AzTrials
AzTrials

Acute Myeloid Leukemia (AML)Phase 3NCT03512197

A Global Study of the Efficacy and Safety of Midostaurin + Chemotherapy in Newly Diagnosed Patients With FLT3 Mutation Negative (FLT3-MN) Acute Myeloid Leukemia (AML)

A Phase 3 study testing Midostaurin and Chemotherapy in people with Acute Myeloid Leukemia (AML), run by Novartis Pharmaceuticals at 133 sites in 20 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

The purpose of this study was to confirm the preliminary evidence from early clinical trials that midostaurin may provide clinical benefit not only to AML patients with the FLT3-mutations but also in FLT3-MN (SR<0.05) AML (FLT3 mutant to wild type signal ratio below the 0.05 clinical cut-off). Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Acute Myeloid Leukemia (AML)
What is being tested?
Midostaurin and Chemotherapy
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
511 participants
Where
133 sitesin 20 countries
Study dates
20 Jul 2018to 12 Feb 2021
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

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 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Diagnosis of AML (≥20% blasts in the bone marrow based on WHO 2016 classification). Patients with APL with PML-RARA are not eligible.
  • Suitability for intensive induction chemotherapy in the judgment of the investigator
  • Documented absence of an ITD and TKD activating mutation at codons D835 and I836 in the FLT3 gene, as determined by analysis in a Novartis designated laboratory using a validated clinical trial assay with clinical cutoff of 0.05 mutant to wild type signal ratio
  • Age ≥18 years
  • Laboratory values that indicate adequate organ function assessed locally at the screening visit

Who cannot join

  • Central nervous system (CNS) leukemia
  • Therapy-related secondary AML
  • Isolated extramedullary leukemia
  • Prior therapy for leukemia or myelodysplasia
  • AML after antecedent myelodysplasia (MDS) with prior cytotoxic treatment (e.g., azacytidine or decitabine)
  • Prior treatment with a FLT3 inhibitor (e.g., midostaurin, quizartinib, sorafenib)

Trial locations and contacts

133 sites in 20 countries.

133 sites in 20 countries

Germany38 sites

  • Novartis Investigative Site

    Bad Saarow, 15526

  • Novartis Investigative Site

    Berlin, 10967

  • Novartis Investigative Site

    Berlin, 12351

  • Novartis Investigative Site

    Berlin, 13125

  • Novartis Investigative Site

    Berlin, 13353

  • Novartis Investigative Site

    Bochum, 44892

  • Novartis Investigative Site

    Bonn, 53105

  • Novartis Investigative Site

    Braunschweig, 38114

  • Novartis Investigative Site

    Darmstadt, 64283

  • Novartis Investigative Site

    Duisburg, 47166

  • Novartis Investigative Site

    Düsseldorf, 40225

  • Novartis Investigative Site

    Eschweiler, 52249

  • Novartis Investigative Site

    Essen Werden, 45239

  • Novartis Investigative Site

    Flensburg, 24939

  • Novartis Investigative Site

    Giessen, 35392

  • Novartis Investigative Site

    Göttingen, 37075

  • Novartis Investigative Site

    Hamburg, 20099

  • Novartis Investigative Site

    Hamburg, 20246

  • Novartis Investigative Site

    Hanover, 30625

  • Novartis Investigative Site

    Homburg, 66421

  • Novartis Investigative Site

    Karlsruhe, 76133

  • Novartis Investigative Site

    Kiel, 24116

  • Novartis Investigative Site

    Leipzig, 04103

  • Novartis Investigative Site

    Lübeck, 23538

  • Novartis Investigative Site

    Magdeburg, 39120

  • Novartis Investigative Site

    Mainz, 55131

  • Novartis Investigative Site

    München, 80377

  • Novartis Investigative Site

    Osnabrück, 49076

  • Novartis Investigative Site

    Paderborn, 33098

  • Novartis Investigative Site

    Regensburg, Bavaria, 93053

  • Novartis Investigative Site

    Rostock, 18057

  • Novartis Investigative Site

    Schwerin, Brandenburg, 19049

  • Novartis Investigative Site

    Siegen, 57072

  • Novartis Investigative Site

    Stuttgart, 70176

  • Novartis Investigative Site

    Stuttgart, 70376

  • Novartis Investigative Site

    Ulm, 89081

  • Novartis Investigative Site

    Würzburg, 97080

  • Novartis Investigative Site

    Zwickau, 08060

Other trials in Germany

Japan20 sites

  • Novartis Investigative Site

    Aomori, 030 8553

  • Novartis Investigative Site

    Bunkyo Ku, Tokyo, 113-8677

  • Novartis Investigative Site

    Bunkyo-ku, Tokyo, 113-8603

  • Novartis Investigative Site

    Fukuoka, Fukuoka, 812-8582

  • Novartis Investigative Site

    Fukushima, Fukushima, 960 1295

  • Novartis Investigative Site

    Fukuyama, Hiroshima, 720-0001

  • Novartis Investigative Site

    Gifu, Gifu, 500 8513

  • Novartis Investigative Site

    Hamamatsu, Shizuoka, 432-8580

  • Novartis Investigative Site

    Hirakata, Osaka, 573-1191

  • Novartis Investigative Site

    Isehara, Kanagawa, 259-1193

  • Novartis Investigative Site

    Kyoto, 606 8507

  • Novartis Investigative Site

    Matsuyama, Ehime, 790-8524

  • Novartis Investigative Site

    Nagasaki, Nagasaki, 852-8501

  • Novartis Investigative Site

    Nagoya, Aichi-ken, 466-8650

  • Novartis Investigative Site

    Okayama, Okayama-ken, 701-1192

  • Novartis Investigative Site

    Osaka, 534-0021

  • Novartis Investigative Site

    Ōsaka-sayama, Osaka, 589 8511

  • Novartis Investigative Site

    Shimotsuke, Tochigi, 329-0498

  • Novartis Investigative Site

    Tsukuba, Ibaraki, 305-8576

  • Novartis Investigative Site

    Yamagata, 990 9585

Other trials in Japan

Italy18 sites

  • Novartis Investigative Site

    Alessandria, AL, 15100

  • Novartis Investigative Site

    Ancona, AN, 60126

  • Novartis Investigative Site

    Bergamo, BG, 24127

  • Novartis Investigative Site

    Brescia, BS, 25123

  • Novartis Investigative Site

    Catania, CT, 95123

  • Novartis Investigative Site

    Milan, MI, 20162

  • Novartis Investigative Site

    Modena, MO, 41124

  • Novartis Investigative Site

    Palermo, PA, 90146

  • Novartis Investigative Site

    Perugia, PG, 06129

  • Novartis Investigative Site

    Pescara, PE, 65124

  • Novartis Investigative Site

    Piacenza, PC, 29100

  • Novartis Investigative Site

    Reggio Calabria, RC, 89124

  • Novartis Investigative Site

    Roma, RM, 00133

  • Novartis Investigative Site

    Roma, RM, 00161

  • Novartis Investigative Site

    Roma, RM, 00168

  • Novartis Investigative Site

    Taranto, TA, 74100

  • Novartis Investigative Site

    Torino, TO, 10126

  • Novartis Investigative Site

    Vicenza, VI, 36100

Other trials in Italy

Spain11 sites

  • Novartis Investigative Site

    Barakaldo, Basque Country, 48903

  • Novartis Investigative Site

    Barcelona, 08041

  • Novartis Investigative Site

    Cáceres, Extremadura, 10003

  • Novartis Investigative Site

    Córdoba, Andalusia, 14004

  • Novartis Investigative Site

    L'Hospitalet de Llobregat, Catalonia, 08907

  • Novartis Investigative Site

    Madrid, 28034

  • Novartis Investigative Site

    Madrid, 28041

  • Novartis Investigative Site

    Salamanca, Castille and León, 37007

  • Novartis Investigative Site

    Seville, Andalusia, 41013

  • Novartis Investigative Site

    Valencia, 46026

  • Novartis Investigative Site

    Zaragoza, 50009

Other trials in Spain

France9 sites

  • Novartis Investigative Site

    Angers, 49033

  • Novartis Investigative Site

    Avignon, 84000

  • Novartis Investigative Site

    Bayonne, Bayonne Cedex, 64109

  • Novartis Investigative Site

    Dijon, 21034

  • Novartis Investigative Site

    Lille, 59037

  • Novartis Investigative Site

    Nantes, 44093

  • Novartis Investigative Site

    Paris, 75012

  • Novartis Investigative Site

    Pierre-Bénite, 69495

  • Novartis Investigative Site

    Toulouse, 31059

Other trials in France

Australia4 sites

  • Novartis Investigative Site

    Murdoch, Western Australia, 6150

  • Novartis Investigative Site

    Prahran, Victoria, 3181

  • Novartis Investigative Site

    Westmead, New South Wales, 2145

  • Novartis Investigative Site

    Woolloongabba, Queensland, 4102

Other trials in Australia

Taiwan4 sites

  • Novartis Investigative Site

    Kaohsiung City, 83301

  • Novartis Investigative Site

    Kuei Shan Chiang, Taoyuan Taiwan ROC, 33305

  • Novartis Investigative Site

    Putzu City, Chiayi Hsien, 61363

  • Novartis Investigative Site

    Taipei, 10002

Other trials in Taiwan

United States3 sites

  • Dana Farber Cancer Institute

    Boston, Massachusetts, 02115

  • University of Chicago Medical Center .

    Chicago, Illinois, 60637

  • Oregon Health and Science Univ

    Portland, Oregon, 97239

Other trials in United States

Austria3 sites

  • Novartis Investigative Site

    Linz, A-4010

  • Novartis Investigative Site

    Vienna, 1090

  • Novartis Investigative Site

    Vienna, 1140

Other trials in Austria

Belgium3 sites

  • Novartis Investigative Site

    Antwerp, 2060

  • Novartis Investigative Site

    Bruges, 8000

  • Novartis Investigative Site

    Roeselare, 8800

Other trials in Belgium

Brazil3 sites

  • Novartis Investigative Site

    Porto Alegre, Rio Grande do Sul, 90035-003

  • Novartis Investigative Site

    São Paulo, São Paulo, 04014-002

  • Novartis Investigative Site

    São Paulo, 01221 900

Other trials in Brazil

Israel3 sites

  • Novartis Investigative Site

    Haifa, 3109601

  • Novartis Investigative Site

    Petah Tikva, 4941492

  • Novartis Investigative Site

    Tel Aviv, 6423906

Other trials in Israel

Turkey (Türkiye)3 sites

  • Novartis Investigative Site

    Adana, 01330

  • Novartis Investigative Site

    Ankara, 06100

  • Novartis Investigative Site

    Aydin, 09100

Other trials in Turkey (Türkiye)

Czechia2 sites

  • Novartis Investigative Site

    Brno-Bohunice, 625 00

  • Novartis Investigative Site

    Plzen-Bory, 30599

Other trials in Czechia

Norway2 sites

  • Novartis Investigative Site

    Bergen, NO-5021

  • Novartis Investigative Site

    Oslo, 0424

Other trials in Norway

Portugal2 sites

  • Novartis Investigative Site

    Lisbon, 1099 023

  • Novartis Investigative Site

    Porto, 4200-072

Other trials in Portugal

Switzerland2 sites

  • Novartis Investigative Site

    Bern, 3010

  • Novartis Investigative Site

    Zurich, 8091

Other trials in Switzerland

Argentina1 site

  • Novartis Investigative Site

    CABA, Buenos Aires, C1118AAT

Other trials in Argentina

Bulgaria1 site

  • Novartis Investigative Site

    Sofia, 1756

Other trials in Bulgaria

Poland1 site

  • Novartis Investigative Site

    Gdansk, 80 952

Other trials in Poland

About this study

Official title: A Phase III, Randomized, Double-blind Study of Chemotherapy With Daunorubicin or Idarubicin and Cytarabine for Induction and Intermediate Dose Cytarabine for Consolidation Plus Midostaurin (PKC412) or Chemotherapy Plus Placebo in Newly Diagnosed Patients With FLT-3 Mutation Negative Acute Myeloid Leukemia (AML)

The purpose of this study was to confirm the preliminary evidence from early clinical trials that midostaurin may provide clinical benefit not only to AML patients with the FLT3-mutations but also in FLT3-MN (SR<0.05) AML (FLT3 mutant to wild type signal ratio below the 0.05 clinical cut-off).

This study evaluated the efficacy and safety of midostaurin in combination with daunorubicin or idarubicin and cytarabine for induction and intermediate-dose cytarabine for consolidation, and midostaurin single agent post-consolidation therapy in newly diagnosed patients with FLT3-MN (SR<0.05) AML.

Read the detailed description

This was a multi-center, multinational, randomized, double-blind Phase III study using a group sequential design. Subjects were stratified according to age (<60 vs. ≥ 60 years). Subjects within each stratum were randomized in a 1:1 ratio into one of two treatment arms: Midostaurin + chemotherapy 'or' Placebo + chemotherapy.

The study consisted of the following phases:

Screening/randomization phase: Subjects had to sign informed consent form before screening for enrollment. Subjects started chemotherapy at day 1 and were randomized at day 8.

Induction phase: All subjects received at least one cycle (28 days) of induction therapy with continuous infusion cytarabine (D1 - D7) and daunorubicin or idarubicin (D1 - D3) (induction 1). Subjects who did not achieve CR or CRi with adequate blood count recovery after Induction 1 received a second cycle with intermediate-dose cytarabine (D1 - D3) and daunorubicin or idarubicin (D1 - D3) (induction 2). Subjects who did not achieve CR or CRi with adequate blood recovery after induction 2 discontinued study treatment and were followed for survival.

Consolidation phase: Subjects who achieved CR or CRi with adequate blood count recovery after induction with one or two cycles of induction proceeded to consolidation therapy with either 3 or 4 cycles respectively of intermediate-dose cytarabine (D1 - D3), or to Hematopoietic Stem Cells Transplantation (HSCT) with or without preceding consolidation cycles.

Post-consolidation phase: Subjects who maintained CR or CRi with adequate blood count recovery at the end of the consolidation phase received 12 cycles (28 days/cycle) of continuous therapy with midostaurin or placebo twice daily at 50 mg. Subjects who underwent HSCT after achieving CR or CRi with adequate blood count recovery received midostaurin or placebo twice daily 50 mg post-transplant therapy, continuously, for up to 12 cycles (28 days/cycle). Post HSCT post-consolidation therapy began >30 days but not later than 100 days following HSCT.

Follow-up phase: All enrolled subjects were followed through the treatment period and until relapse/treatment failure, thereafter for start of new line of therapy and survival.

Being tested
Midostaurin, Placebo, Chemotherapy
Keywords
Newly diagnosed FLT3-mutation negative, PKC412, midostaurin, cytarabine, daunorubicin, idarubicin, acute myeloid leukemia, AML, FLT3-MN (SR<0.05), combination treatment, induction failure, event free survival, EFS, minimal residual disease
Medical subject headings
Leukemia, Myeloid, Acute, Neoplasm, Residual

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • MidostaurinDrug

    Also called PKC412

    Midostaurin was provided as 25 mg capsules 8PC, was supplied as double-blind in blister packs and taken orally.

  • PlaceboDrug

    Placebo was provided as 25 mg soft gelatin capsules 8PC, was supplied as double-blind in blister packs and taken orally.

  • ChemotherapyDrug

    Along with the study drug/placebo, chemotherapy was given as well: either Daunorubicin or Idarubicin and Cytarabine - all taken by i.v.

Study groups in NCT03512197
Study groupRoleWhat they receive
Midostaurin + chemotherapyExperimentalParticipants received Midostaurin in Induction 50mg twice daily on Day 8 until 48 hrs before start of next cycle. During Induction 2 and consolidation 50mg twice daily on Day 4 until 48 hrs before start of next cycle. During post-consolidation 50mg twice daily for 28 consecutive days of each 28-day treatment cycle up to 12 cycles. For participants who could not tolerate the protocol-specified dosing schedule, dose interruptions and/or reductions were either recommended or mandated allowing participants to continue the study treatment. Chemotherapy consisted of daunorubicin or idarubicin and cytarabine for induction and intermediate dose cytarabine for consolidation.
Receives: Drug: Midostaurin; Drug: Chemotherapy
Placebo + chemotherapyPlacebo comparatorParticipants received matching placebo to midostaurin with same dose, plus chemotherapy. Chemotherapy consisted of daunorubicin or idarubicin and cytarabine for induction and intermediate dose cytarabine for consolidation
Receives: Drug: Placebo; Drug: Chemotherapy

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Event Free Survival (EFS)

    EFS was defined as the time from randomization to failure to obtain a complete remission (CR) or Complete remission with incomplete hematologic recovery (CRi) with adequate blood count recovery in induction, relapse after CR or CRi with adequate blood count recovery or death due to any cause, whichever occurred first as assessed by the investigator.

    Measured: From date of Randomization up to approx. 30 months

Secondary outcomes (18)

  1. Overall Survival (OS) (Key Secondary)

    OS was defined as the time from randomization to date of death due to any cause. Patients entered the survival follow-up phase once they completed the safety follow up period (30 days after the last dose of midostaurin/placebo) in case of induction failure or if they had relapsed during post-treatment follow-up. Patients were then contacted by telephone every 3 months +/- 2 weeks or had a visit to follow up on their survival status, per Kaplan-Meier estimates.

    Measured: Between randomization to date of death up to approx. 30 months

  2. Percentage of Participants With Complete Remission (CR) and Complete Remission With Incomplete Hematological Recovery (CRi) But With Adequate Blood Count Recovery Rate.

    Assessment was based on the International Working Group (IWG) criteria for AML as per investigator assessment.

    CR: Bone marrow: < 5% blasts no blasts with Auer rods; Peripheral blood: neutrophils ≥ 1.0 x 109/L platelets ≥ 100 x 109/L, no blasts; No evidence of extramedullary disease (such as central nervous system (CNS) or soft tissue involvement); Transfusion independent.

    CRi with adequate blood count recovery is defined as the following:

    Bone marrow < 5% blasts no blasts with Auer rods Peripheral blood Neutrophils >= 1.0 x 109/L and 50 x 109/L <=platelets < 100 x 109/L no blasts No evidence of extramedullary disease (such as CNS or soft tissue involvement).

    Measured: At maximum 93 days from induction therapy start

  3. Percentage of Participants With Minimal Residual Disease (MRD) Negative Status

    MRD- rate was defined as the rate of participants reaching MRD at any timepoint. Participants with leukemic blasts below 0.1% were considered as MRD-negative based on leukemia-associated immunophenotype (LAIP).

    MRD was derived from bone marrow and blood data using cellular and molecular technologies and MRD status was measured using the flow cytometry assessments for LAIP irrespective of the investigator's overall clinical response assessment.

    Measured: from start of treatment up to end of post-consolidation (approximately 17 months)

  4. Percentage of Participants With Minimal Residual Disease (MRD) Negative Status During Post-consolidation Phase

    MRD- rate was defined as the rate of participants reaching MRD at any timepoint during Post-consolidation phase. Participants with leukemic blasts below 0.1% were considered as MRD-negative based on leukemia-associated immunophenotype (LAIP).

    MRD was derived from bone marrow and blood data using cellular and molecular technologies and MRD status was measured using the flow cytometry assessments for LAIP irrespective of the investigator's overall clinical response assessment.

    Measured: from start of post-consolidation to end of post-consolidation phase (up to 12 months)

  5. Time to Measurable Residual Disease (MRD) Negativity by Flow Cytometry

    Time to MRD- is defined as time from randomization to first occurrence of MRD-. Participants with leukemic blasts below 0.1% were considered as MRD-negative based on leukemia-associated immunophenotype (LAIP).

    MRD was derived from bone marrow and blood data using cellular and molecular technologies and MRD status was measured using the flow cytometry assessments for LAIP irrespective of the investigator's overall clinical response assessment.

    Measured: From date of Randomization up to approx. 17 months

  6. Disease-free Survival (DFS)

    DFS as measured from the date of first CR or CRi with adequate blood count recovery to relapse or death due to any cause, whichever occurred first. Participants who did not relapse nor die were censored at the last adequate response assessment. Assessment was based on the IWG criteria for AML as per investigator assessment

    Measured: From date of CR or CRi with adequate blood count recovery up to approx. 30 months

  7. Cumulative Incidence of Relapse (CIR)

    Cumulative Incidence of Relapse (CIR) was defined for participants with CR or CRi with adequate blood count recovery and was the time from achieving the CR or CRi with adequate blood count recovery until the onset of relapse from CR or CRi with adequate blood recovery. Participants without relapse were censored at the last adequate response assessment. Participants who died without relapse were counted as a competing cause of failure.

    Measured: From date of CR or CRi with adequate blood count recovery up to approx. 30 months

  8. Cumulative Incidence of Death (CID)

    Cumulative Incidence of Death (CID) was defined for all participants achieving CR or CRi with adequate blood count recovery measured from the date of achievement of CR or CRi until the date of death due to any reason. Participants not known to have died were censored on the last contact date. Participants who experienced relapse were counted as a competing cause of failure.

    Measured: From date of CR or CRi with adequate blood count recovery up to approx. 30 months

  9. Time to CR or CRi With Adequate Blood Count Recovery

    Time to CR or CRi with adequate blood count recovery was defined as the time from randomization to CR or CRi with adequate blood count recovery whichever occurred first

    Measured: At maximum 93 days from induction therapy start

  10. Time to Partial and Full Neutrophil Recovery

    The time to neutrophil recovery was assessed for the following criteria: Partial neutrophil recovery: Number of days from start of treatment to the first day neutrophils ≥0.5 x 10^9/L.

    Full neutrophil recovery: Number of days from start of treatment to the first day neutrophils ≥1.0 x 10^9/L

    Measured: At maximum 93 days from induction therapy start

  11. Time to Partial and Full Platelet Recovery

    Time to platelet recovery was assessed for the following criteria: Partial platelet recovery: Number of days from start of treatment to the first day platelets ≥50 x 10^9/L.

    Full platelet recovery: Number of days from start of treatment to the first day platelets ≥100 x 10^9/L.

    Measured: At maximum 93 days from induction therapy start

  12. Plasma Concentrations for Midostaurin and Its Metabolites: CGP52421 and CGP62221 for Non-poor Metabolizers

    Serial pharmacokinetics (PK) samples were collected in Non-poor metabolizer participants to assess the plasma concentrations of midostaurin, CGP52421 and CGP62221.

    Measured: from Induction (IND) phase 0hr (predose) to Post-consolidation phase (POSTCONS) 12hr

  13. AUC0-t: Pharmacokinetic (PK) Parameter for Midostaurin and Its Metabolites: CGP52421 and CGP62221 at Cycle 1, Day 8

    The AUC from time zero to a measurable concentration sampling time (t) (mass x time x volume-1). Note: as the last sampling time was at 12 h, AUC0-12h was determined after the first dose, reported at Cycle 1, Day 8

    Measured: 0 - 12 hrs

  14. AUClast: Pharmacokinetic (PK) Parameter for Midostaurin and Its Metabolites: CGP52421 and CGP62221 at Cycle 1, Day 8

    The AUC from time zero to the last measurable concentration sampling time after the first dose reported at Cycle 1, Day 8

    Measured: 0 - 12 hrs

  15. Cmax: Pharmacokinetic (PK) Parameter for Midostaurin and Its Metabolites: CGP52421 and CGP62221 at Cycle 1, Day 8

    The maximum (peak) observed plasma drug concentration after the first dose administration reported at Cycle 1, Day 8

    Measured: 0 - 12 hrs

  16. Tmax: Pharmacokinetic (PK) Parameter for Midostaurin and Its Metabolites: CGP52421 and CGP62221 at Cycle 1, Day 8

    The time to reach maximum (peak) plasma, blood, serum, or other body fluid drug concentration after single dose administration reported at Cycle 1, Day 8

    Measured: 0 - 12 hrs

  17. Total Score for Each Time Point for the Functional Assessment of Cancer Therapy-Leukemia (FACT-Leu)

    The total FACT-Leu score consists of 44 items with total scores ranging from 0 to 176. Higher scores indicate better health-related quality of life ( HRQoL). Negative changes from baseline indicate a worsening of HRQoL while positive changes indicate an improvement in HRQoL.

    Measured: From date of Randomization up to approx. 18 months

  18. Scores for Each Time Point for the EQ5D-5L (a Visual Analogue Scale (VAS))

    The EQ5D-5L questionnaire assesses 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 5 response options (no problems, slight problems, moderate problems, severe problems and extreme problems) that reflect increasing levels of difficulty. The patient is asked to indicate his/her current health state by selecting the most appropriate level in each of the 5 dimensions. The questionnaire also included a Visual Analogue Scale (VAS), where the patient is asked to rate current health status on a scale of 0 to 100, with 0 being the worst imaginable health state.

    Measured: From date of Randomization up to approx. 18 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted30 Apr 2018
  2. Study start20 Jul 2018
  3. Primary completion12 Feb 2021
  4. Study completion12 Feb 2021

Resources

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 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
511 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03512197
Sponsor's protocol ID
CPKC412E2301
Other IDs
2017-003540-21, 2017
Status last verified
Aug 2023
First submitted
26 Mar 2018
Last updated
21 Aug 2023
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data is currently available according to the process described on www.clinicalstudydatarequest.com.

Publications

  • Sechaud R, Peters T, Gu H, Rahmanzadeh G, Sharma GK, Guichard N, Menssen HD. Pharmacogenomic, pharmacokinetic, and safety analysis of CYP3A4/CYP3A5 polymorphisms of midostaurin in patients with acute myeloid leukemia. Eur J Clin Pharmacol. 2025 Dec 23;82(1):19. doi: 10.1007/s00228-025-03971-y.

    PubMed 41432755

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Midostaurin and Chemotherapy in people with Acute Myeloid Leukemia (AML), run by Novartis Pharmaceuticals at 133 sites in 20 countries.

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 5 inclusion and 6 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: finished, as of the sponsor's last update on 21 Aug 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 133 sites in Germany, Japan, Italy, Spain, France and 15 more countries. 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. Some participants in this study may receive a placebo. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 18 Jun 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Event Free Survival (EFS) (time frame: From date of Randomization up to approx. 30 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03512197, which also lists 1 linked publication.

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 21 Aug 2023; AzTrials last refreshed its copy on 8 Nov 2026.

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