Neoplasms

Immune System Diseases

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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.

AK002

Marcus Maurer, MD

This is a Phase 1 study to investigate the safety and tolerability of AK002 in patients with indolent systemic mastocytosis (ISM).

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AK002

A Phase 1, Single Ascending Dose and Multiple Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AK002 in Patients With Indolent Systemic Mastocytosis

Allakos Inc.

Blood and Lymph Conditions

Hypersensitivity

Hematologic Diseases

Immune Complex Diseases

Antigen-Antibody Complex

Bezuclastinib will be administered orally, once daily continuously for 28-day cycles

Bezuclastinib Tablets (Formulation A)

Bezuclastinib Tablets (Formulation B)

Placebo will be administered orally, once daily continuously for 28-day cycles

Placebo Tablets

Hina Jolin, PharmD

Rachael Easton, MD, PhD

This is a multi-part, randomized, double-blind, placebo-controlled Phase 2 clinical study comparing the safety and efficacy of bezuclastinib (CGT9486) plus best supportive care (BSC) with placebo plus BSC in patients with nonadvanced systemic mastocytosis (NonAdvSM), including indolent systemic mastocytosis and smoldering systemic mastocytosis, whose symptoms are not adequately controlled by BSC. This study will be conducted in three parts. Patients in Parts 1a, 1b and 2 will receive bezuclastinib or placebo, and may roll over onto Part 3 to receive treatment with bezuclastinib.

In Part 1a and 1b of the study, patients with NonAdvSM will be randomly assigned to 1 of 2 dose levels of bezuclastinib plus BSC, or to placebo plus BSC. Upon analysis of the Part 1 data, a dose will be selected for Part 2. In Part 2, patients with NonAdvSM will be randomly assigned to the selected dose of bezuclastinib plus BSC, or to placebo plus BSC.

Patients who complete Part 1 or Part 2 may participate in Part 3 in which all patients will receive bezuclastinib plus BSC.

(Summit) A Study to Evaluate the Efficacy and Safety of CGT9486 Versus Placebo in Patients With Indolent or Smoldering Systemic Mastocytosis

A Multi-Part, Randomized, Double-Blind, Placebo-Controlled Phase 2 Clinical Study of The Safety and Efficacy of CGT9486 in Subjects With Nonadvanced Systemic Mastocytosis

Selection of the recommended dose to be used in subsequent parts of the study.

Mean absolute change in a disease-specific patient reported outcome (PRO)

Scale of 0-100, higher numbers represent more severe impairment to quality of life.

Scale of 0-100, higher numbers represent better symptom outcomes

Response rate based on reduction in disease specific PRO

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
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AK002	NCT02808793	Indolent System...	AK002	18 Years - 65 Years	Allakos Inc.
Midostaurin in Indolent Systemic Mastocytosis	NCT01920204	Indolent System...	Midostaurin,	18 Years -	University Medical Center Groningen
(Summit) A Study to Evaluate the Efficacy and Safety of CGT9486 Versus Placebo in Patients With Indolent or Smoldering Systemic Mastocytosis	NCT05186753	SSM Mastocytosis, I... Mastocytosis, S... Mastocytosis	Bezuclastinib T... Bezuclastinib T... Placebo Tablets	18 Years -	Cogent Biosciences, Inc.

Spots Global Cancer Trial Database for ism

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Spots Global Cancer Trial Database for ism

Every month we try and update this database with for ism cancer trials from around the world to help patients and their families find trials that might be right for them. We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Every month we try and update this database with for ism cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.