Acute Myeloid LeukemiaPhase 1/2NCT04982354
Induction Therapy for Patients With FLT3 Mutated Acute Myeloid Leukemia
A Phase 1/2 study testing CPX-351, Midostaurin and Busulfan in people with Acute Myeloid Leukemia, run by Guenther Koehne at 1 site in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 74 years
- Conditions the study is for
- Acute Myeloid Leukemia
- What is being tested?
- CPX-351, Midostaurin and Busulfan
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 5 Jul 2022to 1 Aug 2032
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 74 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients must have a Karnofsky (adult) Performance Status of at least 70%.
- Patients must have adequate organ function
Who cannot join
- 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).
Trial locations and contacts
1 site in 1 country.
United States1 site
Miami Cancer Institute at Baptist Health of South Florida
Miami, Florida, 33176
About this study
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
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.
- Conditions
- Acute Myeloid Leukemia
- Being tested
- CPX-351, Midostaurin, Busulfan, Melphalan, Fludarabine
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CPX-351Drug
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.
MidostaurinDrug
Also called Rydapt
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.
BusulfanDrug
Also called Myleran
0.8 mg/kg/dose every six hours x 12 doses administered intravenously
MelphalanDrug
Also called Alkeran
70 mg/m2/day x 2 doses administered intravenously
FludarabineDrug
Also called Fludara
25 mg/m2/day x 5 doses administered intravenously
CD34+ selected allogeneic stem cell transplant from an HLA-compatible donorBiological
Allogeneic stem cell transplant infused intravenously
| Study group | Role | What they receive |
|---|---|---|
| Investigational Treatment | Experimental | Daunorubicin-cytarabine liposome (CPX-351) Plus FLT3-inhibitor (Midostaurin) Induction Therapy followed by Busulfan/Melphalan/Fludarabine Conditioning therapy and CD34+-selected allografts. Receives: Drug: CPX-351; Drug: Midostaurin; Drug: Busulfan; Drug: Melphalan; Drug: Fludarabine; Biological: CD34+ selected allogeneic stem cell transplant from an HLA-compatible donor |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in the complete remission rate
Assess the complete remission rate following induction therapy with CPX-351 plus midostaurin when administered to patients
Measured: 3, 6, 12 and 24 months
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.
Measured: 3, 6, 12 and 24 months
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.
Measured: 3, 6, 12 and 24 months
Secondary outcomes (2)
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)
Measured: 3, 6, 12 and 24 months
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.
Measured: 3, 6, 12 and 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jul 2021
- Study start5 Jul 2022
- Primary completion1 Aug 2031 (estimated)
- Study completion1 Aug 2032 (estimated)
Why it stopped: Could not accrue
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Guenther Koehne
- Sponsor type
- University, hospital or other
- Collaborators
- Jazz Pharmaceuticals
- Responsible party
- Sponsor-investigator, Guenther Koehne, Baptist Health South Florida
- Investigators
- Guenther Koehne, MD. PhD, Principal investigator, Miami Cancer Institute at Baptist Health of South Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04982354
- Sponsor's protocol ID
- 2019-KOE-003
- Status last verified
- Sep 2024
- First submitted
- 13 Jul 2021
- Last updated
- 1 Oct 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04982354
- Acronym / sponsor ID
- 2019-KOE-003
- Conditions
- Acute Myeloid Leukemia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Could not accrue
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Guenther KoehneUniversity, hospital or other
- Collaborators
- Jazz Pharmaceuticals
- Study start
- 5 Jul 2022 (actual)
- Primary completion
- 1 Aug 2031 (estimated)
- Study completion
- 1 Aug 2032 (estimated)
- First posted
- 29 Jul 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Investigational Treatment | Experimental | Drug: CPX-351, Drug: Midostaurin, Drug: Busulfan, Drug: Melphalan, Drug: Fludarabine, Biological: CD34+ selected allogeneic stem cell transplant from an HLA-compatible donorDaunorubicin-cytarabine liposome (CPX-351) Plus FLT3-inhibitor (Midostaurin) Induction Therapy followed by Busulfan/Melphalan/Fludarabine Conditioning therapy and CD34+-selected allografts. |
Who may qualify
- Ages
- 18 to 74 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Patients must have a Karnofsky (adult) Performance Status of at least 70%.
- Patients must have adequate organ function
Key exclusion criteria (4 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CPX-351For 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 | Investigational Treatment |
| MidostaurinAlso: RydaptThe 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. | Drug | Investigational Treatment |
| BusulfanAlso: Myleran0.8 mg/kg/dose every six hours x 12 doses administered intravenously | Drug | Investigational Treatment |
| MelphalanAlso: Alkeran70 mg/m2/day x 2 doses administered intravenously | Drug | Investigational Treatment |
| FludarabineAlso: Fludara25 mg/m2/day x 5 doses administered intravenously | Drug | Investigational Treatment |
| CD34+ selected allogeneic stem cell transplant from an HLA-compatible donorAllogeneic stem cell transplant infused intravenously | Biological | Investigational Treatment |
Primary outcome
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
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.
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.
2 secondary outcomes
Change in the rate of Minimal Residual Disease (MRD) negativity
Time frame: 3, 6, 12 and 24 months
Correlation of Minimal Residual Disease (MRD)
Time frame: 3, 6, 12 and 24 months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Oct 2024.
- Miami Cancer Institute WebsiteLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Guenther Koehne, MD. PhD principal investigator
Miami Cancer Institute at Baptist Health of South Florida
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing CPX-351, Midostaurin and Busulfan in people with Acute Myeloid Leukemia, run by Guenther Koehne at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 1 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in the complete remission rate (time frame: 3, 6, 12 and 24 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Oct 2024.