NCT01920204

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. Because this form of mastocytosis is not considered life-threatening, mast cell eradication has never been applied and patients receive only symptomatic therapy with histamine blockers. Midostaurin, a c-KIT inhibitor has shown activity regarding symptom control and decrease of malignant mast cells in patients with aggressive systemic mastocytosis (ASM) or mast cell leukemia

Trial Health

43
At Risk

Trial Health Score

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

Trial has exceeded expected completion date
Enrollment
20

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Aug 2013

Geographic Reach
1 country

1 active site

Status
unknown

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

August 1, 2013

Completed
6 days until next milestone

First Submitted

Initial submission to the registry

August 7, 2013

Completed
2 days until next milestone

First Posted

Study publicly available on registry

August 9, 2013

Completed
1.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2015

Completed
2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2015

Completed
Last Updated

January 19, 2015

Status Verified

January 1, 2015

Enrollment Period

1.6 years

First QC Date

August 7, 2013

Last Update Submit

January 15, 2015

Conditions

Indolent Systemic Mastocytosis

Keywords

MastocytosisISMSSM

Outcome Measures

Primary Outcomes (1)

  • Symptom Scoring

    Percent change in the total score ("Sumscore") of all symptoms assessed by the Mastocytosis Symptom Assessment Form (MSAF) after 12 weeks.

    12 weeks

Secondary Outcomes (3)

  • Persistence of improvements

    6 months

  • Mast cell burden

    6 months

  • Adverse events

    6 months

Study Arms (1)

Midostaurin

EXPERIMENTAL

Treatment with Midostaurin, twice daily 100 mg orally for 6 months continuously.

Drug: Midostaurin,

Interventions

Midostaurin,DRUG

Midostaurin, twice daily 100 mg orally, continuously for 6 months

Also known as: PKC412
Midostaurin

Eligibility Criteria

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

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University Medical Center Groningen

Groningen, Provincie Groningen, 9700RB, Netherlands

Location

Related Publications (1)

  • van Anrooij B, Oude Elberink JNG, Span LFR, de Monchy JGR, Rosati S, Mulder AB, Kluin-Nelemans JC. Midostaurin in patients with indolent systemic mastocytosis: An open-label phase 2 trial. J Allergy Clin Immunol. 2018 Sep;142(3):1006-1008.e7. doi: 10.1016/j.jaci.2018.06.003. Epub 2018 Jun 8. No abstract available.

    PMID: 29890238DERIVED

MeSH Terms

Conditions

Mastocytosis, SystemicMastocytosis

Interventions

midostaurin

Condition Hierarchy (Ancestors)

Neoplasms, Connective TissueNeoplasms, Connective and Soft TissueNeoplasms by Histologic TypeNeoplasmsMast Cell Activation DisordersImmune System Diseases

Study Officials

  • J.C. Kluin-Nelemans, MD, PhD

    University Medical Center Groningen

    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
PRINCIPAL INVESTIGATOR
PI Title
Prof.dr.

Study Record Dates

First Submitted

August 7, 2013

First Posted

August 9, 2013

Study Start

August 1, 2013

Primary Completion

March 1, 2015

Study Completion

May 1, 2015

Last Updated

January 19, 2015

Record last verified: 2015-01

Locations

NL(1)