Pemigatinib After Chemotherapy for the Treatment of Newly Diagnosed Acute Myeloid Leukemia
NCT ID: NCT04659616
Last Updated: 2025-12-19
Study Results
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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RECRUITING
PHASE1
32 participants
INTERVENTIONAL
2021-01-14
2026-12-31
Brief Summary
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Detailed Description
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I. To determine the maximum tolerated dose of pemigatinib (INC054828) following standard of care induction chemotherapy.
SECONDARY OBJECTIVES:
I. To assess preliminary efficacy associated with study intervention. II. To assess the safety profile of the study intervention. III. To evaluate time-to- marrow recovery between cycles of pemigatinib.
EXPLORATORY OBJECTIVES:
I. Assess minimal residual disease (MRD) by fluorescent in situ hybridization (FISH), reverse transcriptase-polymerase chain reaction (RT-PCR), and/or next-generation sequencing of acute myeloid leukemia (AML) genetic abnormalities in bone marrow and blood.
II. Quantify FGF2/FGFR immunohistochemical staining of marrow core biopsies and compare to historical controls at various timepoints.
III. Assess ex vivo sensitivity of patient-derived mononuclear cells to pemigatinib with and without FGF2 supplementation.
IV. Evaluate pemigatinib-induced changes in stromal expression using cultured bone marrow samples and compare to historical controls.
V. Evaluate the impact of pemigatinib on the need for intravenous (IV) phosphate replacement after chemotherapy.
OUTLINE: This is a dose-escalation study of pemigatinib followed by a dose-expansion study.
INDUCTION: Patients receive cytarabine IV on days 1-7, daunorubicin IV on days 1-3, and pemigatinib orally (PO) once daily (QD) on days 8-21 in the absence of disease progression or unacceptable toxicity. Patients with hematologic count recovery (assessed between days 25-42) after induction proceed to consolidation therapy. Patients undergo echocardiography (ECHO) during screening and as clinically indicated on study. Patients undergo blood sample collection and bone marrow aspirate and biopsy during screening and cycle 11 day 21 on study.
CONSOLIDATION: Patients receive high dose cytarabine IV twice daily (BID) on days 1, 3, and 5 of each cycle, and pemigatinib PO QD on days 8-21 of each cycle. Treatment repeats every 28 days for up to 4 cycles in the absence of disease progression of unacceptable toxicity. Patients undergo ECHO as clinically indicated and blood sample collection and bone marrow biopsy and aspirate at the end of consolidation.
After completion of study treatment, patients are followed up every 3 months for up to 24 months.
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Treatment (cytarabine, daunorubicin, pemigatinib)
INDUCTION: Patients receive cytarabine IV on days 1-7, daunorubicin IV on days 1-3, and pemigatinib PO QD on days 8-21 in the absence of disease progression or unacceptable toxicity. Patients with hematologic count recovery (assessed between days 25-42) after induction proceed to consolidation therapy. Patients undergo ECHO during screening and as clinically indicated on study. Patients undergo blood sample collection and bone marrow aspirate and biopsy during screening and cycle 11 day 21 on study.
CONSOLIDATION: Patients receive high dose cytarabine IV BID on days 1, 3, and 5 of each cycle, and pemigatinib PO QD on days 8-21 of each cycle. Treatment repeats every 28 days for up to 4 cycles in the absence of disease progression of unacceptable toxicity. Patients undergo ECHO as clinically indicated and blood sample collection and bone marrow biopsy and aspirate at the end of consolidation.
Biospecimen Collection
Undergo blood sample collection
Bone Marrow Aspirate
Undergo bone marrow biopsy and aspirate
Bone Marrow Biopsy
Undergo bone marrow biopsy and aspiratie
Cytarabine
Given IV
Daunorubicin
Given IV
Electrocardiography
Undergo ECHO
Pemigatinib
Given PO
Interventions
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Biospecimen Collection
Undergo blood sample collection
Bone Marrow Aspirate
Undergo bone marrow biopsy and aspirate
Bone Marrow Biopsy
Undergo bone marrow biopsy and aspiratie
Cytarabine
Given IV
Daunorubicin
Given IV
Electrocardiography
Undergo ECHO
Pemigatinib
Given PO
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Cytogenetic abnormalities not classified as favorable
* t(6;9)(p23;q34.1); DEK-NUP214
* t(v;11q23.3); KMT2A rearranged
* t(9;22)(q34.1;q11.2); BCR-ABL1
* inv(3)(q21.3q26.2) or t(3;3)(q21.3;q26.2); GATA2,MECOM(EVI1)
* -5 or del(5q); -7; -17/abn(17p)
* Complex karyotype - defined as three or more unrelated chromosome abnormalities in the absence of 1 of the WHO-designated recurring translocations or inversions, that is, t(8;21), inv(16) or t(16;16), t(9;11), t(v;11)(v;q23.3), t(6;9), inv(3) or t(3;3); AML with BCR-ABL1
* Monosomal karyotype - defined by the presence of 1 single monosomy (excluding loss of X or Y) in association with at least 1 additional monosomy or structural chromosome abnormality (excluding core-binding factor AML)
* Mutated RUNX1 (without favorable risk cytogenetics/mutations)
* Mutated BCOR (without favorable risk cytogenetics/mutations)
* Mutated EZH2 (without favorable risk cytogenetics/mutations)
* Mutated ASXL1 (without favorable risk cytogenetics/mutations)
* Mutated SF3B1 (without favorable risk cytogenetics/mutations)
* Mutated SRSF2 (without favorable risk cytogenetics/mutations)
* Mutated STAG2 (without favorable risk cytogenetics/mutations)
* Mutated U2AF1, (without favorable risk cytogenetics/mutations)
* Mutated ZRSR2 (without favorable risk cytogenetics/mutations)
* Mutated TP53 (mono- or biallelic) at a variant allele fraction of at least 10%
Exclusion Criteria
* Individuals positive for hepatitis B core antibody who are receiving intravenous immunoglobulin (IVIg) are eligible if HepB PCR is negative
* A screening QT interval by Fridericia's Correction Formula (QTcF) interval \> 480 ms will result in exclusion.
* For participants with an intraventricular conduction delay (QRS interval \> 120 ms), the JTc interval may be used in place of the QTc with approval from Sponsor-Investigator. The JTc must be =\< 340 ms if JTc is used in place of the QTc.
* Use of CYP3A4 inhibitors should be avoided but, if medically necessary, is permitted with a dose reduction of study drug
* Use of moderate CYP3A4 inhibitors are permitted.
* Based on the low overall bioavailability of topical ketoconazole, there are no restrictions on topical ketoconazole
18 Years
ALL
No
Sponsors
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Incyte Corporation
INDUSTRY
Oregon Health and Science University
OTHER
OHSU Knight Cancer Institute
OTHER
Responsible Party
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Elie Traer, MD PhD
Principal Investigator
Principal Investigators
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Elie Traer, MD PhD
Role: PRINCIPAL_INVESTIGATOR
OHSU Knight Cancer Institute
Locations
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OHSU Knight Cancer Institute
Portland, Oregon, United States
UT Southwestern/Simmons Cancer Center-Dallas
Dallas, Texas, United States
Countries
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Central Contacts
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OHSU Knight Cancer Institute Clinical Trials Information
Role: CONTACT
Phone: 503-494-1080
Email: [email protected]
Facility Contacts
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Elie Traer, MD PhD
Role: primary
Yazan Madanat, MD
Role: primary
References
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Al-Bazaz M, Forstreuter A, Hammada I, Hille J, Wagner JN, Reinert J, Wehrhahn J, Bokemeyer C, Fiedler W. Acute Lymphoblastic Leukemia Characterized by Rare BCR::FGFR1 Translocation: A Case Report With Literature Review. Case Rep Hematol. 2025 Oct 23;2025:8892036. doi: 10.1155/crh/8892036. eCollection 2025.
Other Identifiers
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NCI-2020-05267
Identifier Type: REGISTRY
Identifier Source: secondary_id
STUDY00020009
Identifier Type: OTHER
Identifier Source: secondary_id
STUDY00020009
Identifier Type: -
Identifier Source: org_study_id