View clinical trials related to Mastocytosis.
Filter by:BLU-285-2405 is a multi-center, synthetic control, observational and retrospective study designed to compare clinical outcomes for avapritinib compared with best available therapy for patients with AdvSM.
This phase I trial studies the best dose and side effects of flotetuzumab for the treatment of patients with blood cancers (hematological malignancies) that have spread to other places in the body (advanced) and have come back after a period of improvement (relapsed) or does not respond to treatment (refractory). Flotetuzumab is a monoclonal antibody that may interfere with the ability of cancer cells to grow and spread.
This study evaluates TL-895, a potent, orally-available and highly selective irreversible tyrosine kinase inhibitor for the treatment of Myelofibrosis (Cohorts 1-3) or Indolent Systemic Mastocytosis (Cohort 5). Participants must be diagnosed with Myelofibrosis and be relapsed/refractory (e.g., having failed prior therapy), intolerant, or ineligible to receive JAKi treatment, or be diagnosed with Indolent Systemic Mastocytosis.
The symptoms caused by mast cell disorders can have a significant impact on the state of health of individuals, constituting a real burden for them, and consequently altering their quality of life. It therefore seems important to clarify the impact on the quality of life, on the psycho-affective sphere, on professional life and on the direct and indirect costs caused by the disease, as well as on the "patient's remaining burden". It seems possible by a longitudinal study (patient follow-up over 1 year). Primary objective is Assessment of quality of life in adult patient with mast cell diseases at M0.
Cutaneous mastocytosis can be isolated or associated with systemic involvement. Urticaria pigmentosa affects around 80 to 85% of adult patients with cutaneous mastocytosis. It is also frequently present in patients with mastocytosis associated with systemic involvement (80% of patients in our experience). This skin damage is one of the causes of deterioration in quality of life in patients with mastocytosis, through the loss of self-esteem, due to the appearance of lesions. However there are not treatment for urticaria pigmentosa. Skin involvement in mastocytosis is linked to the accumulation of abnormal mast cells in the dermis. However, the mast cells are not pigmented and the brown-brown color characteristic of Urticaria pigmentosa is explained by melanin pigmentation of the epidermal basal layer.
The purpose of this study is to evaluate the efficacy and safety of oral masitinib versus placebo in the treatment of patients suffering from smouldering or indolent systemic mastocytosis with severe symptoms of mast cell mediator release, unresponsive to optimal symptomatic treatment.
Background: Mast cells help the body fight disease and heal wounds. People with indolent systemic mastocytosis (ISM) make too many mast cells. This causes pain, tiredness, digestive problems, and other symptoms. Researchers think the drug sarilumab could help. Objective: To see if sarilumab is a safe and effective treatment for people with ISM. Eligibility: Adults ages 18-75 with ISM who are enrolled in NIH study 02-I-0277 Design: Participants will be screened with: - Physical exam - Medical history - Blood and urine tests - Questionnaires - Bone marrow removed by a needle inserted into the hip bone - Ultrasound of the abdomen - Photographs of the skin Participants will repeat some screening tests at study visits. Participants will have a baseline visit in the hospital for 3 days. They will: - Be assigned to get either the study drug or a placebo. They will not know which one they get. - Have a skin punch biopsy: An instrument will remove a small piece of skin. - Get their first drug dose injected under their skin Participants will keep a side effect and medication diary during the study. Participants will visit the clinic to get a drug dose every 2 weeks, for a total of 8 doses. Participants will have a visit 2 weeks after their final dose. It will last up to 2 days. Participants will have another visit 12 weeks later. Participants may then continue this study for 1 more year. Those who continue will get sarilumab, even if they previously got the placebo, every 2 weeks. They will have visits every 6 weeks, and then every 3 months.
This phase II trial studies how well flotetuzumab works in treating patients with CD123 positive blood cancer that has come back or does not respond to treatment. Immunotherapy with monoclonal antibodies, such as flotetuzumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread.
This is a Phase 2, randomized, double-blind, placebo-controlled study comparing the efficacy and safety of avapritinib + best supportive care (BSC) with placebo + BSC in patients with indolent systemic mastocytosis (ISM) whose symptoms are not adequately controlled by BSC. The study will be conducted in 3 parts. All patients will receive treatment with avapritinib during Part 3 including those rolling over from the placebo group.
Mastocytosis is a heterogeneous rare disease. A 27-item questionnaire to assess the quality of life of patients with cutaneous/ indolent systemic mastocytosis was developed in the German language and validated in the English language in 2015. The team of the University Charité Berlin and the company Moxie - intellectual proprietor of the questionnaire - proposed the use of the recommendations of Baiardini et al. 2010, for linguistic adaptation of this questionnaire in other languages.