Acute Myeloid LeukemiaPhase 2NCT03207334
iCare4: Genomic Signatures With Midostaurin in Acute Myeloid Leukemia (UF-HEM-004)
A Phase 2 study testing Midostaurin and Cytarabine in people with Acute Myeloid Leukemia, run by University of Florida at 1 site in United States.
Trial summary
This is an open label, single arm study of midostaurin in patients with relapsed or refractory AML. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Acute Myeloid Leukemia
- What is being tested?
- Midostaurin and Cytarabine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2018to Jan 2022
- Phase
- Phase 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 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Written informed consent obtained from the subject prior to registration on the study and the ability for the subject to adhere to the study visit schedule and all the study-related procedures.
- A diagnosis of relapsed or refractory AML. For the purpose of this study, refractory AML is defined as failure to achieve CR after one cycle of induction chemotherapy and relapsed AML is defined as any evidence of disease recurrence after achieving CR of any duration.
- Both males and females ≥ eighteen years of age
- Karnofsky Performance Status of ≥ 70% or Eastern Cooperative Oncology Group (ECOG) performance status < 2 (unless poor performance status is related to the disease).
- Adequate organ function defined as:
- AST and ALT < 2.5 times the upper limit of normal (ULN),
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- Serum bilirubin < 2.5 x ULN, and
- Serum creatinine < 1.5 mg/dL or creatinine clearance > 50 mL/min.
- Laboratory values can be outside of this range if secondary to AML disease.
- An ejection fraction of > 45% confirmed by echocardiogram.
- Life expectancy of greater than one month.
- Subjects who previously received midostaurin are allowed if the last dose of midostaurin was given ≥ six months prior to disease relapse.
- Females who are non-pregnant and non-nursing.
- Females of reproductive age and males must agree to avoid getting pregnant or to father a child while on therapy and for five months after the last dose of chemotherapy.
- Women of child-bearing potential (WOCBP) must either agree to continued abstinence from heterosexual intercourse or begin one of the following acceptable methods of birth control: IUD, tubal ligation, or partner must use a latex condom during any sexual contact, even if the partner has undergone a successful vasectomy. Hormonal contraception is an inadequate method of birth control.
- Males must use a latex condom during any sexual contact with WOCBP, even if they have undergone a successful vasectomy (while on therapy and for five months after the last dose of chemotherapy)
Who cannot join
- Age ≥ of seventy-six years.
- Subjects with the inability to swallow oral medications.
- Clinical evidence of active CNS leukemia.
- A medical history of receiving an allogeneic hematopoietic stem cell transplantation (HSCT).
- Subjects must not have any uncontrolled or intercurrent illness including, but not limited to,
- ongoing or active infection,
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- symptomatic congestive heart failure,
- unstable angina pectoris,
- cardiac arrhythmia,
- myocardial infarction within three months,
- poorly controlled hypertension,
- uncontrolled diabetes, or psychiatric illness/social situation that would limit compliance with protocol requirements.
- Females or males of childbearing potential who are unwilling or unable to use an acceptable method to avoid pregnancy for the entire study period and for at least five months after the last dose of chemotherapy.
- Impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of PKC412.
- Previous therapy with midostaurin within six months prior to relapse.
- History of any other disease, metabolic dysfunction, physical examination finding, or clinical laboratory finding giving reasonable suspicion of a disease or condition that contraindicates the use of protocol therapy or that might affect the interpretation of the results of the study or that puts the subject at high risk for treatment complications, in the opinion of the treating physician.
- Prisoners or subjects who are involuntarily incarcerated.
- Subjects who are compulsorily detained for treatment of either a psychiatric or physical illness.
- Subjects demonstrating an inability to comply with the study and/or follow-up procedures.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase II, Open-Label Clinical Efficacy Study Defining Genomic Signatures That Correlate With Midostaurin Response in Relapsed or Refractory Acute Myeloid Leukemia (AML)
This is an open label, single arm study of midostaurin in patients with relapsed or refractory AML.
Read the detailed description
Clinical trials have shown that the multi-kinase inhibitor midostaurin improves survival in patients with AML. The main objective of this study is to investigate its effect (in conjunction with cytarabine) on progression free survival, as well as overall survival and morphologic response rate in patients with relapsed or refractory AML. This study will also attempt to identify specific populations of patients with relapsed or refractory AML who best respond to treatment with midostaurin to enhance treatment planning.
- Conditions
- Acute Myeloid Leukemia
- Being tested
- Midostaurin, Cytarabine
- Keywords
- acute myeloid leukemia, genomic signatures, midostaurin, relapsed, refractory, PKC412
- Medical subject headings
- Leukemia, Myeloid, Acute, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MidostaurinDrug
Also called PKC412, Rydapt
Induction Phase: 50 mg orally twice daily beginning on day 7 and ending 48 hours prior to beginning the consolidation phase
Consolidation Phase: 50 mg orally twice daily beginning on Day 6 and ending 48 hours prior to either the start of the next cycle or beginning conditioning therapy for allogenic stem cell transplant.
CytarabineDrug
Also called Depocyt
Induction Phase: 3 g/m2 (patients 18-65 years old) or 1 g/m2 (patients > 65 years old or those with co-morbidities precluding a higher dose) by vein over 3 hours every 12 hours on Days 1-6.
Consolidation Phase: 3 g/m2 (patients 18-65 years old) or 1 g/m2 (patients > 65 years old or those with co-morbidities precluding a higher dose) by vein over 3 hours every 12 hours on Days 1, 3, and 5 of each cycle.
| Study group | Role | What they receive |
|---|---|---|
| Midostaurin and Cytarabine | Experimental | Treatment will consist of 3 phases: induction (a 28-42 day cycle), followed by consolidation (up to 4 cycles). Each cycle in the consolidation phase will last 28 days. Subjects will proceed from one phase to the next if they have achieved or maintained a complete remission or complete remission with incomplete blood count recovery by International Working Group 2003 response criteria. Patients may receive a allogeneic hematopoietic stem cell transplant between the end of the induction phase. Receives: Drug: Midostaurin; Drug: Cytarabine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Progression Free Survival
Compare the progression free survival of patients receiving midostaurin and cytarabine to that historically achieved in patients receiving cytarabine alone
Measured: Up to 6 months
Secondary outcomes (3)
Number of participants with treatment-related adverse events as assessed by National Cancer Institute (NCI) Common Terminology for Adverse Events(CTCAE) v4.0
Assess the incidence and severity of toxicities of midostaurin and cytarabine using CTCAE v4.0
Measured: Up to 2.5 years
Overall survival
Compare the overall survival of patients receiving midostaurin and cytarabine to that historically achieved in patients receiving cytarabine alone
Measured: Up to 3.5 years
Morphologic response rate
Compare the morphologic response rate (using International Working Group 2003 criteria) of patients receiving midostaurin and cytarabine to that historically achieved in patients receiving cytarabine alone
Measured: Up to 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Jul 2017
- Study startNov 2018 (estimated)
- Primary completionJan 2021 (estimated)
- Study completionJan 2022 (estimated)
Why it stopped: Insufficient funding
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.
- More trials run by University of FloridaThe sponsor's other registered studies.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- All patients will receive midostaurin and cytarabine.
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Florida
- Sponsor type
- University, hospital or other
- Collaborators
- Novartis Pharmaceuticals
- Responsible party
- Sponsor
- Investigators
- Maxim Norkin, MD, PhD, Principal investigator, University of Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03207334
- Sponsor's protocol ID
- UF-HEM-004
- Other IDs
- CPKC412AUS61T, IRB201701934
- Status last verified
- Oct 2018
- First submitted
- 26 Jun 2017
- Last updated
- 22 Oct 2018
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Oct 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Open-Label Clinical Efficacy Study Defining Genomic Signatures That Correlate With Midostaurin Response in Relapsed or Refractory Acute Myeloid Leukemia (AML)
- ClinicalTrials.gov ID
- NCT03207334
- Acronym / sponsor ID
- UF-HEM-004
- Conditions
- Acute Myeloid Leukemia
- Keywords
- acute myeloid leukemia, genomic signatures, midostaurin, relapsed, refractory, PKC412
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Insufficient funding
- Enrolment
- 0 participants (actual)
- Lead sponsor
- University of FloridaUniversity, hospital or other
- Collaborators
- Novartis Pharmaceuticals
- Study start
- Nov 2018 (estimated)
- Primary completion
- Jan 2021 (estimated)
- Study completion
- Jan 2022 (estimated)
- First posted
- 2 Jul 2017 (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 an open label, single arm study of midostaurin in patients with relapsed or refractory AML.
Detailed description
Clinical trials have shown that the multi-kinase inhibitor midostaurin improves survival in patients with AML. The main objective of this study is to investigate its effect (in conjunction with cytarabine) on progression free survival, as well as overall survival and morphologic response rate in patients with relapsed or refractory AML. This study will also attempt to identify specific populations of patients with relapsed or refractory AML who best respond to treatment with midostaurin to enhance treatment planning.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- All patients will receive midostaurin and cytarabine.
| Arm | Type | Receives |
|---|---|---|
| Midostaurin and Cytarabine | Experimental | Drug: Midostaurin, Drug: CytarabineTreatment will consist of 3 phases: induction (a 28-42 day cycle), followed by consolidation (up to 4 cycles). Each cycle in the consolidation phase will last 28 days. Subjects will proceed from one phase to the next if they have achieved or maintained a complete remission or complete remission with incomplete blood count recovery by International Working Group 2003 response criteria. Patients may receive a allogeneic hematopoietic stem cell transplant between the end of the induction phase. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- Written informed consent obtained from the subject prior to registration on the study and the ability for the subject to adhere to the study visit schedule and all the study-related procedures.
- A diagnosis of relapsed or refractory AML. For the purpose of this study, refractory AML is defined as failure to achieve CR after one cycle of induction chemotherapy and relapsed AML is defined as any evidence of disease recurrence after achieving CR of any duration.
- Both males and females ≥ eighteen years of age
- Karnofsky Performance Status of ≥ 70% or Eastern Cooperative Oncology Group (ECOG) performance status < 2 (unless poor performance status is related to the disease).
- Adequate organ function defined as:
Key exclusion criteria (19 listed)
- Age ≥ of seventy-six years.
- Subjects with the inability to swallow oral medications.
- Clinical evidence of active CNS leukemia.
- A medical history of receiving an allogeneic hematopoietic stem cell transplantation (HSCT).
- Subjects must not have any uncontrolled or intercurrent illness including, but not limited to,
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 |
|---|---|---|
| MidostaurinAlso: PKC412, RydaptInduction Phase: 50 mg orally twice daily beginning on day 7 and ending 48 hours prior to beginning the consolidation phase Consolidation Phase: 50 mg orally twice daily beginning on Day 6 and ending 48 hours prior to either the start of the next cycle or beginning conditioning therapy for allogenic stem cell transplant. | Drug | Midostaurin and Cytarabine |
| CytarabineAlso: DepocytInduction Phase: 3 g/m2 (patients 18-65 years old) or 1 g/m2 (patients > 65 years old or those with co-morbidities precluding a higher dose) by vein over 3 hours every 12 hours on Days 1-6. Consolidation Phase: 3 g/m2 (patients 18-65 years old) or 1 g/m2 (patients > 65 years old or those with co-morbidities precluding a higher dose) by vein over 3 hours every 12 hours on Days 1, 3, and 5 of each cycle. | Drug | Midostaurin and Cytarabine |
Primary outcome
Progression Free Survival
Time frame: Up to 6 months
Compare the progression free survival of patients receiving midostaurin and cytarabine to that historically achieved in patients receiving cytarabine alone
3 secondary outcomes
Number of participants with treatment-related adverse events as assessed by National Cancer Institute (NCI) Common Terminology for Adverse Events(CTCAE) v4.0
Time frame: Up to 2.5 years
Overall survival
Time frame: Up to 3.5 years
Morphologic response rate
Time frame: Up to 6 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 22 Oct 2018.
- Individual participant dataSharing planned: No
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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.
Maxim Norkin, MD, PhD principal investigator
University of Florida
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Midostaurin and Cytarabine in people with Acute Myeloid Leukemia, run by University of Florida 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 16 inclusion and 19 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 22 Oct 2018. 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 phase mean?
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?
Progression Free Survival (time frame: Up to 6 months). 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 22 Oct 2018.