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DRUG CLASS:

CD33-targeted antibody-drug conjugate

2d
GO-TAG: Tagraxofusp-erzs, an IL-3 Diphtheria Fusion Protein, in Combination With Gemtuzumab Ozogamicin in Patients With Relapsed/Refractory AML (clinicaltrials.gov)
P1, N=7, Terminated, Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | N=36 --> 7 | Trial completion date: Dec 2028 --> Jul 2025 | Active, not recruiting --> Terminated; Upon analysis of the first 7 participants accrued, it was determined that this combination treatment is not efficacious as treatment for patients with relapsed or refractory AML
Enrollment change • Trial completion date • Trial termination
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CD33 (CD33 Molecule) • IL3RA (Interleukin 3 Receptor Subunit Alpha)
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Mylotarg (gemtuzumab ozogamicin) • Elzonris (tagraxofusp-erzs)
8d
CHIP-AML22: a complex clinical trial in de novo pediatric AML patients, including a gemtuzumab ozogamicin randomization and targeted therapy with quizartinib in eligible subgroups, within the NOPHO-DB-SHIP consortium. (PubMed, Trials)
The risk-based approach and use of targeted therapies in CHIP-AML22 illustrate a shift toward more personalized treatment. Besides improving event-free survival, this study aims to contribute to the international consensus on strategies to reduce toxicity for all patients. The design of this study provides a dynamic framework, allowing for the potential introduction of emerging therapeutic options in the future.
Journal
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CD33 (CD33 Molecule)
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CD33 positive
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Vanflyta (quizartinib) • Mylotarg (gemtuzumab ozogamicin) • dexrazoxane
29d
A Phase Ia/Ib Trial of Revumenib Combined With Cytarabine, Daunorubicin, and Gemtuzumab Ozogamicin (GO) in Frontline and Relapsed /Refractory Pediatric Acute Leukemia Patients (clinicaltrials.gov)
P1, N=0, Withdrawn, M.D. Anderson Cancer Center | N=32 --> 0 | Trial completion date: Dec 2034 --> May 2026 | Initiation date: Dec 2027 --> May 2026 | Not yet recruiting --> Withdrawn | Trial primary completion date: Dec 2032 --> May 2026
Enrollment change • Trial completion date • Trial initiation date • Trial withdrawal • Trial primary completion date
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KMT2A (Lysine Methyltransferase 2A) • NUP98 (Nucleoporin 98 And 96 Precursor 2)
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cytarabine • Mylotarg (gemtuzumab ozogamicin) • daunorubicin • Revuforj (revumenib)
1m
Enrollment open
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ABL1 (ABL proto-oncogene 1) • BCR (BCR Activator Of RhoGEF And GTPase) • RUNX1 (RUNX Family Transcription Factor 1) • RUNX1T1 (RUNX1 Partner Transcriptional Co-Repressor 1)
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Venclexta (venetoclax) • cytarabine • Mylotarg (gemtuzumab ozogamicin) • daunorubicin • Starasid (cytarabine ocfosfate)
1m
A PBD-dimer containing antibody drug conjugate targeting CCRL2 for high-risk MDS/AML including TP53-mutated disease. (PubMed, Blood Adv)
The anti-CCRL2 ADC demonstrated strong CCRL2-selective cytotoxicity against cell lines derived from MDS/AML patients with TP53 mutations and erythroid features, surpassing the cytotoxic effects observed with gemtuzumab and PBD-conjugated anti-CD33 and anti-CD123 ADCs...This agent also suppressed the leukemic growth of TP53-mutated MDS/AML cell line xenografts, improving mice survival and decreasing the leukemic burden in patient-derived TP53-mutated MDS/AML xenografts. In conclusion, our study introduces CCRL2 as a potential new therapeutic target in high-risk MDS/AML including TP53-mutated subsets.
Journal
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TP53 (Tumor protein P53) • CD123 (Interleukin 3 Receptor Subunit Alpha) • IL3RA (Interleukin 3 Receptor Subunit Alpha) • CCRL2 (C-C Motif Chemokine Receptor Like 2)
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TP53 mutation
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Mylotarg (gemtuzumab ozogamicin)
1m
A Study of BL-M11D1 in Patients With Relapsed/Refractory Myelodysplastic Syndromes (clinicaltrials.gov)
P1/2, N=92, Not yet recruiting, Sichuan Baili Pharmaceutical Co., Ltd.
New P1/2 trial
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CD33 (CD33 Molecule)
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BL-M11D1
2ms
Analysis of Clinical Impact of CD33 rs12459419 Single-Nucleotide Polymorphism in AML Treated with Intensive Chemotherapy Without Gemtuzumab Ozogamicin. (PubMed, Int J Mol Sci)
To our knowledge, this is the first study to investigate the prognostic impact of the CD33 rs12459419 SNP per se on outcome and survival in adult AML patients treated with chemotherapy without GO. Validation in larger patient cohorts is required to conclusively rule out a prognostic role of the CD33 rs12459419 SNP in AML.
Retrospective data • Journal
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CD33 (CD33 Molecule)
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CD33 positive
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Mylotarg (gemtuzumab ozogamicin)
2ms
CRISPR-Cas9 CD33-deleted allogeneic hematopoietic cell transplantation with gemtuzumab ozogamicin maintenance in AML: a phase 1/2 trial. (PubMed, Nat Med)
In summary, trem-cel demonstrated safe, rapid, robust engraftment, and GO maintenance was administered without prolonged hematologic toxicity. ClinicalTrials.gov identifier: NCT04849910 .
P1/2 data • Journal
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CD33 (CD33 Molecule)
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Mylotarg (gemtuzumab ozogamicin)
2ms
SWOG-S0703: Azacitidine and Gemtuzumab Ozogamicin in Treating Older Patients With Previously Untreated Acute Myeloid Leukemia (clinicaltrials.gov)
P2, N=133, Active, not recruiting, National Cancer Institute (NCI) | Trial completion date: Mar 2026 --> Mar 2027
Trial completion date
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CD4 (CD4 Molecule)
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azacitidine • Mylotarg (gemtuzumab ozogamicin)
2ms
MyeChild 01: International Randomised Phase III Clinical Trial in Children With Acute Myeloid Leukaemia (clinicaltrials.gov)
P3, N=700, Recruiting, University of Birmingham | Active, not recruiting --> Recruiting
Enrollment open
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cytarabine • cyclophosphamide • Mylotarg (gemtuzumab ozogamicin) • mitoxantrone • fludarabine IV • busulfan
2ms
Enrollment change
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FLT3 (Fms-related tyrosine kinase 3) • BCL2 (B-cell CLL/lymphoma 2)
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FLT3-ITD mutation
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Venclexta (venetoclax) • cytarabine • azacitidine • Mylotarg (gemtuzumab ozogamicin) • fludarabine IV
2ms
Concomitant Clonal CBFB::MYH11 and PDGFRB Fusions in a Case of De Novo Acute Myeloid Leukemia. (PubMed, Hematol Rep)
The patient achieved complete remission following the standard 7 + 3 induction chemotherapy regimen for AML with gemtuzumab ozogamicin. This case illustrates the diagnostic challenges posed by concomitant class-defining alterations in hematologic neoplasms and underscores the importance of integrated genomic assessment.
Journal
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PDGFRB (Platelet Derived Growth Factor Receptor Beta) • WT1 (WT1 Transcription Factor) • GOLGA4 (Golgin A4) • CDKN1A (Cyclin-dependent kinase inhibitor 1A)
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CBFB-MYH11 fusion
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Mylotarg (gemtuzumab ozogamicin)