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Gemtuzumab Ozogamicin

Phase 3

Acute Myeloid Leukemia | Small molecule | Oncology |AbbVie Inc.|Last Updated: Apr 28, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedACTIVE_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment130
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05183035Venetoclax in Children With Relapsed Acute Myeloid Leukemia (AML)PHASE3 RECRUITING 130Oct 1, 2022Apr 1, 2031Apr 28, 202689 United States, Australia +18
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Study Endpoints
Primary Endpoints
Overall Survival (OS)
Up to 5 years
Secondary Endpoints
Morphology Event Free Survival (EFS)
Up to 5 years
Flow-based Event Free Survival (EFS)
Up to 5 years
Flow-based Overall Response Rate (ORR)
Up to Day 84
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Arm A: Control Arm without VenetoclaxACTIVE_COMPARATORDuring Cycle 1 (42-day-cycles), participants will receive 30 mg/m\^2 of fludarabine followed by 2 g/m\^2 of cytarabine on Days 1-5. Gemtuzumab 3 mg/m\^2 will be given on Day 6 (only for participants with CD33 expression on leukemia blasts). During Cycle 2 participants will receive 30 mg/m\^2 of fludarabine followed by 2 g/m\^2 of cytarabine on Days 1-5. After Cycle 2 participants are assessed for HSCT or azacitidine maintenance therapy.
Arm B: Experimental Arm with VenetoclaxEXPERIMENTALDuring Cycle 1 (42-day-cycles), participants will receive 300 mg adult dose equivalent of venetoclax once on Day 1 followed by 600 mg adult dose equivalent of venetoclax on Days 2-21. Participants will also receive 30 mg/m\^2 of fludarabine followed by 2 g/m\^2 of cytarabine on Days 8-12. Gemtuzumab 3 mg/m\^2 will be given on Day 13 (only for participants with CD33 expression on leukemia blasts). During Cycle 2, participants will receive 600 mg adult dose equivalent of venetoclax on Days 1-21. Participants will receive 30 mg/m\^2 of fludarabine followed by 2 g/m\^2 of cytarabine on Days 1-5. After Cycle 2 participants are assessed for HSCT or azacitidine maintenance therapy in combination with venetoclax.
Interventions
NameTypeDescription
FludarabineDRUGIntravenous (IV) infusion
CytarabineDRUGIntravenous (IV) infusion
Gemtuzumab OzogamicinDRUGIntravenous (IV) infusion
AzacitidineDRUGIntravenous (IV) infusion or subcutaneous injection
VenetoclaxDRUGOrally via tablet or powder suspension
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Eligibility Criteria
Age Range29 Days — 21 Years
SexALL
Healthy VolunteersNo
Study Sites89

Inclusion Criteria * Participants must have enrolled on APAL2020SC, NCT Number: NCT04726241 prior to enrollment on ITCC-101/APAL2020D. (This is only applicable for participants in USA/Canada/Australia/New Zealand sites/Blood Cancer United territory). * Participants must be ≥ 29 days of age and ≤ 21...

Countries:United StatesAustraliaAustriaBelgiumCanadaCzechiaDenmarkFinlandFranceGermanyIsraelItalyJapanNetherlandsNew ZealandNorwayPortugalSpainSwedenSwitzerland
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Recent Changes (Last 90 Days)
LOWMay 24, 2026NCT05183035studyFirstPostDate: changed