Status:
UNKNOWN
Midostaurin in Indolent Systemic Mastocytosis
Lead Sponsor:
University Medical Center Groningen
Conditions:
Indolent Systemic Mastocytosis
Eligibility:
All Genders
18+ years
Phase:
PHASE2
Brief Summary
Rationale: Patients with indolent or smoldering systemic mastocytosis can have severe disabling symptoms. Almost all patients have fatigue, a compromised quality of life, hampering normal functioning....
Detailed Description
Objective: Primary: To study in a pilot phase II trial the efficacy of midostaurin administered at an oral dose of 100 mg twice daily in patients with indolent or smoldering systemic mastocytosis on ...
Eligibility Criteria
Inclusion
- Patients with Indolent Systemic Mastocytosis (ISM) or Smouldering Systemic Mastocytosis (SSM) according to the WHO criteria
- Presence of the D816V c-KIT mutation
- Serum tryptase \> 20 mg/l
- Serious mediator-related symptoms that cannot be controlled by H1 and H2 blocking drugs. Symptoms will be scored by an adapted MSAF (mastocytosis symptom assessment form) with at least:
- a pre-study score of 4 or more on 3 non-related items,
- or a pre-study score of 5 or more on 2 non-related items.
- one item from the scoring list can be replaced by flushes 7 or more per week or anaphylactic attacks 1 or more per week.
- Age \>18 years
- Willingness to apply optimal contraceptive measures (double barrier method, both men and women) for women below the age of 55, men at all ages; for both: if sexually active.
- Written informed consent
Exclusion
- Aggressive systemic mastocytosis, mast cell leukemia, or ASM with or without accompanying non-clonal related non-mast cell disorder (SM-ANHMD).
- Any known other present malignancy, non-melanoma skin cancers excluded
- History of malignancy within the last 5 years, non-melanoma skin cancers excluded
- Any serious comorbidity interfering with therapy compliance and follow-up compliance
- Pregnancy
- Patients not willing or who are not able to comply with contraceptive measures
Key Trial Info
Start Date :
August 1 2013
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
May 1 2015
Estimated Enrollment :
20 Patients enrolled
Trial Details
Trial ID
NCT01920204
Start Date
August 1 2013
End Date
May 1 2015
Last Update
January 19 2015
Active Locations (1)
Enter a location and click search to find clinical trials sorted by distance.
1
University Medical Center Groningen
Groningen, Provincie Groningen, Netherlands, 9700RB