Acute Myeloid Leukemia RefractoryPhase 1/2NCT05211570
AB8939 in Patients With Relapsed/Refractory Acute Myeloid Leukemia
A Phase 1/2 study testing AB8939 and Venetoclax in people with Acute Myeloid Leukemia Refractory and Acute Myeloid Leukemia, in Relapse, run by AB Science at 10 sites in 4 countries.
Trial summary
The primary objective is to define the safety and tolerability of AB8939 in patients with AML by determining the dose-limiting toxicities, the maximum tolerated dose, and the recommended dose for dose expansion study. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia Refractory, Acute Myeloid Leukemia, in Relapse, Myelodysplastic Syndrome Acute Myeloid Leukemia
- What is being tested?
- AB8939 and Venetoclax
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 78 participants
- Where
- 10 sitesin 4 countries
- Study dates
- 1 Jun 2022to Dec 2026
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
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
- Patients with documented diagnosis of acute myeloid leukemia (AML) based on the last version of the World Health Organization classification and eligible to second or third line of treatment.
- Patients with documented diagnosis of refractory melyodisplastic syndrome in second or third line of treatment, and with high risk at prognosis based on the IPSS-R scoring system.
- ECOG performance status ≤ 1
- Patients are able to understand, sign, and date the written informed consent form at screening visit prior to any protocol-specific procedures
- Patients are able and willing to comply with study procedures as per protocol, including bone marrow biopsies
- Patients with documented diagnosis of acute myeloid leukemia (AML) based on the last version of the World Health Organization classification and eligible to second or third line of treatment.
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- ECOG performance status ≤ 2
- Patients are able to understand, sign, and date the written informed consent form at screening visit prior to any protocol-specific procedures
- Patients are able and willing to comply with study procedures as per protocol, including bone marrow biopsies
Who cannot join
- Patients eligible to a standard of care
- Patients eligible to hematopoietic stem cell transplantation (HSCT) at the time of inclusion
- Patients diagnosed with acute promyelocytic leukemia (M3)
- Patients with clinically active CNS leukemia
- Patients with HSCT within 100 days prior to the first administration of AB8939
- Women who are lactating/breastfeeding or who plan to breastfeed while on study
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- Women with a positive pregnancy test
- Other protocol-defined inclusion/exclusion criteria may apply
- EXPANSION COHORT STUDY
- Patients eligible to a standard of care
- Patients eligible to hematopoietic stem cell transplantation (HSCT) at the time of inclusion
- Patients diagnosed with acute promyelocytic leukemia (M3)
- Patients with clinically active CNS leukemia
- Patients with HSCT within 100 days prior to the first administration of AB8939
- Women who are lactating/breastfeeding or who plan to breastfeed while on study
- Women with a positive pregnancy test
- Other protocol-defined inclusion/exclusion criteria may apply
Trial locations and contacts
10 sites in 4 countries. Contact the study team or a site near you to ask about taking part.
10 sites in 4 countries
Spain7 sites, 7 recruiting
- Recruiting
General University Hospital of Alicantet (Hospital General Universitario Dr. Balmis de Alicante)
Alicante
- Recruiting
Hospital San Pedro de Alcantara
Cáceres
- Recruiting
Hospital Universitario 12 de Octubre
Madrid
- Recruiting
Hospital Universitario Quirónsalud
Madrid
- Recruiting
MD Anderson Cancer Center Madrid
Madrid
- Recruiting
Clínica Universidad de Navarra
Pamplona
- Recruiting
Virgen del Rocío University Hospital (Hospital Universitario Virgen del Rocío)
Seville
United States1 site, 1 recruiting
- Not yet recruiting
MD Anderson Cancer Center
Houston, Texas, 77030
France1 site, 1 recruiting
- Not yet recruiting
Institut Paoli Calmettes
Marseille
Greece1 site, 1 recruiting
- Recruiting
National and Kapodistrian University of Athens NKUA · Department of Hematology and Bone marrow Transplantation Unit
Athens
About this study
Official title: A Phase 1/2 Study to Assess the Safety, Pharmacokinetics, and Efficacy of Daily Intravenous AB8939 in Patients With Relapsed/Refractory Acute Myeloid Leukemia
The primary objective is to define the safety and tolerability of AB8939 in patients with AML by determining the dose-limiting toxicities, the maximum tolerated dose, and the recommended dose for dose expansion study.
Read the detailed description
This is a Phase 1/2, open-label, multi-center, non-randomized, 2-part study in patients with refractory and relapsed AML and refractory myelodysplastic syndrome.
Study AB18001 has a multi-stage design. The first part is a dose escalation study that aims to determine the safety, tolerability and pharmacokinetic profiles of consecutive daily intravenous administration of AB8939 in patients with refractory or relapsed AML or patients with refractory myelodysplastic syndrome, and to determine the recommended dose for the second-stage dose expansion study. This dose expansion study aims to determine the schedule for a Phase 2 trial in patients with relapsed/refractory AML and to also provide an early efficacy assessment of AB8939.
- Conditions
- Acute Myeloid Leukemia Refractory, Acute Myeloid Leukemia, in Relapse, Myelodysplastic Syndrome Acute Myeloid Leukemia
- Being tested
- AB8939, Venetoclax
- Medical subject headings
- Leukemia, Myeloid, Acute, GATA2 Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AB8939Drug
Intravenous injection (from an initial dose of 0.9 mg/m²)
VenetoclaxDrug
the recommended starting dose for AML indication is 100 mg. Dose escalating regimen as per SmPC.
| Study group | Role | What they receive |
|---|---|---|
| AB8939 | Experimental | AB8939 administered as a single agent Receives: Drug: AB8939 |
| AB8939 plus Venetoclax | Experimental | AB8939 administered in combination with venetoclax Receives: Drug: AB8939; Drug: Venetoclax |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rate of dose limiting toxicity (DLT)
Identification of the Maximal Tolerated Dose for different dosing schedules
Measured: Up to 56 days
Secondary outcomes (1)
Objective Response Rate
The proportion of patients who have a partial or complete response to therapy
Measured: Up to 56 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jan 2022
- Study start1 Jun 2022
- Primary completionAug 2026 (estimated)
- Study completionDec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Acute Myeloid Leukemia RefractoryStudies of the same condition, open and under way.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- Open-label, multi-centre, non-randomized, 2-part study
- Masking
- None (open label)
- Enrolment
- 78 participants (estimated)
Sponsor and investigators
- Lead sponsor
- AB Science
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Norbert Vey, MD, Principal investigator, Institut Paoli Calmettes, Marseille, FranceNicholas Short, MD, Principal investigator, MD Anderson Cancer Center, Houston, Texas
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05211570
- Sponsor's protocol ID
- AB18001
- Other IDs
- 2020-005122-28
- Status last verified
- Sep 2025
- First submitted
- 12 Jan 2022
- Last updated
- 2 Oct 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Goubard A, Humbert M, Mansfield C, Hermine O, Dubreuil P, et al. In Vivo Assessment of the Next Generation Microtubule-Destabilizing Agent AB8939 in Patient-derived Xenograft Models of Acute Myeloid Leukemia. Blood (2019) 134 (Supplement_1): 5142. doi.org/10.1182/blood-2019-127143
Goubard A, Humbert M, Mansfield C, Hermine O, Dubreuil P, et al. AB8939, a Microtubule-Destabilizing Agent with Potential to Overcome Multidrug Resistance, is Active Across the Range (M0-M7) of Acute Myeloid Leukemia Subtypes. Blood (2019) 134 (Supplement_1): 5154. doi.org/10.1182/blood-2019-127021
Humbert M, Goubard A, Mansfield C, Hermine O, Dubreuil P, et al. Anticancer Activity of a Highly Potent Small Molecule Tubulin Polymerization Inhibitor, AB8939. Blood (2019) 134 (Supplement_1): 2075. doi.org/10.1182/blood-2019-122540
Study at a glance
From the registry record, last updated by the sponsor on 2 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2 Study to Assess the Safety, Pharmacokinetics, and Efficacy of Daily Intravenous AB8939 in Patients With Relapsed/Refractory Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT05211570
- Acronym / sponsor ID
- AB18001
- Conditions
- Acute Myeloid Leukemia Refractory, Acute Myeloid Leukemia, in Relapse, Myelodysplastic Syndrome Acute Myeloid Leukemia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 78 participants (estimated)
- Lead sponsor
- AB ScienceIndustry (pharma, biotech, devices)
- Study start
- 1 Jun 2022 (actual)
- Primary completion
- Aug 2026 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 27 Jan 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective is to define the safety and tolerability of AB8939 in patients with AML by determining the dose-limiting toxicities, the maximum tolerated dose, and the recommended dose for dose expansion study.
Detailed description
This is a Phase 1/2, open-label, multi-center, non-randomized, 2-part study in patients with refractory and relapsed AML and refractory myelodysplastic syndrome.
Study AB18001 has a multi-stage design. The first part is a dose escalation study that aims to determine the safety, tolerability and pharmacokinetic profiles of consecutive daily intravenous administration of AB8939 in patients with refractory or relapsed AML or patients with refractory myelodysplastic syndrome, and to determine the recommended dose for the second-stage dose expansion study. This dose expansion study aims to determine the schedule for a Phase 2 trial in patients with relapsed/refractory AML and to also provide an early efficacy assessment of AB8939.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Open-label, multi-centre, non-randomized, 2-part study
| Arm | Type | Receives |
|---|---|---|
| AB8939 | Experimental | Drug: AB8939AB8939 administered as a single agent |
| AB8939 plus Venetoclax | Experimental | Drug: AB8939, Drug: VenetoclaxAB8939 administered in combination with venetoclax |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Patients with documented diagnosis of acute myeloid leukemia (AML) based on the last version of the World Health Organization classification and eligible to second or third line of treatment.
- Patients with documented diagnosis of refractory melyodisplastic syndrome in second or third line of treatment, and with high risk at prognosis based on the IPSS-R scoring system.
- ECOG performance status ≤ 1
- Patients are able to understand, sign, and date the written informed consent form at screening visit prior to any protocol-specific procedures
- Patients are able and willing to comply with study procedures as per protocol, including bone marrow biopsies
Key exclusion criteria (17 listed)
- Patients eligible to a standard of care
- Patients eligible to hematopoietic stem cell transplantation (HSCT) at the time of inclusion
- Patients diagnosed with acute promyelocytic leukemia (M3)
- Patients with clinically active CNS leukemia
- Patients with HSCT within 100 days prior to the first administration of AB8939
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AB8939Intravenous injection (from an initial dose of 0.9 mg/m²) | Drug | AB8939, AB8939 plus Venetoclax |
| Venetoclaxthe recommended starting dose for AML indication is 100 mg. Dose escalating regimen as per SmPC. | Drug | AB8939 plus Venetoclax |
Primary outcome
Rate of dose limiting toxicity (DLT)
Time frame: Up to 56 days
Identification of the Maximal Tolerated Dose for different dosing schedules
1 secondary outcome
Objective Response Rate
Time frame: Up to 56 days
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Oct 2025.
- BackgroundGoubard A, Humbert M, Mansfield C, Hermine O, Dubreuil P, et al. In Vivo Assessment of the Next Generation Microtubule-Destabilizing Agent AB8939 in Patient-derived Xenograft Models of Acute Myeloid Leukemia. Blood (2019) 134 (Supplement_1): 5142. doi.org/10.1182/blood-2019-127143
- BackgroundGoubard A, Humbert M, Mansfield C, Hermine O, Dubreuil P, et al. AB8939, a Microtubule-Destabilizing Agent with Potential to Overcome Multidrug Resistance, is Active Across the Range (M0-M7) of Acute Myeloid Leukemia Subtypes. Blood (2019) 134 (Supplement_1): 5154. doi.org/10.1182/blood-2019-127021
- BackgroundHumbert M, Goubard A, Mansfield C, Hermine O, Dubreuil P, et al. Anticancer Activity of a Highly Potent Small Molecule Tubulin Polymerization Inhibitor, AB8939. Blood (2019) 134 (Supplement_1): 2075. doi.org/10.1182/blood-2019-122540
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Norbert Vey, MD principal investigator
Institut Paoli Calmettes, Marseille, France
Nicholas Short, MD principal investigator
MD Anderson Cancer Center, Houston, Texas
10 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing AB8939 and Venetoclax in people with Acute Myeloid Leukemia Refractory and Acute Myeloid Leukemia, in Relapse, run by AB Science at 10 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 2 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in Spain, United States, France, Greece. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Clinical Study Coordinator (+33(0)147200014, clinical@ab-science.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Rate of dose limiting toxicity (DLT) (time frame: Up to 56 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05211570, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Oct 2025; AzTrials last refreshed its copy on 6 Nov 2026.