Objective of this study is to determine the efficacy of midostaurin in patients with ASM or MCL with/without an associated hematological clonal non-mast cell lineage disease.
ID
Source
Brief title
Condition
- Leukaemias
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
To determine the efficacy of midostaurin in patients with ASM or MCL with or
without an AHNMD when administered orally at a dose of 100 mg b.i.d.
continuously as measured by overall response rate.
Secondary outcome
To evaluate the duration of response
To evaluate the time to response
To evaluate overall survival
To evaluate the safety and tolerability of midostaurin in patients with ASM or
MCL (with or without an associated hematological clonal non-mast cell lineage
disease).
To characterize t he KIT mutational status at baseline and after 6 cycles of
therapy and evaluate potential associations with efficacy outcomes.
Background summary
Aggressive Systemic Mastocytosis (ASM) and Mast Cell Leukemia (MCL) are
myeloproliferative neoplasms with limited treatment options and generally poor
prognosis. Both are diseases in which the body produces excessive amounts of a
particular type of white blood cell named mast cell. To date, there have been
limited treatment options for ASM and MCL. Existing treatments such as
interferon-alpha, steroids, and cladribine exhibit variable response rates in
advanced mast cell disease and these are usually partial in nature.
Study objective
Objective of this study is to determine the efficacy of midostaurin in patients
with ASM or MCL with/without an associated hematological clonal non-mast cell
lineage disease.
Study design
Na het doorlopen van de screeningsfase zal de patient, indien hij/zij aan de
in-/exclusie criteria voldoet, starten met midostaurin behandeling. Patienten
zullen 2 maal daags midostaurin innemen. Patienten kunnen met de behandeling
doorgaan zolang midostaurin goed verdragen wordt of totdat de ziekte verergert.
After the screenings period the patient will, in case patient meets all
in-/exclusion criteria, start with midostaurin treatment. Midostaurin will be
dosed twice daily. Treatment can be continued till progressive disease or
unacceptable toxicity (whichever comes first).
Intervention
Patients will be treated with twice daily 100 mg midostaurin.
A cycles is 28 days.
Patients take their medication continuously.
Study burden and risks
After the screeningsperiod the patient will be hospitalized during cycle 1 for
3 days, to closely monitor for any potential side effects, adverse events and
to obtain pharmacokinetic drug levels.
For the following visits the patient is asked to come to the clinic weekly
during the first month, biweekly during the second month, then once every month
for the following 10 months and subsequently every 3 months thereafter.
Evaluation of response will be done via bonemarrow assessments, bonescans and
DEXA scans.
Raapopseweg 1
6800 LZ Arnhem
NL
Raapopseweg 1
6800 LZ Arnhem
NL
Listed location countries
Age
Inclusion criteria
Adult patients with diagnosis of one major plus at least one minor criterion, or the presence of at least 3 minor criteria according to WHO criteria for Systemic Mastocytosis (Valent et al. 2001);Patient must present with at least one measurable C-Finding.;For patients with MCL: bone marrow aspirate smears must show 20% or more immature mast cells.
Exclusion criteria
Patients who have demonstrated relapse to more than two prior regimen of SM treatment-regardless of treatment regimen for supportive care (e.g. symptom limiting therapies);Patients who have aggressive systemic mastocytosis with eosinophilia and known positivity for the FIP1L1-PDGFR fusion unless they have demonstrated relapse or disease progression on prior imatinib therapy;Patients on imatinib therapy and known to be KIT D816V negative unless they have demonstrated relapse, resistance or intolerance to imatinib.;Patients with any pulmonary infiltrate including those suspected to be of infectious origin.
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2008-0002800-4-NL |
| CCMO | NL23439.042.08 |