Pemigatinib After Chemotherapy for the Treatment of Newly Diagnosed Acute Myeloid Leukemia

NCT04659616 | Recruiting Phase 1 DMC FDA Drug

• 2 other identifiers: STUDY00020009NCI-2020-05267
• Study Type: interventional
• Target: 32
• Locations: 1 country
• Sites: 2

Brief Summary

This phase I trial identifies the best dose and clinical benefit of giving pemigatinib following standard induction chemotherapy in patients with newly diagnosed acute myeloid leukemia. Pemigatinib selectively inhibits FGFR (fibroblast growth factor receptor) activity, a receptor that may contribute to the growth of leukemia cells. The genetic changes responsible for activating the growth of leukemia cells can be unique to each patient and can change during the course of the disease. Chemotherapy drugs, such as cytarabine and daunorubicin work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.

Conditions

Acute Myeloid Leukemia

Outcome Measures

Primary Outcomes (1)

• Incidence of dose limiting toxicities (DLTs)

  Will be summarized using the proportion and exact binomial confidence interval. DLTs will be summarized at each dose level by severity and major organ site according to the Common Terminology Criteria for Adverse Events (CTCAE) version (v)5.0.

  From cycle 1 day 8, until 14 days after cycle 1 day 28 (up to 42 days)

Secondary Outcomes (8)

• Rate of composite complete remission (cCR)

  From cycle 1 day 1, until blood cell count recovery after induction (day 25-42)

• Duration of remission (DOR)

  Date of first response (>= immune complete remission) till date of documented relapse assessed up to 2 years from last dose of study intervention

• Event-free survival (EFS)

  From cycle 1 day 1, until date of primary refractory disease, progression, relapse, or death from any cause, assessed up to 2 years from last dose of study intervention

• Relapse-free survival (RFS)

  Date of first response (>= complete remission with incomplete blood count recovery [CRi]), until date of relapse or death from any cause, assessed up to 2 years from last dose of study intervention

• Overall survival (OS)

  From cycle 1 day 1, until date of death from any cause, assessed up to 2 years from last dose of study intervention

Study Arms (1)

Treatment (cytarabine, daunorubicin, pemigatinib)

EXPERIMENTAL

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.

Procedure: Biospecimen Collection; Procedure: Bone Marrow Aspirate; Procedure: Bone Marrow Biopsy; Drug: Cytarabine; Drug: Daunorubicin; Procedure: Electrocardiography; Drug: Pemigatinib

Interventions

Biospecimen Collection PROCEDURE

Undergo blood sample collection

Also known as: Biological Sample Collection, Biospecimen Collected, Specimen Collection

Treatment (cytarabine, daunorubicin, pemigatinib)

Bone Marrow Aspirate PROCEDURE

Undergo bone marrow biopsy and aspirate

Also known as: BONE MARROW, LIQUID, Human Bone Marrow Aspirate

Treatment (cytarabine, daunorubicin, pemigatinib)

Pemigatinib DRUG

Given PO

Also known as: INCB054828, Pemazyre

Treatment (cytarabine, daunorubicin, pemigatinib)

Bone Marrow Biopsy PROCEDURE

Undergo bone marrow biopsy and aspiratie

Also known as: Biopsy of Bone Marrow, Biopsy, Bone Marrow

Treatment (cytarabine, daunorubicin, pemigatinib)

Cytarabine DRUG

Given IV

Also known as: .beta.-Cytosine arabinoside, 1-.beta.-D-Arabinofuranosyl-4-amino-2(1H)pyrimidinone, 1-.beta.-D-Arabinofuranosylcytosine, 1-Beta-D-arabinofuranosyl-4-amino-2(1H)pyrimidinone, 1-Beta-D-arabinofuranosylcytosine, 1.beta.-D-Arabinofuranosylcytosine, 2(1H)-Pyrimidinone, 4-Amino-1-beta-D-arabinofuranosyl-, 2(1H)-Pyrimidinone, 4-amino-1.beta.-D-arabinofuranosyl-, Alexan, Ara-C, ARA-cell, Arabine, Arabinofuranosylcytosine, Arabinosylcytosine, Aracytidine, Aracytin, Aracytine, Beta-Cytosine Arabinoside, CHX-3311, Cytarabinum, Cytarbel, Cytosar, Cytosine Arabinoside, Cytosine-.beta.-arabinoside, Cytosine-beta-arabinoside, Erpalfa, Starasid, Tarabine PFS, U 19920, U-19920, Udicil, WR-28453

Treatment (cytarabine, daunorubicin, pemigatinib)

Daunorubicin DRUG

Given IV

Also known as: Daunomycin, Daunorrubicina, DNR, Leukaemomycin C, Rubidomycin, Rubomycin C

Treatment (cytarabine, daunorubicin, pemigatinib)

Electrocardiography PROCEDURE

Undergo ECHO

Also known as: ECG, EKG

Treatment (cytarabine, daunorubicin, pemigatinib)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• t(9;11)(p21.3;q23.3); MLLT3-KMT2A
• 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)
• 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)

You may not qualify if:

• Except for commonly observed calcifications in soft tissues such as the skin, kidney tendon, or vessels due to injury, disease, or aging in the absence of systemic mineral imbalance)
• Individuals positive for hepatitis B core antibody who are receiving intravenous immunoglobulin (IVIg) are eligible if HepB PCR is negative
• 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

Sponsors & Collaborators

• OHSU Knight Cancer Institute: lead
• Incyte Corporation: collaborator
• Oregon Health and Science University: collaborator

Study Sites (2)

OHSU Knight Cancer Institute

Portland, Oregon, 97239, United States

RECRUITING

UT Southwestern/Simmons Cancer Center-Dallas

Dallas, Texas, 75390, United States

RECRUITING

Related Publications (1)

• 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.

  PMID: 41180818 DERIVED

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Interventions

Specimen Handling; Fluid Therapy; Biopsy; Cytarabine; Daunorubicin; pemigatinib

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Investigative Techniques; Drug Therapy; Therapeutics; Cytodiagnosis; Cytological Techniques; Diagnostic Techniques, Surgical; Surgical Procedures, Operative; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Arabinonucleosides; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Anthracyclines; Naphthacenes; Polycyclic Aromatic Hydrocarbons; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Organic Chemicals; Polycyclic Compounds; Aminoglycosides; Glycosides; Carbohydrates

Study Officials

• Elie Traer, MD PhD

  OHSU Knight Cancer Institute

  PRINCIPAL INVESTIGATOR

Central Study Contacts

OHSU Knight Cancer Institute Clinical Trials Information

CONTACT

503-494-1080; trials@ohsu.edu

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: August 20, 2020
• First Posted: December 9, 2020
• Study Start: January 14, 2021
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: December 19, 2025

Record last verified: 2025-12

Locations

US (2)