Mastocytosis, Systemic clinical trials
The registry lists 56 studies for Mastocytosis, Systemic across more than 10 countries. 16 are recruiting or about to start. 29 of them are mainly about Mastocytosis, Systemic (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-12. Counting its narrower condition Leukemia, Mast-Cell too: 100 studies, 18 open.
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A study of drug A after surgery for early breast cancer
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· Sites: 4 added, including Seoul and Busan
· Enrollment: 300 → 450 participants
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Who runs them
Blueprint Medicines Corporation (10) · Novartis Pharmaceuticals (4) · University Hospital, Toulouse (3) · Cogent Biosciences, Inc. (3) · M.D. Anderson Cancer Center (3) · National Institute of Allergy and Infectious Diseases (NIAID) (3) · The First Affiliated Hospital of Soochow University (3) · Jason Robert Gotlib (3)
What is being tested here
Drugs under test in Mastocytosis, Systemic studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
| Drug | Open | Phase 3 | All |
|---|---|---|---|
| Avapritinib | 1 | 0 | 4 |
| Bezuclastinib | 1 | 0 | 2 |
| Masitinib | 1 | 2 | 2 |
| Elenestinib | 1 | 1 | 2 |
| Flixebrutinib | 1 | 0 | 1 |
| Midostaurin | 0 | 0 | 3 |
| Flotetuzumab | 0 | 0 | 2 |
| Imatinib | 0 | 0 | 2 |
| Everolimus | 0 | 0 | 1 |
| Denosumab | 0 | 1 | 1 |
Drug classes tested here Tyrosine kinase inhibitors (TKIs) 5 open
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 5
- Nov 2026A Study of MGD024 in Patients With Relapsed or Refractory Hematologic Malignancies · MacroGenics
- Mar 2027Avapritinib With Decitabine in Patients With SM-AHN · H. Lee Moffitt Cancer Center and Research Institute
- Jun 2027(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 · Blueprint Medicines Corporation
- Jul 2027Angioedema Biomarker Research Study · Foundation For Rare Disease Research
- Aug 2027Precision Medicine for Indolent Systemic Mastocytosis (PRISM) · Mayo Clinic
Who these studies take
Of the 14 Mastocytosis, Systemic studies recruiting now, 12 (86%) set no upper age limit and 2 (14%) stop at 75 or younger. 4 (29%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 1 (7%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 3 (21%) | 3 (21%) |
| 18 | 14 (100%) | 14 (100%) |
| 30 | 14 (100%) | 14 (100%) |
| 40 | 14 (100%) | 14 (100%) |
| 50 | 14 (100%) | 14 (100%) |
| 65 | 14 (100%) | 14 (100%) |
| 75 | 14 (100%) | 14 (100%) |
| 85 | 12 (86%) | 12 (86%) |
Across all 56 studies, recruiting or not: 14% stop at 75 or younger and 2% at 65; 20% take children; 0% are for women only and 0% for men only; 5% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
What changed recently
From the registry's own updates since Oct 14, 2026.
Newly registered1
- New Italian Mastocytosis RegistryNov 4, 2026NCT07917156
Studies
Recruiting first, then most recently updated. Showing 50 of 56. Browse all 56 → or search them.
- Avapritinib Rollover StudyRecruitingNCT06748001Phase 49 countries
- New Italian Mastocytosis RegistryRecruitingNCT079171561 country
- (HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic MastocytosisRecruitingNCT04910685Phase 2/325 countries
- A Non-Interventional Study in Participants With Indolent Systemic Mastocytosis (ISM) in GermanyRecruitingNCT072556381 country
- (Apex) Bezuclastinib in Patients With Advanced Systemic MastocytosisRecruitingNCT04996875Phase 214 countries
- An Observational Study in Participants With Indolent Systemic Mastocytosis (ISM)RecruitingNCT072649591 country
- Angioedema Biomarker Research StudyRecruitingNCT062106981 country
- Precision Medicine for Indolent Systemic Mastocytosis (PRISM)Not yet recruitingNCT077578931 country
- Expanded Access to Bezuclastinib for Patients With NonAdvanced Systemic Mastocytosis or Advanced Systemic MastocytosisAvailableNCT069157661 country
- Peripheral Blood KIT-D816V Mutation in Adult Systemic MastocytosisRecruitingNCT075625421 country
- Avapritinib With Decitabine in Patients With SM-AHNRecruitingNCT06327685Phase 11 country
- Mastocytosis From Pediatric Age to Adulthood: Local Registry of Cutaneous and Systemic MastocytosisRecruitingNCT071424731 country
- Study of TL-895 in Subjects With Myelofibrosis or Indolent Systemic MastocytosisRecruitingNCT04655118Phase 213 countries
- Observational Study for the Evaluation of Incidence of Systemic Mastocytosis in t(8;21) Acute Myeloid LeukemiaRecruitingNCT055044081 country
- Evaluation of the DALY on a Cohort of Patients With Indolent Systemic MastocytosisRecruitingNCT059233721 country
- Masitinib in Severe Indolent or Smoldering Systemic Mastocytosis Unresponsive to Optimal Symptomatic TreatmentRecruitingNCT04333108Phase 38 countries
- (Summit) A Study to Evaluate the Efficacy and Safety of CGT9486 Versus Placebo in Patients With Indolent or Smoldering Systemic MastocytosisActive, not recruitingNCT05186753Phase 215 countries
- (PATHFINDER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, in Patients With Advanced Systemic MastocytosisCompletedNCT03580655Phase 212 countries
- (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 MastocytosisActive, not recruitingNCT03731260Phase 213 countries
- A Study of MGD024 in Patients With Relapsed or Refractory Hematologic MalignanciesActive, not recruitingNCT05362773Phase 11 country
- Everolimus (RAD001) as Therapy for Patients With Systemic MastocytosisCompletedNCT00449748Phase 21 country
- Interest of Denosumab Treatment in Osteoporosis Associated to Systemic MastocytosisCompletedNCT03401060Phase 31 country
- Flotetuzumab for the Treatment of Relapsed or Refractory Advanced CD123-Positive Hematological MalignanciesCompletedNCT04681105Phase 11 country
- Thalidomide in Treating Patients With Relapsed or Progressive Systemic MastocytosisCompletedNCT007695871 country
- Study of Elenestinib (BLU-263) in Advanced Systemic Mastocytosis (AdvSM) and and Other KIT Altered Hematologic MalignanciesTerminatedNCT05609942Phase 17 countries
- Quality of Life and Disease-related Symptoms in Individuals With Systemic MastocytosisActive, not recruitingNCT060650071 country
- Safety and Efficacy of Subcutaneous Sarilumab in Improving the Quality of Life in People With Indolent Systemic MastocytosisCompletedNCT03770273Phase 21 country
- Managed Access Programs for PKC412, MidostaurinNo longer availableNCT05219266
- A Safety, Tolerability and PK Study of DCC-2618 in Patients With Advanced MalignanciesCompletedNCT02571036Phase 16 countries
- A Registry for Patients With Systemic Mastocytosis in ChinaStatus unknownNCT060136831 country
- (EXPLORER) Study of BLU-285 in Patients With Advanced Systemic Mastocytosis (AdvSM) and Relapsed or Refractory Myeloid MalignanciesCompletedNCT02561988Phase 12 countries
- GTB-3550 Tri-Specific Killer Engager (TriKE®) for High Risk Hematological MalignanciesTerminatedNCT03214666Phase 1/21 country
- Study of Mast Cell PrecursorsCompletedNCT000017561 country
- Retrospective Study Assessing the Effect of Avapritinib Versus Best Available Therapy in Patients With AdvSMCompletedNCT046954315 countries
- A Study of Oral AMN107 in Adults With Chronic Myelogenous Leukemia (CML) or Other Hematologic MalignanciesCompletedNCT00109707Phase 1/222 countries
- Mast Cell Connect: A Registry for Patients With MastocytosisCompletedNCT026202541 country
- Flotetuzumab in Treating Patients With Recurrent or Refractory CD123 Positive Blood CancerWithdrawnNCT03739606Phase 2
- Use of Tamoxifen in Systemic MastocytosisCompletedNCT013349961 country
- Masitinib in Severe Indolent or Smoldering Systemic MastocytosisCompletedNCT00814073Phase 32 countries
- Adaptation of the Questionnaire "Regarding Patient's Quality of Life With Mastocytosis" in the French LanguageCompletedNCT036328111 country
- Diagnostic Value of Bone Marrow Tryptase in Systemic MastocytosisCompletedNCT02441166Not applicable2 countries
- A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AK002CompletedNCT02808793Phase 11 country
- Brentuximab Vedotin in Treating Patients With Advanced Systemic Mastocytosis or Mast Cell LeukemiaCompletedNCT01807598Phase 21 country
- Efficacy and Safety of Midostaurin in Patients With Aggressive Systemic Mastocytosis or Mast Cell LeukemiaCompletedNCT00782067Phase 212 countries
- Phase 2 Midostaurin in Aggressive Systemic Mastocytosis and Mast Cell LeukemiaCompletedNCT00233454Phase 21 country
- Ibrutinib in Treating Patients With Advanced Systemic MastocytosisTerminatedNCT02415608Phase 21 country
- Imatinib Mesylate in Patients With Various Types of Malignancies Involving Activated Tyrosine Kinase EnzymesCompletedNCT00171912Phase 21 country
- Patient-Reported Outcome Questionnaire for Systemic MastocytosisCompletedNCT023802221 country
- Treatment of Indolent Systemic Mastocytosis With PA101CompletedNCT02478957Phase 25 countries
- Cladribine Plus Pegylated Interpheron Alfa-2a in Systemic MastocytosisStatus unknownNCT01602939Phase 2/31 country