NCT01830361

Brief Summary

To assess the efficacy of tyrosine-kinase inhibitor midostaurin in c-KIT or FLT3-ITD mutated t(8;21) AML. To assess the efficacy of midostaurin depending on the type of c-KIT mutation

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
18

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Mar 2013

Longer than P75 for phase_2

Geographic Reach
1 country

10 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

March 13, 2013

Completed
12 days until next milestone

First Submitted

Initial submission to the registry

March 25, 2013

Completed
18 days until next milestone

First Posted

Study publicly available on registry

April 12, 2013

Completed
6.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 30, 2019

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 30, 2019

Completed
Last Updated

August 6, 2020

Status Verified

August 1, 2020

Enrollment Period

6.6 years

First QC Date

March 25, 2013

Last Update Submit

August 5, 2020

Conditions

Acute Myeloid Leukemia

Keywords

AMLAcute Myeloid Leukemiac-KITFLT3-ITDt(8;21)chemotherapymidostaurin

Outcome Measures

Primary Outcomes (1)

  • Event-free Survival

    2-year Event-free Survival

Secondary Outcomes (7)

  • Time to relapse

    2-years

  • Overall survival

    2-years

  • Relapse-free survival

    2-years

  • morphologic and molecular CR rate

    2-years

  • incidence of AEs/SAEs

    until 30 days after end of treatment

  • +2 more secondary outcomes

Study Arms (1)

midostaurin (PKC412), capsules

EXPERIMENTAL

midostaurin 50 mg (two 25 mg capsules) is given in combination with the second of two induction cycles and in combination with three cycles of high-dose cytarabine (HiDAC) consolidation chemotherapies and maintenance treatment in patients with c-kit or FLT3-ITD positive t(8;21) AML in an open-label one-arm design. The first cycle of induction is not part of the study.

Drug: midostaurin (PKC412)

Interventions

midostaurin (PKC412)DRUG

Midostaurin 50 mg (2 capsules) twice daily days 8-21 in induction II + consolidation I-III; maintenance treatment twice daily continuously for 12 months

Also known as: Rydapt
midostaurin (PKC412), capsules

Eligibility Criteria

Age18 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Diagnosis of c-KIT mutated t(8;21) AML i.e.
  • \>20% myeloid blasts in bone marrow and/or peripheral blood at initial diagnosis
  • Plus cytogenetic diagnosis of aberration t(8;21)/AML1-ETO
  • Plus mutation of c-KIT gene (mut-KIT17 or mut-KIT8) or FLT3-ITD mutation or both c-KIT and FLT3-ITD mutations
  • Chemoresponsive disease as determined by early bone marrow assessment on day 14-16 after first cycle of induction therapy with cytarabine in combination with daunorubicine or idarubicine, or mitoxantrone- Fit for further intensive chemotherapy
  • Age 18-65 years
  • ECOG performance status of 0-2
  • Life expectancy of at least 12 weeks

You may not qualify if:

  • Primary refractory or previously relapsed AML
  • Non-eligibility for high-dose cytarabine based consolidation, e.g. intolerance to cytarabine
  • Inability to swallow oral medications
  • Symptomatic congestive heart failure
  • Bilirubin \>2.5 x upper limit of normal

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (10)

Klinikum Chemnitz gGmbH, Klinik für Innere Medizin III

Chemnitz, Germany

Location

Universitätsklinikum Dresden Medizinische Klinik und Poliklinik I

Dresden, Germany

Location

Universitätsklinikum Erlangen, Medizinische Klinik 5

Erlangen, Germany

Location

Klinikum der Johann-Wolfgang-Goethe Universität

Frankfurt am Main, Germany

Location

Universitätsklinikum Heidelberg

Heidelberg, Germany

Location

Universitätsklinikum Jena, Klinik für Innere Medizin II

Jena, Germany

Location

Universitätsklinikum Gießen und Marburg GmbH

Marburg, Germany

Location

Universitätsklinikum Münster

Münster, Germany

Location

Städtisches Klinikum Nord

Nuremberg, Germany

Location

Klinikum der Universität Regensburg

Regensburg, Germany

Location

Related Links

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Interventions

midostaurin

Condition Hierarchy (Ancestors)

Leukemia, MyeloidLeukemiaNeoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic Diseases

Study Officials

  • Christoph Röllig, Prof. Dr.

    Medizinische Fakultät der TU Dresden, Medizinische Klinik und Poliklinik I

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 25, 2013

First Posted

April 12, 2013

Study Start

March 13, 2013

Primary Completion

October 30, 2019

Study Completion

October 30, 2019

Last Updated

August 6, 2020

Record last verified: 2020-08

Locations

DE(10)