View clinical trials related to Mastocytosis.
Filter by:Systemic mastocytosis with an associated hematologic neoplasm (SM-AHN) is a challenging disease to treat. Targeted KIT inhibitors have been approved for this indication based on their ability to control the mastocytosis portion of the disease, but patients frequently experience progression of the concomitant myeloid malignancy (i.e. the AHN). Using a combination approach to treat both aspects of the disease has the potential to provide enhanced disease control; however, overlapping toxicity is a concern. In this study, investigators aim to study the safety and tolerability of combined avapritinib and decitabine for the treatment of SM-AHN.
This clinical trial aims to evaluate and compare novel and commercially available diagnostic assays through blood tests for the differential diagnosis and comprehensive assessment of patients experiencing recurrent angioedema attacks, including both urticarial and non-urticarial angioedema. The primary objective is to assess the efficiency of novel diagnostic assays, both individually and in combination, in comparison to currently available commercial tests. The ultimate goal is to establish the feasibility of developing an affordable and accurate laboratory test capable of diagnosing the diverse etiological manifestations of angioedema.
Systemic Mastocytosis is a rare and complex disease caused by accumulation of mast cells. The skin, bones, gastrointestinal tract, bone marrow and liver are the organs most often affected. Symptoms can vary greatly between patients. The study aims to describe the Swedish cohort's self-rated quality of life and levels of disease-related symptoms.
The Registry for Patients With Systemic Mastocytosis in China is a voluntary, observational database that will capture demographic, socioeconomic, and disease information with systemic mastocytosis. No experimental intervention is involved.
Mastocytosis is a group of pathologies characterized by the accumulation and/or clonal proliferation of abnormal mast cells in various organs. The impact of mastocytosis on health, quality of life, the psychoaffective sphere and on professional life constitutes the burden of this disease. The DALY is the disability-adjusted life expectancy, which takes into account the life expectancy and the number of years "lost" due to illness, disability or early death. Due to the major impact of the disease this study evaluates the DALY in indolent mastocytosis patients
The goal of this clinical trial is to evaluate elenestinib (BLU-263) in participants with Advanced Systemic Mastocytosis (AdvSM), SM with an associated hematologic neoplasm (SM-AHN), and other hematologic malignancies. The main questions it aims to answer are: - Determine Recommended Dose of elenestinib (BLU-263) monotherapy for participants with AdvSM - Safety and tolerability of elenestinib (BLU-263) monotherapy - Efficacy of elenestinib (BLU-263) monotherapy in participants with AdvSM - Determine Recommended Dose of elenestinib (BLU-263) in combination with azacitidine in participants with AdvSM - Safety and tolerability of elenestinib (BLU-263) in combination with azacitidine - Efficacy of elenestinib (BLU-263) in combination with azacitidine in participants with AdvSM The estimated study duration for each participant will be approximately 4 years: 2 years of treatment followed by 2 years of follow-up. Participants may be required to attend monthly visits for the first six months, followed by quarterly visits for the remainder of the study.
The observational study aimed at evaluating the incidence of systemic mastocytosis associated with t(8;21) AML in patients with de novo t(8;21) AML and their responses to first induction, and the prognosis from standard therapy.
To evaluate the efficacy and safety of two dosing schemes of oral masitinib versus matching placebo in the treatment of patients suffering from severe MCAS with handicap unresponsive to optimal symptomatic treatment.
The aim of the study is to confirm the association between hypermobile Ehlers Danlos syndrome (hEDS) and mast cell activation syndrome (MCAS) in patients with digestive disorders managed in allergology departments.
CP-MGD024-01 is a Phase 1, open-label, multi-center study of MGD024 as a single agent in patients with select blood cancers that have not responded to treatment with standard therapies or who have relapsed after treatment. The study is designed to determine the safety, tolerability, pharmacokinetics (affect of the body on the drug), pharmacodynamic (affect of the drug on the body), immunogenicity (development of antibodies against the drug), and preliminary anti-cancer effect of MGD024. Patients will receive treatment with MGD024 in consecutive 28-day cycles for a study treatment period of up to 12 cycles (approximately 1 year) or until treatment or study discontinuation criteria are met. Response assessments will be performed after Cycle 1 and then after every even numbered cycle starting with Cycle 2 until progression or study treatment discontinuation. Patients will be checked for side effects throughout the study.