Clinical trials: Aggressive Systemic Mastocytosis (ASM)
Advanced Systemic Mastocytosis
Study of Elenestinib (BLU-263) in Advanced Systemic Mastocytosis (AdvSM) and and Other KIT Altered Hematologic Malignancies
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.
- Trial name
- AZURE (NCT05609942)
- Testing
- BLU-263, Azacitidine
- Where
- 11 sites in United States, Belgium and 5 more
- Volunteers
- 9 people
- Sponsor
- Blueprint Medicines Corporation
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.
Advanced Systemic Mastocytosis, Aggressive Systemic Mastocytosis
Retrospective Study Assessing the Effect of Avapritinib Versus Best Available Therapy in Patients With AdvSM
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.
- Conditions
- Advanced Systemic Mastocytosis, Aggressive Systemic Mastocytosis, Systemic Mastocytosis With an Associated Hematological Neoplasm, Mast Cell Leukemia
- Trial name
- NCT04695431
- Where
- 6 sites in United States, Austria and 3 more
- Volunteers
- 317 people
- Sponsor
- Blueprint Medicines Corporation
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
Leukemia
Efficacy and Safety of Midostaurin in Patients With Aggressive Systemic Mastocytosis or Mast Cell Leukemia
The purpose of this study was to determine the efficacy and safety of twice daily (bid) oral midostaurin in patients with Aggressive Systemic Mastocytosis (ASM) or Mast Cell Leukemia (MCL) with or without an Associated…
- Trial name
- NCT00782067
- Testing
- Midostaurin (PKC412)
- Where
- 29 sites in United States, Australia and 10 more
- Volunteers
- 116 people
- Sponsor
- Novartis Pharmaceuticals
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
FMS-Like Tyrosine Kinase 3 (FLT3)-Mutated Acute Myeloid Leukemia, Acute Myeloid Leukemia
Managed Access Programs for PKC412, Midostaurin
The purpose of this registration is to list Managed Access Programs (MAPs) related to PKC4, Midostaurin.
- Conditions
- FMS-Like Tyrosine Kinase 3 (FLT3)-Mutated Acute Myeloid Leukemia, Acute Myeloid Leukemia, Aggressive Systemic Mastocytosis, Mast Cell Leukemia
- Trial name
- NCT05219266
- Testing
- midostaurin
- Sponsor
- Novartis Pharmaceuticals
High-risk Myelodysplastic Syndromes, Acute Myelogenous Leukemia
GTB-3550 Tri-Specific Killer Engager (TriKE®) for High Risk Hematological Malignancies
This is a multi-center Phase I/II clinical trial of GTB-3550 (CD16/IL-15/CD33) tri-specific killer cell engager (TriKE®) for the treatment of CD33-expressing high risk myelodysplastic syndromes, refractory/relapsed acute myeloid…
- Conditions
- High-risk Myelodysplastic Syndromes, Acute Myelogenous Leukemia, Systemic Mastocytosis, Mast Cell Leukemia
- Trial name
- NCT03214666
- Testing
- GTB-3550 TriKE® Phase I, GTB-3550 TriKE® Phase II
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- GT Biopharma, Inc.
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.
Aggressive Systemic Mastocytosis, Systemic Mastocytosis-associated Hematologic Non-mast Cell Disease
(EXPLORER) Study of BLU-285 in Patients With Advanced Systemic Mastocytosis (AdvSM) and Relapsed or Refractory Myeloid Malignancies
This is a Phase 1, open-label, dose-escalation study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and antineoplastic activity of avapritinib (also known as BLU-285), administered…
- Conditions
- Aggressive Systemic Mastocytosis, Systemic Mastocytosis-associated Hematologic Non-mast Cell Disease, Mast Cell Leukemia, Relapsed or Refractory Myeloid Malignancies
- Trial name
- NCT02561988
- Testing
- Avapritinib
- Where
- 11 sites in United States, United Kingdom
- Volunteers
- 86 people
- Sponsor
- Blueprint Medicines Corporation
Mastocytosis
Mast Cell Connect: A Registry for Patients With Mastocytosis
The Mast Cell Connect Registry is a voluntary, observational database that will capture demographic, socioeconomic, and disease information directly from patients with mastocytosis via a secure web-based tool.
- Trial name
- NCT02620254
- Where
- United States
- Volunteers
- 743 people
- Sponsor
- Blueprint Medicines Corporation
Aggressive Systemic Mastocytosis (ASM), SM w Assoc Clonal Hema Non-mast Cell Lineage Disease (SM-AHNMD)
Patient-Reported Outcome Questionnaire for Systemic Mastocytosis
The purpose of this study is to explore the key symptoms of individuals with systemic mastocytosis. Interviews of enrolled individuals will be conducted to learn about the disease symptoms and condition.
- Conditions
- Aggressive Systemic Mastocytosis (ASM), SM w Assoc Clonal Hema Non-mast Cell Lineage Disease (SM-AHNMD), Mast Cell Leukemia (MCL), Smoldering Systemic Mastocytosis (SSM)
- Trial name
- NCT02380222
- Testing
- Interview
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Adelphi Values LLC
Gastrointestinal Stromal Tumors, Advanced Systemic Mastocytosis
A Safety, Tolerability and PK Study of DCC-2618 in Patients With Advanced Malignancies
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…
- Conditions
- Gastrointestinal Stromal Tumors, Advanced Systemic Mastocytosis, Advanced Cancers
- Trial name
- NCT02571036
- Testing
- DCC-2618
- Where
- 26 sites in United States, Canada and 4 more
- Volunteers
- 282 people
- Sponsor
- Deciphera Pharmaceuticals, LLC