5 completed, 4 recruiting, 3 unknown, 1 active not recruiting. 6 Phase 2, 3 Phase 3, 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 6 | Completed 2, Unknown 2, Active not recruiting 1, Recruiting 1 |
| Phase 3 | 3 | Completed 1, Recruiting 1, Unknown 1 |
| Phase 1 | 1 | Completed 1 |
Follow Indolent Systemic Mastocytosis: new trials by email Literature Review of Indolent Systemic Mastocytosis trials Research Report on Indolent Systemic Mastocytosis
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| An Observational Study in Participants With Indolent Systemic Mastocytosis (ISM) Indolent Systemic Mastocytosis | Recruiting | Blueprint Medicines Corporation | 150 | 2026-04-15 | 2026-09-04 | |
| A Non-Interventional Study in Participants With Indolent Systemic Mastocytosis (ISM) in Germany Avapritinib - Indolent Systemic Mastocytosis | Recruiting | Blueprint Medicines Corporation | 80 | 2025-12-09 | 2026-10-07 | |
| Angioedema Biomarker Research Study Venipuncture - Angioedema, Angioedemas, Hereditary | Recruiting | Foundation For Rare Disease Research | 600 | 2024-01-15 | 2026-08-24 | |
| (HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis Elenestinib, Placebo - Indolent Systemic Mastocytosis, Smoldering Systemic Mastocytosis | Phase 2 / Phase 3 | Recruiting | Blueprint Medicines Corporation | 534 | 2021-11-30 | 2026-10-07 |
| Study of TL-895 in Subjects With Myelofibrosis or Indolent Systemic Mastocytosis TL-895, Placebo - Myelofibrosis, Indolent Systemic Mastocytosis | Phase 2 | Unknown | Telios Pharma, Inc. | 121 | 2020-10-22 | 2024-05-10 |
| Masitinib in Severe Indolent or Smoldering Systemic Mastocytosis Unresponsive to Optimal Symptomatic Treatment Masitinib, Placebo, Best Supportive Care - Indolent Systemic Mastocytosis | Phase 3 | Unknown | AB Science | 140 | 2020-07-01 | 2023-05-06 |
| Safety and Efficacy of Subcutaneous Sarilumab in Improving the Quality of Life in People With Indolent Systemic Mastocytosis sarilumab, Placebo - Indolent Systemic Mastocytosis | Phase 2 | Completed | National Institute of Allergy and Infectious Diseases (NIAID) | 21 | 2019-06-19 | 2024-12-17 |
| (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 Avapritinib, Placebo - Indolent Systemic Mastocytosis | Phase 2 | Active not recruiting | Blueprint Medicines Corporation | 251 | 2019-04-16 | 2026-10-02 |
| Adaptation of the Questionnaire "Regarding Patient's Quality of Life With Mastocytosis" in the French Language A structured interview with every patient - Cutaneous Mastocytosis, Indolent Systemic Mastocytosis | Completed | University Hospital, Toulouse | 30 | 2018-11-14 | 2019-07-17 | |
| A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AK002 AK002 - Indolent Systemic Mastocytosis | Phase 1 | Completed | Allakos Inc. | 25 | 2016-06-01 | 2019-03-13 |
| Treatment of Indolent Systemic Mastocytosis With PA101 PA101, Placebo - Mastocytosis, Systemic Mastocytosis | Phase 2 | Completed | Patara Pharma | 41 | 2015-06-01 | 2016-09-29 |
| Midostaurin in Indolent Systemic Mastocytosis Midostaurin, - Indolent Systemic Mastocytosis | Phase 2 | Unknown | University Medical Center Groningen | 20 | 2013-08-01 | 2015-01-19 |
| Masitinib in Severe Indolent or Smoldering Systemic Mastocytosis Masitinib, Placebo, Best Supportive Care - Indolent Systemic Mastocytosis | Phase 3 | Completed | AB Science | 135 | 2008-12-01 | 2019-12-03 |
Mastocytosis, Systemic (13) · Mastocytosis (8) · Blushing (2) · Piebaldism (2) · Pruritus (2) · Angioedema (1) · Angioedemas, Hereditary (1) · Mastocytosis, Cutaneous (1) · Primary Myelofibrosis (1) · Urticaria (1)
Immune System Diseases (13) · Mast Cell Activation Disorders (13) · Neoplasms (13) · Neoplasms by Histologic Type (13) · Neoplasms, Connective Tissue (13) · Neoplasms, Connective and Soft Tissue (13) · Mastocytosis (5) · Skin Diseases (4) · Skin and Connective Tissue Diseases (4) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (3) · Genetic Diseases, Inborn (3) · Albinism (2) · Amino Acid Metabolism, Inborn Errors (2) · Behavior (2) · Communication (2)
avapritinib (2) · masitinib (2) · AK002 (1) · Phlebotomy (1) · midostaurin (1) · sarilumab (1)
Blueprint Medicines Corporation (4) · AB Science (2) · Allakos Inc. (1) · Foundation For Rare Disease Research (1) · National Institute of Allergy and Infectious Diseases (NIAID) (1) · Patara Pharma (1) · Telios Pharma, Inc. (1) · University Hospital, Toulouse (1) · University Medical Center Groningen (1)
France (7) · Germany (7) · United States (7) · Netherlands (5) · Italy (4) · Spain (4) · Belgium (3) · Poland (3) · United Kingdom (3) · Australia (2) · Brazil (2) · Denmark (2) · Norway (2) · Romania (2) · Sweden (2)
Cristina Bulai Livideanu, MD (1) · Cristina Bulai Livideanu, MD, MSc (1) · Frank Siebenhaar, MD (1) · Hirsh D Komarow, M.D. (1) · J.C. Kluin-Nelemans, MD, PhD (1) · Lili Wan, PhD (1) · Marcus Maurer, MD (1) · Olivier Lortholary, MD, PhD (1)
PMID 28069279, PMID 29890238, PMID 34663404, PMID 36048877, PMID 38320129, PMID 40529483, PMID 41244098, PMID 41690487
2016 (1), 2018 (1)