Clinical Trials

The primary purpose of Part 1 of this study is to determine the Recommended Phase 2 Dose (RP2D) in patients with Indolent Systemic Mastocytosis (ISM) for use in Part 2 and Part 3 of the study.

The primary purpose of Part 2 of this study is to determine the effect of avapritinib in reducing symptoms in Indolent Systemic Mastocytosis (ISM) and Smoldering Systemic Mastocytosis (SSM) patients, as compared to placebo.

The primary purpose of Part 3 of this study is to assess the long-term safety and effectiveness of avapritinib in Indolent Systemic Mastocytosis (ISM) and Smoldering Systemic Mastocytosis (SSM) patients.

Specific aims:

1. Measurement of urinary arachidonic acid metabolites such as prostaglandins PGD2, PGE2, 8-isoprostane and TBXB2 in mastocytosis.

2. Comparison of these novel markers to conventional markers of tryptase and the well characterized n-methyl histamine and 11β-prostaglandinF2α

In this study, the investigators will determine the utility of Tamoxifen, a non-cytotoxic agent, to improve quality of life, biochemical parameters, and bone marrow involvement in systemic mastocytosis patients having 1) up to 20% bone marrow infiltration by mast cells and/or 2) mediator-release symptoms which are not controlled by tolerated doses of standard "non-cytotoxic" medications regardless of the percentage bone marrow involvement by mastocytosis. The dose of Tamoxifen will be 20 mg/day and the duration of treatment will be for one year. Patients currently taking interferon alfa, imatinib mesylate, or cladribine will be excluded until these medications have been stopped.

The purpose of this study is to identify biomarkers that can show differences in mast cell disease (Mastocytosis) status and activity for help with diagnosis and prognosis.

The purpose of this study to determine recommended dose (RD) of BLU-263 for patients with Indolent Systemic Mastocytosis.

The purpose of this study is to investigate CGT9486 for the treatment of patients with Advanced Systemic Mastocytosis (AdvSM), including patients with Aggressive SM. (ASM), System Mastocytosis (SM) with Associated Hematologic Neoplasm (SM-AHN), and Mast Cell Leukemia (MCL).

The purpose of this study is to attempt to establish new mast cell lines from patients with systemic mastocytosis using peripheral blood and/or bone marrow.

This is a non-randomized open label multi-center study. Patients with high-risk myeloproliferative neoplasms (systemic mastocytosis [SM], advanced symptomatic hypereosinoophic disorder [PED], myelofibrosis [MF], and chronic myelomonocytic leukemia [CMML]) will be treated with SL-401, which will be administered as a brief intravenous infusion for 3 consecutive days initially every 21 days for 4 cycles; every 28 days for cycles 5-7; then every 42 days. Stage 1 will consist of a period in which several doses of SL-401 are evaluated. The Stage 2 portion will enroll up to 18 patients with each of the 4 myeloproliferative malignancies: SM, PED, MF, and CMML. ...

This study is being done to store blood cells, genetic material, blood serum and tissue biopsies so that they can be used in laboratory studies now and in the future to find causes of the rare disorder of mast cells and/or eosinophils and factors that may contribute to disease progression and treatment response.

This is a Phase 1, open-label, first-in-human (FIH) dose-escalation study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and preliminary antitumor activity of DCC-2618, administered orally (PO), in adult patients with advanced malignancies. The study consists of 2 parts, a dose-escalation phase and an expansion phase.

The purpose of this study is to develop a data-driven approach that enables healthcare providers to “prescribe” exercise in the appropriate dose in a manner analogous to prescribing a drug.