy data of the recommended Part 2 dose(s) as determined from the Part 1 Dose Escalation. Receives: Biological: GEN3018 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part 1: Number of Participants with Dose-limiting Toxicities (DLTs)
Measured: 28 days
Parts 1 and 2: Number of Participants with Adverse Events (AEs)
Measured: Up to approximately 36 months
Secondary outcomes (10)
Parts 1 and 2: Maximum Concentration (Cmax) of GEN3018
Measured: Cycle 1 and 2 (each cycle is 28 days)
Parts 1 and 2: Time to Cmax (tmax) of GEN3018
Measured: Cycle 1 and 2 (each cycle is 28 days)
Parts 1 and 2: Predose Trough Concentration (Ctrough) of GEN3018
Measured: Cycle 1 and 2 (each cycle is 28 days)
Parts 1 and 2: Area Under the Concentration-time Curve from Time 0 to Last Quantifiable Sample (AUClast) of GEN3018
Measured: Cycle 1 and 2 (each cycle is 28 days)
Parts 1 and 2: Elimination Half-life (t1/2) of GEN3018
Measured: Cycle 1 and 2 (each cycle is 28 days)
Parts 1 and 2: Clearance (CL) of GEN3018
Measured: Cycle1 and 2 (each cycle is 28 days)
Parts 1 and 2: Number of Participants with Anti-drug Antibodies (ADAs) Against GEN3018
Measured: Up to approximately 1 year
Parts 1 and 2: Overall Response Rate (ORR)
Measured: Up to approximately 1 year
Parts 1 and 2: Duration of Response (DOR)
Measured: Up to approximately 1 year
Parts 1 and 2: Time to Response (TTR)
Measured: Up to approximately 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Feb 2026
- Study start16 Feb 2026
- Primary completion18 Oct 2029 (estimated)
- Study completion20 Apr 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for R/R AMLStudies of the same condition, open and under way.
- More trials run by GenmabThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 78 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Genmab
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Official, Study director, Genmab
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07384715
- Sponsor's protocol ID
- GCT3018-01
- Other IDs
- 2025-523101-15-00, 2025
- Status last verified
- Oct 2026
- First submitted
- 27 Jan 2026
- Last updated
- 6 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No