A Study to Investigate APL-4098 Alone and/or in Combination With Azacitidine in R/R AML and High-Risk MDS

NCT ID: NCT06372717

Last Updated: 2025-09-25

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

122 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-06-04

Study Completion Date

2027-04-01

Brief Summary

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This is an open-label, Phase 1/2 study to determine the safety, tolerability, and efficacy of APL-4098 alone and/or in combination with azacitidine for the treatment of relapsed or refractory (R/R) acute myeloid leukemia (AML), myelodysplastic syndrome (MDS)/AML and MDS-excess blasts (EB). Participants with the MDS-EB subtype will be eligible for the Phase 1 part of the study only.

Detailed Description

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Conditions

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Acute Myeloid Leukemia Refractory Myelodysplastic Syndrome Acute Myeloid Leukemia Myelodysplastic Syndrome With Excess Blasts Acute Myeloid Leukemia, in Relapse

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Dose Escalation Phase: APL-4098 monotherapy

Dose escalation with different dosing levels of APL-4098.

Group Type EXPERIMENTAL

APL-4098

Intervention Type DRUG

APL-4098 is administered orally in 28-day cycles

Dose Escalation Phase: APL-4098 and azacitidine

Dose escalation with different dosing levels of APL-4098 in combination with azacitidine (75 mg/m2).

Group Type EXPERIMENTAL

Azacitidine and APL-4098

Intervention Type DRUG

Azacitidine is administered at the standard dose of 75mg/m2 on Day 1 - Day 7 of each 28-day cycle; APL-4098 is administered orally.

Phase 2 Dose Expansion: APL-4098 monotherapy

Group Type EXPERIMENTAL

APL-4098

Intervention Type DRUG

APL-4098 is administered orally in 28-day cycles

Phase 2 Dose Expansion: APL-4098 and azacitidine

Group Type EXPERIMENTAL

Azacitidine and APL-4098

Intervention Type DRUG

Azacitidine is administered at the standard dose of 75mg/m2 on Day 1 - Day 7 of each 28-day cycle; APL-4098 is administered orally.

Interventions

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APL-4098

APL-4098 is administered orally in 28-day cycles

Intervention Type DRUG

Azacitidine and APL-4098

Azacitidine is administered at the standard dose of 75mg/m2 on Day 1 - Day 7 of each 28-day cycle; APL-4098 is administered orally.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* 18 years or older
* Confirmed diagnosis of relapsed refractory 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.
* 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 we 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

Exclusion Criteria

* 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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Apollo Therapeutics Ltd

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Sanjay Aggarwal, MD

Role: STUDY_CHAIR

Apollo Therapeutics

Locations

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Monash Health

Clayton, Victoria, Australia

Site Status RECRUITING

St. Vincent's Hospital Melbourne

Fitzroy, Victoria, Australia

Site Status RECRUITING

The Alfred Hospital

Melbourne, Victoria, Australia

Site Status RECRUITING

Hollywood Private Hospital

Nedlands, Western Australia, Australia

Site Status RECRUITING

Royal Perth Hospital

Perth, Western Australia, Australia

Site Status RECRUITING

The Royal Marsden Hospital

London, , United Kingdom

Site Status RECRUITING

Sarah Cannon Research Institute UK

London, , United Kingdom

Site Status RECRUITING

Countries

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Australia United Kingdom

Central Contacts

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Apollo Therapeutics

Role: CONTACT

781-479-2267

Facility Contacts

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Sarah Cannon Research Institute

Role: primary

0044 203 21 95 200

Other Identifiers

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AP30CP01

Identifier Type: -

Identifier Source: org_study_id

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