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Report for Clinical Trial NCT01920204

Developed by Shray Alag, 2020.
SNP Clinical Trial Gene

Phase II Single Arm Open Pilot Study to Demonstrate the Efficacy of Midostaurin in Symptom Improvement and Decrease of Mast Cell Burden in Patients With Indolent or Smoldering Systemic Mastocytosis.

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

NCT01920204 Indolent Systemic Mastocytosis
MeSH:Mastocytosis Mastocytosis, Systemic
HPO:Mastocytosis

1 Interventions

Name: Midostaurin,

Description: Midostaurin, twice daily 100 mg orally, continuously for 6 months
Type: Drug
Group Labels: 1

Midostaurin


Primary Outcomes

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

Measure: Symptom Scoring

Time: 12 weeks

Secondary Outcomes

Description: persistence of improvement symptom score at 6 months.

Measure: Persistence of improvements

Time: 6 months

Description: Percent change in the mast cell burden (bone marrow infiltrate, skin infiltrate, serum tryptase levels) after 6 months.

Measure: Mast cell burden

Time: 6 months

Description: Number and grading of Common Terminology Criteria adverse events during the 6 months of therapy.

Measure: Adverse events

Time: 6 months

Purpose: Treatment

Allocation: N/A

Single Group Assignment


There is one SNP

SNPs


1 D816V

Number and grading of Common Terminology Criteria adverse events during the 6 months of therapy.. Inclusion Criteria: - 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. --- D816V ---

- 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 Inclusion Criteria: - 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. --- D816V ---



HPO Nodes


HPO: