Acute Myeloid Leukemia RefractoryPhase 1NCT06372717
A Study to Investigate APL-4098 Alone and in Combination in Adults With AML or MDS
A Phase 1 study testing APL-4098, Azacitidine and APL-4098 and Azacitidine and Venetoclax and APL-4098 in people with Acute Myeloid Leukemia Refractory and Myelodysplastic Syndrome Acute Myeloid Leukemia, run by Apollo Therapeutics Ltd at 9 sites in 2 countries.
Trial summary
This is an open-label, Phase 1 study to determine the safety, tolerability, and efficacy of APL-4098 alone, and in combination with azacitidine, and in combination with azacitidine plus venetoclax for the treatment of acute myeloid leukemia (AML), myelodysplastic syndrome (MDS)/AML and MDS-excess blasts (EB). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia Refractory, Myelodysplastic Syndrome Acute Myeloid Leukemia, Myelodysplastic Syndrome With Excess Blasts
- What is being tested?
- APL-4098, Azacitidine and APL-4098 and Azacitidine and Venetoclax and APL-4098
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 9 sitesin 2 countries
- Study dates
- 4 Jun 2024to Aug 2027
- Phase
- Phase 1
- 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
- 18 years or older
- Confirmed diagnosis of acute myeloid leukemia (R/R AML), myelodysplastic syndrome (MDS)/ AML, or MDS-excess blasts (MDS-EB) with the following characteristics: R/R AML (primary or secondary, including treatment-related), participant is intolerant to, or considered ineligible for available therapies known to provide clinical benefit; Newly diagnosed (ND AML), previously untreated, and considered unfit for available front line therapies or standard intensive chemotherapy.
- WBC count ≤ 25,000/microliter
- ECOG Performance Status of ≤ 2
- Weight ≥ 40kg
- Female participants of childbearing potential must have negative serum pregnancy test at screening; must not plan to become pregnant or have ova harvested or breastfeed while on study; must be willing to use specific contraception or avoid intercourse
- Male participants must be willing to use specific contraception and not plan to impregnant a female partner or donate sperm while on study
- Participant must be willing and able to provide written informed consent and to comply with the requirements of the trial
Who cannot join
- Certain prior therapies such as: received an allogeneic stem cell transplant within 6 months of screening, received an autologous stem cell transplant within 3 months of screening, received any anti-cancer treatments within 2 weeks of Cycle 1 Day 1, prior radiation therapy within 4 weeks of screening
- Certain medical conditions such as: other malignancies, myocardial infarction within 6 months of screening, symptomatic congestive heart failure, uncontrolled active infection, history of arterial thrombosis within 6 months of screening
- Diagnostic assessments: Left ventricular ejection fraction < 45%, Fridericia's corrected QT interval > 470msec, Aspartate aminotransferase and/or alanine aminotransferase > 3 x upper limit of normal (ULN), total bilirubin > 1.5 x ULN, calculated or measured creatinine clearance < 45 mL/minute (multiply by 0.85 if female)
- Infectious disease: HIV positive, active hepatitis B and/or C
Trial locations and contacts
9 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
9 sites in 2 countries
Australia5 sites, 4 recruiting
- Recruiting
Monash Health
Clayton, Victoria, 3168
- Recruiting
St. Vincent's Hospital Melbourne
Fitzroy, Victoria, 3065
- Recruiting
The Alfred Hospital
Melbourne, Victoria, 3004
- Terminated
Hollywood Private Hospital
Nedlands, Western Australia, 6009
- Recruiting
Royal Perth Hospital
Perth, Western Australia, 6000
United Kingdom4 sites, 4 recruiting
- Recruiting
University Hospital of Wales
Cardiff, CF14 4XW
Steven Knapper
- Recruiting
Beatson West of Scotland Cancer Centre
Glasgow, G120YN
- Recruiting
The Royal Marsden Hospital
London, SM2 5PT
- Recruiting
Sarah Cannon Research Institute UK
London, W1G 0PU
Sarah Cannon Research Institute, 0044 203 21 95 200, enquiries.SCRI@hcahealthcare.co.uk
About this study
Official title: A Phase 1 Study to Assess the Safety and Antitumor Activity of APL-4098 Alone and in Combination With Azacitidine and in Combination With Azacitidine Plus Venetoclax in Adults With Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome/AML (MDS/AML) or Myelodysplastic Syndrome With Excess Blasts (MDS-EB)
This is an open-label, Phase 1 study to determine the safety, tolerability, and efficacy of APL-4098 alone, and in combination with azacitidine, and in combination with azacitidine plus venetoclax for the treatment of acute myeloid leukemia (AML), myelodysplastic syndrome (MDS)/AML and MDS-excess blasts (EB).
- Conditions
- Acute Myeloid Leukemia Refractory, Myelodysplastic Syndrome Acute Myeloid Leukemia, Myelodysplastic Syndrome With Excess Blasts, Acute Myeloid Leukemia, in Relapse, Acute Myeloid Leukemia (AML), Newly Diagnosed Acute Myeloid Leukemia (AML)
- Being tested
- APL-4098, Azacitidine and APL-4098, Azacitidine and Venetoclax and APL-4098
- Medical subject headings
- GATA2 Deficiency, Anemia, Refractory, with Excess of Blasts, Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
APL-4098Drug
APL-4098 is administered orally in 28-day cycles
Azacitidine and APL-4098Drug
Azacitidine is administered at the standard dose on Day 1 - Day 7 of each 28-day cycle; APL-4098 is administered orally.
Azacitidine and Venetoclax and APL-4098Drug
Azacitidine is administered at the standard dose on Day 1 - Day 7 of each 28-day cycle; Venetoclax is administered orally; APL-4098 is administered orally.
| Study group | Role | What they receive |
|---|---|---|
| Dose Escalation Phase: APL-4098 monotherapy | Experimental | Dose escalation with different dosing levels of APL-4098. Receives: Drug: APL-4098 |
| Dose Escalation Phase: APL-4098 and azacitidine | Experimental | Dose escalation with different dosing levels of APL-4098 in combination with azacitidine. Receives: Drug: Azacitidine and APL-4098 |
| APL-4098 and azacitidine and venetoclax | Experimental | Dose escalation with different dosing levels of APL-4098 in combination with azacitidine and venetoclax. Receives: Drug: Azacitidine and Venetoclax and APL-4098 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment Emergent Adverse Events [Safety]
Evaluation of safety parameters including treatment emergent adverse events as detected by hematology, chemistry, coagulation safety labs, physical exams, vital signs, Electrocardiogram results
Measured: Through study completion, approximately one year
Incidence of Dose Limiting Toxicities [Tolerability]
Evaluation of tolerability parameters including dose limiting toxicities as detected by hematology, chemistry, coagulation safety labs, physical exams, vital signs, and Electrocardiogram results
Measured: Cycle 1 Day 1 to Cycle 2 Day 1 (a cycle is 28 days)
Determine Recommended Phase 2 Dose (RP2D)/Recommended dose range (RDR) levels of APL-4098 alone, in combination with azacitidine, and in combination with azacitidine plus venetoclax.
Measured: Approximately one year
Assess the Pharmacokinetics of APL-4098
Evaluate PK parameters: Maximum plasma concentration (Cmax)
Measured: On Days 1, 2, 8, and 15 of Cycle 1, and on Day 1 of Cycle 2 (each cycle is 28 days)
Assess the Pharmacokinetics of APL-4098
Evaluate PK parameters: area under the curve (AUC)
Measured: On Days 1, 2, 8, and 15 of Cycle 1, and on Day 1 of Cycle 2 (each cycle is 28 days)
Assess the Pharmacokinetics of APL-4098
Evaluate PK parameters: Time to peak concentration (Tmax)
Measured: On Days 1, 2, 8, and 15 of Cycle 1, and on Day 1 of Cycle 2 (each cycle is 28 days)
Secondary outcomes (1)
Assess response to disease with APL-4098 alone, in combination with azacitidine, and in combination with azacitidine plus venetoclax.
R/R AML and MDS/AML participants: response is assessed per European LeukemiaNet (ELN) 2022 criteria MDS-EB participants: response is assessed per revised International Working Group 2023 response criteria
Measured: Response is assessed at Cycle 2 Day 1, Cycle 3 Day 1, Cycle 4 Day 1, Cycle 7 Day 1 (each cycle is 28 days long), then every three cycles thereafter (assessed for up to 2 years)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Apr 2024
- Study start4 Jun 2024
- Primary completionJul 2027 (estimated)
- Study completionAug 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Apollo Therapeutics Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Sanjay Aggarwal, MD, Study chair, Apollo Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06372717
- Sponsor's protocol ID
- AP30CP01
- Status last verified
- Oct 2026
- First submitted
- 11 Mar 2024
- Last updated
- 28 Oct 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Study to Assess the Safety and Antitumor Activity of APL-4098 Alone and in Combination With Azacitidine and in Combination With Azacitidine Plus Venetoclax in Adults With Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome/AML (MDS/AML) or Myelodysplastic Syndrome With Excess Blasts (MDS-EB)
- ClinicalTrials.gov ID
- NCT06372717
- Acronym / sponsor ID
- AP30CP01
- Conditions
- Acute Myeloid Leukemia Refractory, Myelodysplastic Syndrome Acute Myeloid Leukemia, Myelodysplastic Syndrome With Excess Blasts, Acute Myeloid Leukemia, in Relapse, Acute Myeloid Leukemia (AML), Newly Diagnosed Acute Myeloid Leukemia (AML)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Apollo Therapeutics LtdIndustry (pharma, biotech, devices)
- Study start
- 4 Jun 2024 (actual)
- Primary completion
- Jul 2027 (estimated)
- Study completion
- Aug 2027 (estimated)
- First posted
- 18 Apr 2024 (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.
This is an open-label, Phase 1 study to determine the safety, tolerability, and efficacy of APL-4098 alone, and in combination with azacitidine, and in combination with azacitidine plus venetoclax for the treatment of acute myeloid leukemia (AML), myelodysplastic syndrome (MDS)/AML and MDS-excess blasts (EB).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Dose Escalation Phase: APL-4098 monotherapy | Experimental | Drug: APL-4098Dose escalation with different dosing levels of APL-4098. |
| Dose Escalation Phase: APL-4098 and azacitidine | Experimental | Drug: Azacitidine and APL-4098Dose escalation with different dosing levels of APL-4098 in combination with azacitidine. |
| APL-4098 and azacitidine and venetoclax | Experimental | Drug: Azacitidine and Venetoclax and APL-4098Dose escalation with different dosing levels of APL-4098 in combination with azacitidine and venetoclax. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 18 years or older
- Confirmed diagnosis of acute myeloid leukemia (R/R AML), myelodysplastic syndrome (MDS)/ AML, or MDS-excess blasts (MDS-EB) with the following characteristics: R/R AML (primary or secondary, including treatment-related), participant is intolerant to, or considered ineligible for available therapies known to provide clinical benefit; Newly diagnosed (ND AML), previously untreated, and considered unfit for available front line therapies or standard intensive chemotherapy.
- WBC count ≤ 25,000/microliter
- ECOG Performance Status of ≤ 2
- Weight ≥ 40kg
Key exclusion criteria (4 listed)
- Certain prior therapies such as: received an allogeneic stem cell transplant within 6 months of screening, received an autologous stem cell transplant within 3 months of screening, received any anti-cancer treatments within 2 weeks of Cycle 1 Day 1, prior radiation therapy within 4 weeks of screening
- Certain medical conditions such as: other malignancies, myocardial infarction within 6 months of screening, symptomatic congestive heart failure, uncontrolled active infection, history of arterial thrombosis within 6 months of screening
- Diagnostic assessments: Left ventricular ejection fraction < 45%, Fridericia's corrected QT interval > 470msec, Aspartate aminotransferase and/or alanine aminotransferase > 3 x upper limit of normal (ULN), total bilirubin > 1.5 x ULN, calculated or measured creatinine clearance < 45 mL/minute (multiply by 0.85 if female)
- Infectious disease: HIV positive, active hepatitis B and/or C
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 |
|---|---|---|
| APL-4098APL-4098 is administered orally in 28-day cycles | Drug | Dose Escalation Phase: APL-4098 monotherapy |
| Azacitidine and APL-4098Azacitidine is administered at the standard dose on Day 1 - Day 7 of each 28-day cycle; APL-4098 is administered orally. | Drug | Dose Escalation Phase: APL-4098 and azacitidine |
| Azacitidine and Venetoclax and APL-4098Azacitidine is administered at the standard dose on Day 1 - Day 7 of each 28-day cycle; Venetoclax is administered orally; APL-4098 is administered orally. | Drug | APL-4098 and azacitidine and venetoclax |
Primary outcome
Incidence of Treatment Emergent Adverse Events [Safety]
Time frame: Through study completion, approximately one year
Evaluation of safety parameters including treatment emergent adverse events as detected by hematology, chemistry, coagulation safety labs, physical exams, vital signs, Electrocardiogram results
Incidence of Dose Limiting Toxicities [Tolerability]
Time frame: Cycle 1 Day 1 to Cycle 2 Day 1 (a cycle is 28 days)
Evaluation of tolerability parameters including dose limiting toxicities as detected by hematology, chemistry, coagulation safety labs, physical exams, vital signs, and Electrocardiogram results
Determine Recommended Phase 2 Dose (RP2D)/Recommended dose range (RDR) levels of APL-4098 alone, in combination with azacitidine, and in combination with azacitidine plus venetoclax.
Time frame: Approximately one year
Assess the Pharmacokinetics of APL-4098
Time frame: On Days 1, 2, 8, and 15 of Cycle 1, and on Day 1 of Cycle 2 (each cycle is 28 days)
Evaluate PK parameters: Maximum plasma concentration (Cmax)
Assess the Pharmacokinetics of APL-4098
Time frame: On Days 1, 2, 8, and 15 of Cycle 1, and on Day 1 of Cycle 2 (each cycle is 28 days)
Evaluate PK parameters: area under the curve (AUC)
Assess the Pharmacokinetics of APL-4098
Time frame: On Days 1, 2, 8, and 15 of Cycle 1, and on Day 1 of Cycle 2 (each cycle is 28 days)
Evaluate PK parameters: Time to peak concentration (Tmax)
1 secondary outcome
Assess response to disease with APL-4098 alone, in combination with azacitidine, and in combination with azacitidine plus venetoclax.
Time frame: Response is assessed at Cycle 2 Day 1, Cycle 3 Day 1, Cycle 4 Day 1, Cycle 7 Day 1 (each cycle is 28 days long), then every three cycles thereafter (assessed for up to 2 years)
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 28 Oct 2026.
- Individual participant dataSharing planned: No
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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.
Sanjay Aggarwal, MD study chair
Apollo Therapeutics
9 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing APL-4098, Azacitidine and APL-4098 and Azacitidine and Venetoclax and APL-4098 in people with Acute Myeloid Leukemia Refractory and Myelodysplastic Syndrome Acute Myeloid Leukemia, run by Apollo Therapeutics Ltd at 9 sites in 2 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 8 inclusion and 4 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 28 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in Australia, United Kingdom. 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 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.
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 Apollo Therapeutics (781-479-2267, AP30@apollotx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Treatment Emergent Adverse Events [Safety] (time frame: Through study completion, approximately one year). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 28 Oct 2026.