Clinical trials: Systemic Mastocytosis
SSM, Mastocytosis, Indolent
(Summit) A Study to Evaluate the Efficacy and Safety of CGT9486 Versus Placebo in Patients With Indolent or Smoldering Systemic Mastocytosis
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…
- Conditions
- SSM, Mastocytosis, Indolent, Mastocytosis, Systemic, Mastocytosis
- Trial name
- NCT05186753
- Testing
- Bezuclastinib Tablets (Formulation A), Bezuclastinib Tablets (Formulation B)
- Where
- 57 sites in United States, Australia and 13 more
- Volunteers
- 237 people
- Sponsor
- Cogent Biosciences, Inc.
Advanced Systemic Mastocytosis, Aggressive Systemic Mastocytosis
(PATHFINDER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, in Patients With Advanced Systemic Mastocytosis
This is an open-label, single arm, Phase 2 study evaluating the efficacy and safety of avapritinib (BLU-285) in patients with advanced systemic mastocytosis (AdvSM), including patients with aggressive SM (ASM), SM with associated…
- Conditions
- Advanced Systemic Mastocytosis, Aggressive Systemic Mastocytosis, Systemic Mastocytosis With an Associated Hematologic Neoplasm, Mast Cell Leukemia
- Trial name
- NCT03580655
- Testing
- Avapritinib
- Where
- 32 sites in United States, Austria and 10 more
- Volunteers
- 107 people
- Sponsor
- Blueprint Medicines Corporation
Indolent Systemic Mastocytosis, Smoldering Systemic Mastocytosis
(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis
This is a randomized, double-blind, placebo-controlled, Phase 2/3 study comparing the efficacy and safety of elenestinib (BLU-263) + symptom directed therapy (SDT) with placebo + SDT in participants with indolent systemic…
- Trial name
- NCT04910685
- Testing
- Elenestinib
- Where
- 88 sites in United States, Argentina and 23 more
- Volunteers
- 534 people
- Sponsor
- Blueprint Medicines Corporation
Indolent Systemic Mastocytosis
A Non-Interventional Study in Participants With Indolent Systemic Mastocytosis (ISM) in Germany
This is a non-interventional study assessing the effectiveness of avapritinib (BLU-285) in the management of ISM in real-world settings in Germany.
- Trial name
- NCT07255638
- Testing
- Avapritinib
- Where
- 12 sites in Germany
- Volunteers
- 80 people
- Sponsor
- Blueprint Medicines Corporation
Indolent Systemic Mastocytosis
(PIONEER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, Versus Placebo in Patients With Indolent Systemic Mastocytosis
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…
- Trial name
- NCT03731260
- Testing
- Avapritinib
- Where
- 49 sites in United States, Belgium and 11 more
- Volunteers
- 251 people
- Sponsor
- Blueprint Medicines Corporation
Advanced Systemic Mastocytosis (AdvSM), SM With an Associated Hematologic Neoplasm (SM-AHN)
(Apex) Bezuclastinib in Patients With Advanced Systemic Mastocytosis
This is an open-label, two-part Phase 2 study investigating CGT9486 for the treatment of patients with Advanced Systemic Mastocytosis (AdvSM), including patients with Aggressive SM (ASM), SM with Associated Hematologic Neoplasm…
- Conditions
- Advanced Systemic Mastocytosis (AdvSM), SM With an Associated Hematologic Neoplasm (SM-AHN), Mast Cell Leukemia (MCL), Aggressive Systemic Mastocytosis (ASM)
- Trial name
- NCT04996875
- Testing
- bezuclastinib
- Where
- 42 sites in United States, Australia and 12 more
- Volunteers
- 140 people
- Sponsor
- Cogent Biosciences, Inc.
Indolent Systemic Mastocytosis
An Observational Study in Participants With Indolent Systemic Mastocytosis (ISM)
This is a Phase 4, non-interventional, observational study to collect detailed data on the clinical characteristics, clinical outcomes and medical management of ISM in real-world settings.
- Trial name
- NCT07264959
- Where
- 7 sites in United States
- Volunteers
- 150 people
- Sponsor
- Blueprint Medicines Corporation
Angioedema, Angioedemas, Hereditary
Angioedema Biomarker Research Study
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…
- Conditions
- Angioedema, Angioedemas, Hereditary, Urticaria, Mastocytosis
- Trial name
- NCT06210698
- Testing
- Venipuncture
- Where
- United States
- Volunteers
- 600 people, healthy volunteers welcome
- Sponsor
- Foundation For Rare Disease Research
Systemic Mastocytosis
Precision Medicine for Indolent Systemic Mastocytosis (PRISM)
This study is being done to better understand how symptoms of Systemic Mastocytosis (SM) relate to biological signals and to better understand how different treatments affect these signals.
- Trial name
- PRISM (NCT07757893)
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Mayo Clinic
Leukemia, Acute Myeloid, Myelodysplastic Syndromes
A Study of MGD024 in Patients With Relapsed or Refractory Hematologic Malignancies
CP-MGD024-01 is a Phase 1, open-label, multi-center study of MGD024 as a single agent in participants with select blood cancers that have not responded to treatment with standard therapies or who have relapsed after treatment.
- Conditions
- Leukemia, Acute Myeloid, Myelodysplastic Syndromes, Classical Hodgkin Lymphoma, Leukemia, B-cell
- Trial name
- NCT05362773
- Testing
- MGD024
- Where
- 7 sites in United States
- Volunteers
- 130 people
- Sponsor
- MacroGenics
Systemic Mastocytoses, Indolent, Systemic Mastocytoses, Aggressive
Expanded Access to Bezuclastinib for Patients With NonAdvanced Systemic Mastocytosis or Advanced Systemic Mastocytosis
The purpose of this expanded access program (EAP) protocol is to provide investigational bezuclastinib to patients with a diagnosis of nonadvanced systemic mastocytosis (NonAdvSM) or advanced systemic mastocytosis (AdvSM) who…
- Trial name
- NCT06915766
- Testing
- Bezuclastinib
- Where
- 23 sites in United States
- Sponsor
- Cogent Biosciences, Inc.
Systemic Mastocytosis
Everolimus (RAD001) as Therapy for Patients With Systemic Mastocytosis
The goal of this clinical research study is to see if RAD001 can help to control the disease in patients with systemic mastocytosis (SM). The safety of this treatment will also be studied.
- Trial name
- NCT00449748
- Testing
- RAD001 (Everolimus)
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- M.D. Anderson Cancer Center
Systemic Mastocytosis, KIT-D816V Mutation
Peripheral Blood KIT-D816V Mutation in Adult Systemic Mastocytosis
This observational study aims to evaluate the diagnostic value and clinical utility of detecting the KIT-D816V mutation in the peripheral blood of adult patients with systemic mastocytosis (SM), using droplet digital PCR (ddPCR).
- Trial name
- PB-KIT in SM (NCT07562542)
- Where
- China
- Volunteers
- 50 people
- Sponsor
- The First Affiliated Hospital of Soochow University
Systemic Mastocytosis With an Associated Hematologic Neoplasm
Avapritinib With Decitabine in Patients With SM-AHN
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…
- Trial name
- NCT06327685
- Testing
- Avapritinib, Decitabine, Decitabine/Cedazuridine
- Where
- 7 sites in United States
- Volunteers
- 34 people
- Sponsor
- H. Lee Moffitt Cancer Center and Research Institute
Osteoporosis, Systemic Mastocytosis
Interest of Denosumab Treatment in Osteoporosis Associated to Systemic Mastocytosis
The study is looking at the efficacy of subcutaneously administrated denosumab 60 mg every 6 months versus placebo after 3 years, by analyze of lumbar spine bone mineral density (BMD) in systemic mastocytosis.
- Trial name
- DenosuMast (NCT03401060)
- Testing
- Denosumab
- Where
- 11 sites in France
- Volunteers
- 24 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Rheumatoid Arthritis, Spondyloarthritis
Pregnancy and Medically Assisted Conception in Rare Diseases
Rare diseases frequently affect women of childbearing age. Pregnancy in these women has become less rare, but remains associated with high levels of complications.
- Conditions
- Rheumatoid Arthritis, Spondyloarthritis, Psoriatic Arthritis, Systemic Lupus Erythematosus
- Trial name
- EGR2 (NCT02450396)
- Where
- France
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Mastocytosis, Systemic
Avapritinib Rollover Study
The primary objective of the rollover study is to evaluate the long-term safety of avapritinib in participants who have completed a Blueprint Medicines sponsored study (parent study) and continued to benefit from avapritinib.
- Trial name
- NCT06748001
- Testing
- Avapritinib
- Where
- 7 sites in Belgium, Canada and 4 more
- Volunteers
- 60 people
- Sponsor
- Blueprint Medicines Corporation
Recurrent Acute Leukemia, Recurrent B Acute Lymphoblastic Leukemia
Flotetuzumab for the Treatment of Relapsed or Refractory Advanced CD123-Positive Hematological Malignancies
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…
- Conditions
- Recurrent Acute Leukemia, Recurrent B Acute Lymphoblastic Leukemia, Recurrent Blastic Plasmacytoid Dendritic Cell Neoplasm, Recurrent Chronic Myelogenous Leukemia, BCR-ABL1 Positive
- Trial name
- NCT04681105
- Testing
- Acetaminophen, Dexamethasone, Diphenhydramine
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- City of Hope Medical Center
Non Neoplastic Condition, Precancerous Condition
Thalidomide in Treating Patients With Relapsed or Progressive Systemic Mastocytosis
RATIONALE: Thalidomide may stop the growth of systemic mastocytosis by blocking blood flow to the disease. PURPOSE: This phase II trial is studying how well thalidomide works in treating patients with relapsed or progressive…
- Trial name
- NCT00769587
- Testing
- thalidomide
- Where
- France
- Sponsor
- Centre Hospitalier Universitaire, Amiens
Cutaneous Mastocytosis, Systemic Mastocytosis
Mastocytosis From Pediatric Age to Adulthood: Local Registry of Cutaneous and Systemic Mastocytosis
Given the renewed focus on mastocytosis (recognized in Italy as a rare disease since 2018) and emerging clinical and immunogenetic knowledge, and considering the rarity of the disease and limited understanding of its progression…
- Trial name
- MaRe (NCT07142473)
- Where
- Italy
- Volunteers
- 600 people
- Sponsor
- Fondazione IRCCS Policlinico San Matteo di Pavia