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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04982354




Registration number
NCT04982354
Ethics application status
Date submitted
13/07/2021
Date registered
29/07/2021
Date last updated
1/10/2024

Titles & IDs
Public title
Induction Therapy for Patients With FLT3 Mutated Acute Myeloid Leukemia
Scientific 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
Secondary ID [1] 0 0
2019-KOE-003
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Acute Myeloid Leukemia 0 0
Condition category
Condition code
Cancer 0 0 0 0
Leukaemia - Acute leukaemia
Cancer 0 0 0 0
Leukaemia - Chronic leukaemia
Cancer 0 0 0 0
Children's - Leukaemia & Lymphoma

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - CPX-351
Treatment: Drugs - Midostaurin
Treatment: Drugs - Busulfan
Treatment: Drugs - Melphalan
Treatment: Drugs - Fludarabine
Treatment: Other - CD34+ selected allogeneic stem cell transplant from an HLA-compatible donor

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.


Treatment: Drugs: 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.

Treatment: Drugs: 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.

Treatment: Drugs: Busulfan
0.8 mg/kg/dose every six hours x 12 doses administered intravenously

Treatment: Drugs: Melphalan
70 mg/m2/day x 2 doses administered intravenously

Treatment: Drugs: Fludarabine
25 mg/m2/day x 5 doses administered intravenously

Treatment: Other: CD34+ selected allogeneic stem cell transplant from an HLA-compatible donor
Allogeneic stem cell transplant infused intravenously
Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change in the complete remission rate
Timepoint [1] 0 0
3, 6, 12 and 24 months
Primary outcome [2] 0 0
Change in Progression Free Survival (PFS)
Timepoint [2] 0 0
3, 6, 12 and 24 months
Primary outcome [3] 0 0
Change in Overall Survival (OS)
Timepoint [3] 0 0
3, 6, 12 and 24 months
Secondary outcome [1] 0 0
Change in the rate of Minimal Residual Disease (MRD) negativity
Timepoint [1] 0 0
3, 6, 12 and 24 months
Secondary outcome [2] 0 0
Correlation of Minimal Residual Disease (MRD)
Timepoint [2] 0 0
3, 6, 12 and 24 months

Eligibility
Key inclusion criteria
* Patients must have a Karnofsky (adult) Performance Status of at least 70%.
* Patients must have adequate organ function
Minimum age
18 Years
Maximum age
74 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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).

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Withdrawn
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
5/07/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/08/2032
Actual
Sample size
Target
Accrual to date
Final
0
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Florida

Funding & Sponsors
Primary sponsor type
Other
Name
Guenther Koehne
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Jazz Pharmaceuticals
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/study/NCT04982354
Trial related presentations / publications
Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed

Contacts
Principal investigator
Name 0 0
Guenther Koehne, MD. PhD
Address 0 0
Miami Cancer Institute at Baptist Health of South Florida
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT04982354