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

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

NCT ID: NCT06372717

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

Conditions: Official terms:
Leukemia
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Preleukemia
Myelodysplastic Syndromes
Anemia, Refractory, with Excess of Blasts
GATA2 Deficiency
Syndrome
Azacitidine

Study type: Interventional

Study phase: Phase 1/Phase 2

Overall status: Recruiting

Study design:

Allocation: Randomized

Intervention model: Sequential Assignment

Primary purpose: Treatment

Masking: None (Open Label)

Intervention:

Intervention type: Drug
Intervention name: APL-4098
Description: APL-4098 is administered orally in 28-day cycles
Arm group label: Dose Escalation Phase: APL-4098 monotherapy
Arm group label: Phase 2 Dose Expansion: APL-4098 monotherapy

Intervention type: Drug
Intervention name: Azacitidine and APL-4098
Description: 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.
Arm group label: Dose Escalation Phase: APL-4098 and azacitidine
Arm group label: Phase 2 Dose Expansion: APL-4098 and azacitidine

Summary: 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.

Criteria for eligibility:
Criteria:
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

Gender: All

Minimum age: 18 Years

Maximum age: N/A

Healthy volunteers: No

Locations:

Facility:
Name: Monash Health

Address:
City: Clayton
Zip: 3168
Country: Australia

Status: Recruiting

Facility:
Name: St. Vincent's Hospital Melbourne

Address:
City: Fitzroy
Zip: 3065
Country: Australia

Status: Recruiting

Facility:
Name: The Alfred Hospital

Address:
City: Melbourne
Zip: 3004
Country: Australia

Status: Recruiting

Facility:
Name: Hollywood Private Hospital

Address:
City: Nedlands
Zip: 6009
Country: Australia

Status: Recruiting

Start date: June 4, 2024

Completion date: April 1, 2027

Lead sponsor:
Agency: Apollo Therapeutics Ltd
Agency class: Industry

Source: Apollo Therapeutics Ltd

Record processing date: ClinicalTrials.gov processed this data on November 12, 2024

Source: ClinicalTrials.gov page: https://clinicaltrials.gov/ct2/show/NCT06372717