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Induction Therapy for Patients With FLT3 Mutated Acute Myeloid Leukemia

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ClinicalTrials.gov Identifier: NCT04982354
Recruitment Status : Recruiting
First Posted : July 29, 2021
Last Update Posted : April 9, 2024
Sponsor:
Collaborator:
Jazz Pharmaceuticals
Information provided by (Responsible Party):
Guenther Koehne, Baptist Health South Florida

Brief Summary:
This is a pilot study designed to identify the effect of daunorubicin-cytarabine liposome (CPX-351) in combination with a FLT3-inhibitor (midostaurin) as induction and consolidation therapy for patients with high-risk FLT3 mutated acute myeloid leukemia (AML) and subsequent CD34+-selected allogeneic stem cell transplant from HLA compatible related or unrelated donors.

Condition or disease Intervention/treatment Phase
Acute Myeloid Leukemia Drug: CPX-351 Drug: Midostaurin Drug: Busulfan Drug: Melphalan Drug: Fludarabine Biological: CD34+ selected allogeneic stem cell transplant from an HLA-compatible donor Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Pilot Study of Daunorubicin-cytarabine Liposome (CPX-351) Plus FLT3-inhibitor (Midostaurin) as Induction Therapy for Patients With FLT3 Mutated Acute Myeloid Leukemia Followed by Consolidation With a CD34+-Selected Allograft
Actual Study Start Date : July 5, 2022
Estimated Primary Completion Date : August 1, 2031
Estimated Study Completion Date : August 1, 2032


Arm Intervention/treatment
Experimental: Investigational Treatment
Daunorubicin-cytarabine liposome (CPX-351) Plus FLT3-inhibitor (Midostaurin) Induction Therapy followed by Busulfan/Melphalan/Fludarabine Conditioning therapy and CD34+-selected allografts.
Drug: CPX-351
For this trial, patients will be treated with CPX-351 100 (daunorubicin 44 mg/m2 and cytarabine 100 mg/m2) for 3 doses on days 1, 3 and 5 of one and on days 1 + 3 of a second cycle of induction therapy, depending on response obtained following the first induction. Thereafter, up to 2 cycles of consolidation therapy of 2 doses on days 1 and 3 of daunorubicin 29 mg/m2 and cytarabine 65 mg/m2 will be administered to the patients.

Drug: Midostaurin
The FLT3 directed inhibitor, midostaurin, will be given at a dose of 50mg twice daily, starting on day 8 through day 21 of each cycle of CPX-351 until admission for allogeneic stem cell transplant.
Other Name: Rydapt

Drug: Busulfan
0.8 mg/kg/dose every six hours x 12 doses administered intravenously
Other Name: Myleran

Drug: Melphalan
70 mg/m2/day x 2 doses administered intravenously
Other Name: Alkeran

Drug: Fludarabine
25 mg/m2/day x 5 doses administered intravenously
Other Name: Fludara

Biological: CD34+ selected allogeneic stem cell transplant from an HLA-compatible donor
Allogeneic stem cell transplant infused intravenously




Primary Outcome Measures :
  1. Change in the complete remission rate [ Time Frame: 3, 6, 12 and 24 months ]
    Assess the complete remission rate following induction therapy with CPX-351 plus midostaurin when administered to patients

  2. Change in Progression Free Survival (PFS) [ Time Frame: 3, 6, 12 and 24 months ]
    to determine the PFS of these patients following allo SCT. To estimate PFS the Kaplan-Meier method will be used.

  3. Change in Overall Survival (OS) [ Time Frame: 3, 6, 12 and 24 months ]
    to determine the OS of these patients following allo SCT. To estimate OS the Kaplan-Meier method will be used.


Secondary Outcome Measures :
  1. Change in the rate of Minimal Residual Disease (MRD) negativity [ Time Frame: 3, 6, 12 and 24 months ]
    Ascertain the rate of MRD negativity by next generation sequencing at sequential time post following induction treatment at complete remission prior to allo Stem Cell Transplantation (SCT)

  2. Correlation of Minimal Residual Disease (MRD) [ Time Frame: 3, 6, 12 and 24 months ]
    Correlation of duration of MRD negative status with duration of complete remission of these patients will be assessed using Spearman's correlation with reported p value.



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Ages Eligible for Study:   18 Years to 74 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patients must have a Karnofsky (adult) Performance Status of at least 70%.
  • Patients must have adequate organ function

Exclusion Criteria:

  • Female patients who are pregnant or breast-feeding
  • Active viral, bacterial or fungal infection
  • Patient seropositive for Human Immunodeficiency Virus (HIV-I /II); Human T-Cell Lymphotrophic Virus (HTLV -I /II)
  • Presence of leukemia in the Central Nervous System (CNS).

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04982354


Contacts
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Contact: Guenther Koehne, MD, PhD 786-596-2000 GuentherK@Baptisthealth.net

Locations
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United States, Florida
Miami Cancer Institute at Baptist Health of South Florida Recruiting
Miami, Florida, United States, 33176
Contact: Guenther Koehne, MD, PhD    786-596-2000    GuentherK@Baptisthealth.net   
Principal Investigator: Guenther Koehne, MD, PhD         
Sponsors and Collaborators
Guenther Koehne
Jazz Pharmaceuticals
Investigators
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Principal Investigator: Guenther Koehne, MD. PhD Miami Cancer Institute at Baptist Health of South Florida
Additional Information:
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Responsible Party: Guenther Koehne, Deputy Director and Chief of Blood and Marrow Transplant, Hematologic Oncology and Benign Hematology, Baptist Health South Florida
ClinicalTrials.gov Identifier: NCT04982354    
Other Study ID Numbers: 2019-KOE-003
First Posted: July 29, 2021    Key Record Dates
Last Update Posted: April 9, 2024
Last Verified: April 2024

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Additional relevant MeSH terms:
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Leukemia
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Neoplasms by Histologic Type
Neoplasms
Hematologic Diseases
Fludarabine
Melphalan
Busulfan
Midostaurin
Antineoplastic Agents
Molecular Mechanisms of Pharmacological Action
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antineoplastic Agents, Alkylating
Alkylating Agents
Myeloablative Agonists
Protein Kinase Inhibitors
Enzyme Inhibitors