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Induction Therapy for Patients With FLT3 Mutated Acute Myeloid Leukemia

Primary Purpose

Acute Myeloid Leukemia

Status
Recruiting
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
CPX-351
Midostaurin
Busulfan
Melphalan
Fludarabine
CD34+ selected allogeneic stem cell transplant from an HLA-compatible donor
Sponsored by
Baptist Health South Florida
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Acute Myeloid Leukemia

Eligibility Criteria

18 Years - 74 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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).

Sites / Locations

  • Miami Cancer Institute at Baptist Health of South FloridaRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Investigational Treatment

Arm Description

Daunorubicin-cytarabine liposome (CPX-351) Plus FLT3-inhibitor (Midostaurin) Induction Therapy followed by Busulfan/Melphalan/Fludarabine Conditioning therapy and CD34+-selected allografts.

Outcomes

Primary Outcome Measures

Change in the complete remission rate
Assess the complete remission rate following induction therapy with CPX-351 plus midostaurin when administered to patients
Change in Progression Free Survival (PFS)
to determine the PFS of these patients following allo SCT. To estimate PFS the Kaplan-Meier method will be used.
Change in Overall Survival (OS)
to determine the OS of these patients following allo SCT. To estimate OS the Kaplan-Meier method will be used.

Secondary Outcome Measures

Change in the rate of Minimal Residual Disease (MRD) negativity
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)
Correlation of Minimal Residual Disease (MRD)
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.

Full Information

First Posted
July 13, 2021
Last Updated
May 1, 2023
Sponsor
Baptist Health South Florida
Collaborators
Jazz Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT04982354
Brief Title
Induction Therapy for Patients With FLT3 Mutated Acute Myeloid Leukemia
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
Study Type
Interventional

2. Study Status

Record Verification Date
May 2023
Overall Recruitment Status
Recruiting
Study Start Date
July 5, 2022 (Actual)
Primary Completion Date
August 1, 2031 (Anticipated)
Study Completion Date
August 1, 2032 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Baptist Health South Florida
Collaborators
Jazz Pharmaceuticals

4. Oversight

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
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Acute Myeloid Leukemia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
20 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Investigational Treatment
Arm Type
Experimental
Arm Description
Daunorubicin-cytarabine liposome (CPX-351) Plus FLT3-inhibitor (Midostaurin) Induction Therapy followed by Busulfan/Melphalan/Fludarabine Conditioning therapy and CD34+-selected allografts.
Intervention Type
Drug
Intervention Name(s)
CPX-351
Intervention Description
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.
Intervention Type
Drug
Intervention Name(s)
Midostaurin
Other Intervention Name(s)
Rydapt
Intervention Description
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.
Intervention Type
Drug
Intervention Name(s)
Busulfan
Other Intervention Name(s)
Myleran
Intervention Description
0.8 mg/kg/dose every six hours x 12 doses administered intravenously
Intervention Type
Drug
Intervention Name(s)
Melphalan
Other Intervention Name(s)
Alkeran
Intervention Description
70 mg/m2/day x 2 doses administered intravenously
Intervention Type
Drug
Intervention Name(s)
Fludarabine
Other Intervention Name(s)
Fludara
Intervention Description
25 mg/m2/day x 5 doses administered intravenously
Intervention Type
Biological
Intervention Name(s)
CD34+ selected allogeneic stem cell transplant from an HLA-compatible donor
Intervention Description
Allogeneic stem cell transplant infused intravenously
Primary Outcome Measure Information:
Title
Change in the complete remission rate
Description
Assess the complete remission rate following induction therapy with CPX-351 plus midostaurin when administered to patients
Time Frame
3, 6, 12 and 24 months
Title
Change in Progression Free Survival (PFS)
Description
to determine the PFS of these patients following allo SCT. To estimate PFS the Kaplan-Meier method will be used.
Time Frame
3, 6, 12 and 24 months
Title
Change in Overall Survival (OS)
Description
to determine the OS of these patients following allo SCT. To estimate OS the Kaplan-Meier method will be used.
Time Frame
3, 6, 12 and 24 months
Secondary Outcome Measure Information:
Title
Change in the rate of Minimal Residual Disease (MRD) negativity
Description
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)
Time Frame
3, 6, 12 and 24 months
Title
Correlation of Minimal Residual Disease (MRD)
Description
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.
Time Frame
3, 6, 12 and 24 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
74 Years
Accepts Healthy Volunteers
No
Eligibility 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).
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Guenther Koehne, MD, PhD
Phone
786-596-2000
Email
GuentherK@Baptisthealth.net
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Guenther Koehne, MD. PhD
Organizational Affiliation
Miami Cancer Institute at Baptist Health of South Florida
Official's Role
Principal Investigator
Facility Information:
Facility Name
Miami Cancer Institute at Baptist Health of South Florida
City
Miami
State/Province
Florida
ZIP/Postal Code
33176
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Guenther Koehne, MD, PhD
Phone
786-596-2000
Email
GuentherK@Baptisthealth.net
First Name & Middle Initial & Last Name & Degree
Guenther Koehne, MD, PhD

12. IPD Sharing Statement

Links:
URL
http://baptisthealth.net/cancer-care/home
Description
Miami Cancer Institute Website

Learn more about this trial

Induction Therapy for Patients With FLT3 Mutated Acute Myeloid Leukemia

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