Neoplasms

Immune System Diseases

All Conditions

Skin and Connective Tissue Diseases

Rare Diseases

Mastocytosis

Mastocytosis, Systemic

Neoplasms, Connective Tissue

Neoplasms, Connective and Soft Tissue

Neoplasms by Histologic Type

Mast Cell Activation Disorders

Systemic Mastocytosis

Antineoplastic Agents

All Drugs and Chemicals

Midostaurin

Protein Kinase Inhibitors

Enzyme Inhibitors

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

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

Midostaurin,

J.C. Kluin-Nelemans, MD, PhD

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

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 mediator symptom reduction, documented by the Mastocytosis Symptom Assessment Questionnaire, measured at 3 months.

Secondary:

1. To study whether symptom improvement persists at 6 months, and whether midostaurin can reduce mast cell infiltration in the skin and bone marrow, documented by decrease of serum tryptase, decrease of urticaria pigmentosa and decrease of bone marrow mast cells.
2. To assess safety and tolerability of midostaurin in the above mentioned settings

Study design: Single arm, open label pilot phase II study.

Study population: Adult patients (n=20) with histologically documented systemic mastocytosis, indolent or smoldering subtype, with severe symptoms, not controlled by histamine 1 and 2 blockers.

Intervention: treatment with Midostaurin, twice daily 100 mg orally for 6 months continuously.

Midostaurin in Indolent Systemic Mastocytosis

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.

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

persistence of improvement symptom score at 6 months.

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

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

University Medical Center Groningen

Prof.dr.

Blood and Lymph Conditions

Leukemia

Leukemia, Myeloid

Leukemia, Myeloid, Acute

Hematologic Diseases

Myeloid Leukemia

Acute Myeloid Leukemia

Acute Non Lymphoblastic Leukemia

Acute Graft Versus Host Disease

Midostaurin was administered in two different dosing options. Either as 2x25mg daily or 2x50mg daily.

Novartis Pharmaceuticals

This was a non-interventional, retrospective registry study, utilizing electronic health record (EHR) data collected in the hospital district of Helsinki and Uusimaa (HUS). Real-world health care resource utilization (HCRU) of AML patients was characterized.

Adult patients (18 years or older) with the inclusion diagnosis, AML (ICD-10 C92.0), between 1.1.2013 - 30.6.2020 were followed from the index date (first ever record of the inclusion diagnosis) until the end of follow-up (30.6.2020), or death. This study did not involve any contacts to patients.

The study inclusion period was changed from 1.1.2013 - 30.6.2020 specified in the protocol to 1.1.2016 - 30.6.2020 due to lack of medication data from pre-2016.

Healthcare Resource Utilization in Adults Diagnosed With Acute Myeloid Leukemia (AML)

Healthcare Resource Utilization in Adults Diagnosed With Acute Myeloid Leukemia (AML) With a Focus on Patients Treated With Rydapt (Midostaurin) in Helsinki and Uusimaa Hospital District

Number of patients initiating midostaurin treatment were reported.

Number of patients who received Midostaurin at induction and consolidation phases were reported.

Duration of Midostaurin at induction and consolidation phase were reported.

Median average dose intensity in midostaurin treatment were reported.

Most frequent comorbidities at the time of AML diagnosis were reported.

Number of patients at the time of diagnosis (All treated AML patients) were reported.

Number of AML patients receiving midostaurin consolidation treatment in inpatient or outpatient care (based on given prescriptions) were reported.

Estimate HCRU and related costs in different disease stages were reported.

Symptoms and General Pathology

Syndrome

Preleukemia

Myelodysplastic Syndromes

Bone Marrow Diseases

Precancerous Conditions

LGH447 monotherapy in patients with AML or MDS

LGH447 + midostaurin in patients with AML

LGH447

LGH447 + midostaurin

This study will assess the safety and preliminary efficacy of escalating doses of LGH447 monotherapy in AML and MDS and LGH447 in combination with midostaurin in AML.

A Safety and Efficacy Study of LGH447 in Patients With Acute Myeloid Leukemia (AML) or High Risk Myelodysplastic Syndrome (MDS)

A Phase I, Multicenter, Open-label Study of Oral LGH447 in Patients With Acute Myeloid Leukemia or High Risk Myelodysplastic Syndrome

Frequency and characteristics of dose limiting toxicities

Includes changes in hematology and blood chemistry values, assessments of physical examinations, vital signs, and electrocardiograms (ECGs)

LGH447 and midostaurin plasma concentrations and basic PK parameters

To assess any preliminary anti-tumor activity in AML or high risk MDS associated with LGH447

To assess any preliminary anti-tumor activity in AML or high risk MDS associated with LGH447 in combination with midostaurin

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Midostaurin in Indolent Systemic Mastocytosis	NCT01920204	Indolent System...	Midostaurin,	18 Years -	University Medical Center Groningen
Midostaurin in Indolent Systemic Mastocytosis	NCT01920204	Indolent System...	Midostaurin,	18 Years -	University Medical Center Groningen
Midostaurin in Indolent Systemic Mastocytosis	NCT01920204	Indolent System...	Midostaurin,	18 Years -	University Medical Center Groningen
Healthcare Resource Utilization in Adults Diagnosed With Acute Myeloid Leukemia (AML)	NCT05488613	Acute Myeloid L...	Midostaurin	18 Years - 99 Years	Novartis
Midostaurin in Indolent Systemic Mastocytosis	NCT01920204	Indolent System...	Midostaurin,	18 Years -	University Medical Center Groningen
Healthcare Resource Utilization in Adults Diagnosed With Acute Myeloid Leukemia (AML)	NCT05488613	Acute Myeloid L...	Midostaurin	18 Years - 99 Years	Novartis
A Safety and Efficacy Study of LGH447 in Patients With Acute Myeloid Leukemia (AML) or High Risk Myelodysplastic Syndrome (MDS)	NCT02078609	AML and High Ri...	LGH447 LGH447 + midost...	18 Years -	Novartis

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