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| ID | Type | Description | Link |
|---|---|---|---|
| 2017-004836-13 | EudraCT Number |
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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 hematologic neoplasm (SM-AHN), and mast cell leukemia (MCL)
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Avapritinib | Experimental | Avapritinib will be administered as an immediate release tablet, orally, continuously, in 28-day cycles |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Avapritinib | Drug | Avapritinib tablet |
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| Measure | Description | Time Frame |
|---|---|---|
| Overall Response Rate (ORR) Based on Modified International Working Group-Myeloproliferative Neoplasms Research and Treatment and European Competence Network on Mastocytosis (mIWG-MRT-ECNM) Response Criteria | ORR was defined as the percentage of participants with a confirmed best response of complete remission (CR), complete remission with partial recovery of peripheral blood counts (CRh), partial remission (PR), or clinical improvement (CI) by mIWG-MRT-ECNM criteria. The mIWG-MRT-ECNM criteria were based on findings from bone marrow biopsy and aspirate, and peripheral blood smear; bone marrow cytogenetics; other extracutaneous tissue biopsies (when available); liver and spleen imaging; and other clinical and laboratory parameters. | Baseline through Month 65 |
| Measure | Description | Time Frame |
|---|---|---|
| Mean Change From Baseline in Advanced Systemic Mastocytosis-Symptom Assessment Form (AdvSM-SAF) Total Symptom Score (TTS) | The AdvSM-SAF is a 10-item questionnaire that assesses eight symptoms specific to AdvSM. All eight symptoms are scored on a scale of 0 (absence of symptoms) to 10 (more severe symptoms) (up to 80 points maximum). Each symptom contributes to the TSS equally. The TSS was generated based on average scores for each 7-day period. An increase in score from 0 (no symptoms) to 80 (worst symptoms represents a worse symptoms outcome. Participants completed the AdvSM-SAF daily using an electronic diary (eDiary). Each cycle is 28 days long. |
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Key Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Stanford Cancer Institute | Stanford | California | 94305 | United States | ||
| Rush University Medical Center |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41604606 | Derived | Gotlib J, Reiter A, Radia DH, Alvarez-Twose I, Deininger MW, George TI, Panse J, Mital A, Pettit KM, Vannucchi AM, Platzbecker U, Hermine O, Elshoury A, Livideanu CB, Mesa R, Ustun C, Triggiani M, Dybedal I, Jurcic JG, Zanotti R, Oh ST, Yacoub A, Hexner EO, Bose P, Lee SG, Sperr WR, Griffiths EA, Butler M, Lubke J, Bidollari I, Lin HM, Rylova S, Dimitrijevic S, Munoz-Gonzalez JI, DeAngelo DJ. Efficacy and safety of avapritinib in advanced systemic mastocytosis: 4-year follow-up of the PATHFINDER study. Blood Adv. 2026 May 26;10(10):3676-3689. doi: 10.1182/bloodadvances.2025017519. | |
| 35790816 |
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The Avapritinib group (N=107) includes all participants treated at a starting dose of 200 milligrams (mg) once daily (N=105) and 2 participants treated at a starting dose of 100 mg once daily. One participant was treated at a starting dose of 100 mg per previous protocol amendment, and 1 participant was treated at a starting dose of 100 mg per principal investigator decision.
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| ID | Title | Description |
|---|---|---|
| FG000 | Avapritinib (100 mg) | Avapritinib was administered once daily as an immediate-release tablet, orally, in 28-day cycles. |
| FG001 | Avapritinib (200 mg) | Avapritinib was administered once daily as an immediate-release tablet, orally, in 28-day cycles. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Apr 21, 2020 | Dec 17, 2025 |
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| Baseline up to Month 6 (Cycle 6, Day 1) |
| ORR Based on mIWG-MRT-ECNM Response Criteria as Assessed by Local Investigator | ORR was defined as the percentage of participants with a confirmed best response of CR, CRh, PR, or CI by mIWG-MRT-ECNM criteria. The mIWG-MRT-ECNM criteria were based on findings from bone marrow biopsy and aspirate, and peripheral blood smear; bone marrow cytogenetics; other extracutaneous tissue biopsies (when available); liver and spleen imaging; and other clinical and laboratory parameters. | Baseline through Month 19 |
| Time-to-Response (TTR) | TTR was defined as the time from first dose to the time of initial evaluation of clinical improvement (CI) or better. Here, 'Overall Number of Participants Analyzed' signifies those participants with a confirmed best response of CR, CRh, PR, and CI by mIWG-MRT-ECNM criteria. | Baseline through Month 65 |
| Duration of Response (DOR) | DOR was defined as the time from initial documentation of a CI or better to the time of initial documentation of confirmed progressive disease (PD) or death due to any cause, whichever occurred first. For responders who had not progressed or died at the time of analysis, DOR was censored at the last response assessment that was stable disease (SD) or better. Here, 'Overall Number of Participants Analyzed' signifies those participants with a confirmed best response of CR, CRh, PR, and CI by mIWG-MRT-ECNM criteria. | Baseline through Month 65 |
| Progression-free Survival (PFS) | PFS was defined as time from first dose to the time of initial documentation of confirmed PD or death due to any cause, whichever occurred first. Participants who had not progressed or died at the time of analysis were censored at the last response assessment that was SD or better. | Baseline through Month 65 |
| Overall Survival (OS) | OS was defined as time from first dose to the time of death due to any cause. Participants who were known to be alive or are lost to follow-up were censored at the last time point they were known to be alive. | Baseline through Month 65 |
| Objective Response Rate | The objective response rate was defined as the number of participants with a confirmed best response of morphologic complete remission, morphologic complete remission with partial recovery of peripheral blood counts, or morphologic partial remission by pure pathologic response (PPR) criteria. The PPR criteria are a modification of the mIWG-MRT-ECNM criteria that define deep responses where direct measure of the disease burden determined the response and focus on objective changes (bone marrow mast cell burden, serum tryptase, and complete blood count). | Baseline through Month 65 |
| Clinical Benefit Rate | The clinical benefit rate was defined as the percentage of participants with a confirmed best response of CR, CRh, PR, CI, and SD by mIWG-MRT-ECNM criteria. | Baseline through Month 65 |
| Percent Change From Baseline in Bone Marrow Mast Cells | Bone marrow biopsies and aspirates and peripheral blood smears were obtained for systemic mastocytosis response assessment according to mIWG-MRT-ECNM criteria. Each cycle is 28 days long. | Baseline up to Month 65 |
| Percent Change From Baseline in Serum Tryptase | Blood samples were collected to characterize the change in serum tryptase concentration during treatment with avapritinib. Each cycle is 28 days long. | Baseline up to Month 65 |
| Percent Change From Baseline in V-kit Hardy-Zuckerman 4 Feline Sarcoma Viral Oncogene Homolog Aspartate 816 Valine (KIT D816V) Mutation Burden | Bone marrow aspirate and PB samples were collected to characterize the KIT D816V mutation allele fraction. Each cycle is 28 days long. | Baseline up to Month 65 |
| Percent Change From Baseline in Liver Volume by Imaging | Assessment of liver volume was performed using serial imaging with magnetic resonance imaging (MRI). Response was defined as resolution of palpable hepatomegaly (CR). Each cycle is 28 days long. | Baseline up to Month 65 |
| Percent Change From Baseline in Spleen Volume by Imaging | Assessment of spleen volume was performed using serial imaging with MRI. Response was defined as ≥35% reduction in spleen volume (PR) or resolution of palpable splenomegaly (CR). Each cycle is 28 days long. | Baseline up to Month 65 |
| Mean Change From Baseline in AdvSM-SAF Skin and Gastrointestinal Domain Scores at Month 10 | The AdvSM-SAF is a 10-item questionnaire that assesses symptoms and functional domains specific to AdvSM. Eight symptoms are scored on a scale of 0 to 10 for severity and 2 symptoms (vomiting and diarrhea) are scored for number of episodes. The skin domain is scored on a scale of 0-30, where 0 represents an absence of symptoms and 30 the most severe symptom experience. Similarly, the gastrointestinal domain is scored on a scale of 0-40, where 0 represents an absence of symptoms and 40 the most severe symptom experience. Skin and gastrointestinal domain scores were generated based on average scores for each 7-day period. An increase in score represents a worse symptoms outcome. Participants completed the AdvSM-SAF daily using an eDiary. | Baseline, Month 10 |
| Mean Change From Baseline in AdvSM-SAF Individual Symptom Scores (Severity) at Month 10 | The AdvSM-SAF is a 10-item questionnaire that assesses symptoms and functional domains specific to AdvSM. Individual symptom scores are generated based on average scores for each 7-day period. Eight symptoms are scored on a scale of 0 to 10 for severity. An increase in score represents a worse symptoms outcome. Participants completed the AdvSM-SAF daily using an eDiary. | Baseline, Month 10 |
| Mean Change From Baseline in AdvSM-SAF Individual Symptom Scores (Episodes) at Month 10 | The AdvSM-SAF is a 10-item questionnaire that assesses symptoms and functional domains specific to AdvSM. Individual symptom scores are generated based on average scores for each 7-day period. Two symptoms (vomiting and diarrhea) are scored for number of episodes. An increase in score represents a worse symptoms outcome. Participants completed the AdvSM-SAF daily using an eDiary. | Baseline, Month 10 |
| Change From Baseline in Patient's Global Impression of Symptom Severity (PGIS) | The PGIS is a single-item scale that assesses a participant's perception of disease symptoms at a point in time. Scores range from 0 to 4 points, with higher values representing worse symptom outcomes. It is widely used to evaluate a participant's overall sense of whether a treatment has been beneficial. Each cycle is 28 days long. | Baseline, Cycle 1, Day 15 and Day 1 of Cycle 2, Cycle 3, Cycle 5, Cycle 7, Cycle 9, Cycle 11, Cycle 14, and Cycle 17 (28 days/cycle) |
| Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30) | Quality of life was assessed using EORTC QLQ-C30, which is a 30-item questionnaire that includes 5 functional domains (physical, cognitive, role, emotional, and social) and a global health status scale. Twenty-eight questions are scored from 1 (not at all) to 4 (very much), while the other 2 are scored from 1 (very poor) to 7 (excellent). The calculated average is standardized using a linear transformation to a standardized scale of 0 to 100, with a decrease in score representing a decrease in quality of life. Each cycle is 28 days long. | Baseline, Cycle 1, Day 15 and Day 1 of Cycle 2, Cycle 3, Cycle 5, Cycle 7, Cycle 9, Cycle 11, Cycle 14, and Cycle 17 (28 days/cycle) |
| Number of Participants Experiencing Treatment-emergent Adverse Events (TEAE) | A TEAE was defined as any adverse events that occurred between the first dose of a study drug through 30 days after the last dose of any study drug. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. | Baseline through Month 65 |
| Maximum Plasma Concentration (Cmax) of a Single Dose of Avapritinib | Blood samples were collected at specified timepoints. Results reported as nanograms/milliliter (ng/mL). | Day 1 of Cycle 1 (1 hour postdose) (28 days/cycle) |
| Correlation Between TSS and Serum Tryptase | The change in baseline for AdvSM-SAF TSS was correlated with the change in baseline for serum tryptase. Spearman correlation coefficients were performed with corresponding scatter plots on change from baseline. | Baseline, Month 10 |
| Chicago |
| Illinois |
| 60612 |
| United States |
| The University of Kansas Cancer Center | Westwood | Kansas | 66205 | United States |
| Dana Farber Cancer Institute | Boston | Massachusetts | 02215 | United States |
| University of Michigan | Ann Arbor | Michigan | 48109 | United States |
| Washington University School of Medicine | St Louis | Missouri | 63110 | United States |
| Roswell Park Comprehensive Cancer Center | Buffalo | New York | 14263 | United States |
| Herbert Irving Comprehensive Cancer Center | New York | New York | 10032 | United States |
| University of Pennsylvania, Abramson Cancer Center | Philadelphia | Pennsylvania | 19104 | United States |
| University of Texas, MD Anderson Cancer Center | Houston | Texas | 77030 | United States |
| Mays Cancer Center | San Antonio | Texas | 78229 | United States |
| Huntsman Cancer Institute | Salt Lake City | Utah | 84112 | United States |
| Medizinische Universität Wien, Universitätsklinik für Innere Medizin I, Klinische Abteilung für Hämatologie und Hämostaseologie | Vienna | 1090 | Austria |
| St. Michael's Hospital | Toronto | Ontario | M5B 1W8 | Canada |
| Odense University Hospital, Department of Haematology | Odense | DK-5000 | Denmark |
| Hôpital Necker-Enfants Malades | Paris | 75015 | France |
| CHU Toulouse - Hôpital Larrey | Toulouse | 31059 | France |
| Uniklinik RWTH Aachen, Klinik für Hämatologie, Onkologie, Hämostaseologie und Stammzelltranslplantation | Aachen | 52074 | Germany |
| Universitätsklinikum Hamburg-Eppendorf, Zentrum für Onkologie | Hamburg | 20246 | Germany |
| Universitätsklinikum Leipzig, Medizinische Klinik und Poliklinik I - Hämatologie und Zelltherapie, lnternistische Onkologie, Hämostaseologie | Leipzig | 04103 | Germany |
| Universitätsmedizin Mannheim III. Medizinische Klinik | Mannheim | 68167 | Germany |
| Klinik und Poliklinik für Innere Medizin III, Klinikum rechts der Isar der TU München | Munich | 81675 | Germany |
| Azienda Ospedaliero-Universitaria Careggi, CRIMM - Centro di Ricerca ed Innovazione per le Malattie Mieloproliferative | Florence | 50134 | Italy |
| A.O. OO.RR. S.Giovanni di Dio e Ruggi d'Aragona, University of Salerno | Salerno | 84131 | Italy |
| Centro Ricerche Cliniche di Verona - Azienda Ospedaliera Universitaria Integrata di Verona | Verona | 37134 | Italy |
| University Medical Center Groningen (UMCG) | Groningen | 9713 GZ | Netherlands |
| Oslo University Hospital-Rikshospitalet, Hematology | Oslo | 0372 | Norway |
| Uniwersyteckie Centrum Kliniczne, Klinika Hematologii i Transplantologii | Gdansk | 80-214 | Poland |
| Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wrocławiu, Klinice Hematologii, Nowotworów Krwi i Transplantacji Szpiku | Wroclaw | 50-367 | Poland |
| lnstituto de Estudios de Mastocitosis de Castilla la Mancha, Hospital Virgen del Valle - Complejo Hospitalario de Toledo | Toledo | 45071 | Spain |
| Beatson West of Scotland Cancer Centre - NHS Greater Glasgow and Clyde | Glasgow | G12 0YN | United Kingdom |
| Guy's and St Thomas' NHS Foundation Trust - Guy's Hospital | London | SE1 9RT | United Kingdom |
| Derived |
| Reiter A, Gotlib J, Alvarez-Twose I, Radia DH, Lubke J, Bobbili PJ, Wang A, Norregaard C, Dimitrijevic S, Sullivan E, Louie-Gao M, Schwaab J, Galinsky IA, Perkins C, Sperr WR, Sriskandarajah P, Chin A, Sendhil SR, Duh MS, Valent P, DeAngelo DJ. Efficacy of avapritinib versus best available therapy in the treatment of advanced systemic mastocytosis. Leukemia. 2022 Aug;36(8):2108-2120. doi: 10.1038/s41375-022-01615-z. Epub 2022 Jul 5. |
| 35640224 | Derived | Reiter A, Schwaab J, DeAngelo DJ, Gotlib J, Deininger MW, Pettit KM, Alvarez-Twose I, Vannucchi AM, Panse J, Platzbecker U, Hermine O, Dybedal I, Lin HM, Rylova SN, Ehlert K, Dimitrijevic S, Radia DH. Efficacy and safety of avapritinib in previously treated patients with advanced systemic mastocytosis. Blood Adv. 2022 Nov 8;6(21):5750-5762. doi: 10.1182/bloodadvances.2022007539. |
| 34873345 | Derived | Gotlib J, Reiter A, Radia DH, Deininger MW, George TI, Panse J, Vannucchi AM, Platzbecker U, Alvarez-Twose I, Mital A, Hermine O, Dybedal I, Hexner EO, Hicks LK, Span L, Mesa R, Bose P, Pettit KM, Heaney ML, Oh ST, Sen J, Lin HM, Mar BG, DeAngelo DJ. Efficacy and safety of avapritinib in advanced systemic mastocytosis: interim analysis of the phase 2 PATHFINDER trial. Nat Med. 2021 Dec;27(12):2192-2199. doi: 10.1038/s41591-021-01539-8. Epub 2021 Dec 6. |
| Received at Least 1 Dose of Study Drug | Safety Population |
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| Pure Pathologic Response-evaluable Population |
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| Response-evaluable Population |
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| COMPLETED |
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| NOT COMPLETED |
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Safety Population: all participants who received at least 1 dose of avapritinib.
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| ID | Title | Description |
|---|---|---|
| BG000 | Avapritinib | Avapritinib was administered once daily as an immediate-release tablet, orally, in 28-day cycles. |
| Units | Counts |
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| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
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| Age, Continuous | Mean | Standard Deviation | Years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Primary | Overall Response Rate (ORR) Based on Modified International Working Group-Myeloproliferative Neoplasms Research and Treatment and European Competence Network on Mastocytosis (mIWG-MRT-ECNM) Response Criteria | ORR was defined as the percentage of participants with a confirmed best response of complete remission (CR), complete remission with partial recovery of peripheral blood counts (CRh), partial remission (PR), or clinical improvement (CI) by mIWG-MRT-ECNM criteria. The mIWG-MRT-ECNM criteria were based on findings from bone marrow biopsy and aspirate, and peripheral blood smear; bone marrow cytogenetics; other extracutaneous tissue biopsies (when available); liver and spleen imaging; and other clinical and laboratory parameters. | Response-evaluable Population: all participants who received ≥1 dose of avapritinib, were deemed evaluable per mIWG-MRT-ECNM criteria at baseline as assessed by study steering committee review and had 1 of the following conditions: ≥2 complete postbaseline bone marrow assessments and had been on study for ≥6 cycles; had an end of study visit. | Posted | Number | 95% Confidence Interval | Percentage of Participants | Baseline through Month 65 |
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| Secondary | Mean Change From Baseline in Advanced Systemic Mastocytosis-Symptom Assessment Form (AdvSM-SAF) Total Symptom Score (TTS) | The AdvSM-SAF is a 10-item questionnaire that assesses eight symptoms specific to AdvSM. All eight symptoms are scored on a scale of 0 (absence of symptoms) to 10 (more severe symptoms) (up to 80 points maximum). Each symptom contributes to the TSS equally. The TSS was generated based on average scores for each 7-day period. An increase in score from 0 (no symptoms) to 80 (worst symptoms represents a worse symptoms outcome. Participants completed the AdvSM-SAF daily using an electronic diary (eDiary). Each cycle is 28 days long. | Safety Population: all participants who received ≥1 dose of avapritinib. Here, 'Number of Participants Analyzed' signifies those participants evaluable for this outcome measure at the specified timepoints. | Posted | Mean | Standard Deviation | Score on a Scale | Baseline up to Month 6 (Cycle 6, Day 1) |
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| Secondary | ORR Based on mIWG-MRT-ECNM Response Criteria as Assessed by Local Investigator | ORR was defined as the percentage of participants with a confirmed best response of CR, CRh, PR, or CI by mIWG-MRT-ECNM criteria. The mIWG-MRT-ECNM criteria were based on findings from bone marrow biopsy and aspirate, and peripheral blood smear; bone marrow cytogenetics; other extracutaneous tissue biopsies (when available); liver and spleen imaging; and other clinical and laboratory parameters. | Response-evaluable Population: all participants who received ≥1 dose of avapritinib, were deemed evaluable per mIWG-MRT-ECNM criteria at baseline as assessed by local investigator and had 1 of the following conditions: ≥2 complete postbaseline bone marrow assessments and had been on study for ≥6 cycles; had an end of study visit. Here, 'Overall Number of Participants Analyzed' signifies those participants evaluable for this outcome measure. | Posted | Number | 95% Confidence Interval | Percentage of Participants | Baseline through Month 19 |
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| Secondary | Time-to-Response (TTR) | TTR was defined as the time from first dose to the time of initial evaluation of clinical improvement (CI) or better. Here, 'Overall Number of Participants Analyzed' signifies those participants with a confirmed best response of CR, CRh, PR, and CI by mIWG-MRT-ECNM criteria. | Response-evaluable Population: all participants who received ≥1 dose of avapritinib, were deemed evaluable per mIWG-MRT-ECNM criteria at baseline as assessed by study steering committee review and had 1 of the following conditions: ≥2 complete postbaseline bone marrow assessments and had been on study for ≥6 cycles; had an end of study visit. | Posted | Median | Full Range | Months | Baseline through Month 65 |
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| Secondary | Duration of Response (DOR) | DOR was defined as the time from initial documentation of a CI or better to the time of initial documentation of confirmed progressive disease (PD) or death due to any cause, whichever occurred first. For responders who had not progressed or died at the time of analysis, DOR was censored at the last response assessment that was stable disease (SD) or better. Here, 'Overall Number of Participants Analyzed' signifies those participants with a confirmed best response of CR, CRh, PR, and CI by mIWG-MRT-ECNM criteria. | Response-evaluable Population: all participants who received ≥1 dose of avapritinib, were deemed evaluable per mIWG-MRT-ECNM criteria at baseline as assessed by study steering committee review and had 1 of the following conditions: ≥2 complete postbaseline bone marrow assessments and had been on study for ≥6 cycles; had an end of study visit. | Posted | Median | 95% Confidence Interval | Months | Baseline through Month 65 |
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| Secondary | Progression-free Survival (PFS) | PFS was defined as time from first dose to the time of initial documentation of confirmed PD or death due to any cause, whichever occurred first. Participants who had not progressed or died at the time of analysis were censored at the last response assessment that was SD or better. | Response-evaluable Population: all participants who received ≥1 dose of avapritinib, were deemed evaluable per mIWG-MRT-ECNM criteria at baseline as assessed by study steering committee review and had 1 of the following conditions: ≥2 complete postbaseline bone marrow assessments, and had been on study for ≥6 cycles; had an end of study visit. | Posted | Median | 95% Confidence Interval | Months | Baseline through Month 65 |
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| Secondary | Overall Survival (OS) | OS was defined as time from first dose to the time of death due to any cause. Participants who were known to be alive or are lost to follow-up were censored at the last time point they were known to be alive. | Safety Population: all participants who received ≥1 dose of avapritinib. | Posted | Median | 95% Confidence Interval | Months | Baseline through Month 65 |
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| Secondary | Objective Response Rate | The objective response rate was defined as the number of participants with a confirmed best response of morphologic complete remission, morphologic complete remission with partial recovery of peripheral blood counts, or morphologic partial remission by pure pathologic response (PPR) criteria. The PPR criteria are a modification of the mIWG-MRT-ECNM criteria that define deep responses where direct measure of the disease burden determined the response and focus on objective changes (bone marrow mast cell burden, serum tryptase, and complete blood count). | Pure Pathologic Response-evaluable Population: all participants who received ≥1 dose of avapritinib and had 1 of the following conditions: ≥2 complete postbaseline bone marrow assessments and had been on study for ≥6 cycles; had an end of study visit. | Posted | Number | 95% Confidence Interval | Percentage of Participants | Baseline through Month 65 |
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| Secondary | Clinical Benefit Rate | The clinical benefit rate was defined as the percentage of participants with a confirmed best response of CR, CRh, PR, CI, and SD by mIWG-MRT-ECNM criteria. | Response-evaluable Population: all participants who received ≥1 dose of avapritinib, were deemed evaluable per mIWG-MRT-ECNM criteria at baseline as assessed by study steering committee review and had 1 of the following conditions: ≥2 complete postbaseline bone marrow assessments and had been on study for ≥6 cycles; had an end of study visit. | Posted | Number | 95% Confidence Interval | Percentage of Participants | Baseline through Month 65 |
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| Secondary | Percent Change From Baseline in Bone Marrow Mast Cells | Bone marrow biopsies and aspirates and peripheral blood smears were obtained for systemic mastocytosis response assessment according to mIWG-MRT-ECNM criteria. Each cycle is 28 days long. | Safety Population: all participants who received ≥1 dose of avapritinib. Here, 'Number Analyzed' signifies those participants evaluable for this outcome measure at the specified timepoint. | Posted | Mean | Standard Deviation | Percent Change | Baseline up to Month 65 |
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| Secondary | Percent Change From Baseline in Serum Tryptase | Blood samples were collected to characterize the change in serum tryptase concentration during treatment with avapritinib. Each cycle is 28 days long. | Safety Population: all participants who received ≥1 dose of avapritinib. Here, 'Number Analyzed' signifies those participants evaluable for this outcome measure at the specified timepoint. | Posted | Mean | Standard Deviation | Percent Change | Baseline up to Month 65 |
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| Secondary | Percent Change From Baseline in V-kit Hardy-Zuckerman 4 Feline Sarcoma Viral Oncogene Homolog Aspartate 816 Valine (KIT D816V) Mutation Burden | Bone marrow aspirate and PB samples were collected to characterize the KIT D816V mutation allele fraction. Each cycle is 28 days long. | Safety Population: all participants who received ≥1 dose of avapritinib. Here, 'Number Analyzed' signifies those participants evaluable for this outcome measure at the specified timepoint. | Posted | Mean | Standard Deviation | Percent Change | Baseline up to Month 65 |
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| Secondary | Percent Change From Baseline in Liver Volume by Imaging | Assessment of liver volume was performed using serial imaging with magnetic resonance imaging (MRI). Response was defined as resolution of palpable hepatomegaly (CR). Each cycle is 28 days long. | Safety Population: all participants who received ≥1 dose of avapritinib. Here, ' Number Analyzed' signifies those participants evaluable for this outcome measure at the specified timepoint. | Posted | Mean | Standard Deviation | Percent Change | Baseline up to Month 65 |
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| Secondary | Percent Change From Baseline in Spleen Volume by Imaging | Assessment of spleen volume was performed using serial imaging with MRI. Response was defined as ≥35% reduction in spleen volume (PR) or resolution of palpable splenomegaly (CR). Each cycle is 28 days long. | Safety Population: all participants who received ≥1 dose of avapritinib. Here, 'Number Analyzed' signifies those participants evaluable for this outcome measure at the specified timepoint. | Posted | Mean | Standard Deviation | Percent Change | Baseline up to Month 65 |
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| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Mean Change From Baseline in AdvSM-SAF Skin and Gastrointestinal Domain Scores at Month 10 | The AdvSM-SAF is a 10-item questionnaire that assesses symptoms and functional domains specific to AdvSM. Eight symptoms are scored on a scale of 0 to 10 for severity and 2 symptoms (vomiting and diarrhea) are scored for number of episodes. The skin domain is scored on a scale of 0-30, where 0 represents an absence of symptoms and 30 the most severe symptom experience. Similarly, the gastrointestinal domain is scored on a scale of 0-40, where 0 represents an absence of symptoms and 40 the most severe symptom experience. Skin and gastrointestinal domain scores were generated based on average scores for each 7-day period. An increase in score represents a worse symptoms outcome. Participants completed the AdvSM-SAF daily using an eDiary. | Safety Population: all participants who received ≥1 dose of avapritinib. Here, 'Number Analyzed' signifies those participants evaluable for this outcome measure at the specified timepoint. | Posted | Mean | Standard Deviation | Score on a Scale | Baseline, Month 10 |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Mean Change From Baseline in AdvSM-SAF Individual Symptom Scores (Severity) at Month 10 | The AdvSM-SAF is a 10-item questionnaire that assesses symptoms and functional domains specific to AdvSM. Individual symptom scores are generated based on average scores for each 7-day period. Eight symptoms are scored on a scale of 0 to 10 for severity. An increase in score represents a worse symptoms outcome. Participants completed the AdvSM-SAF daily using an eDiary. | Safety Population: all participants who received ≥1 dose of avapritinib. Here, 'Number Analyzed' signifies those participants evaluable for this outcome measure at the specified timepoint. | Posted | Mean | Standard Deviation | Score on a Scale | Baseline, Month 10 |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Mean Change From Baseline in AdvSM-SAF Individual Symptom Scores (Episodes) at Month 10 | The AdvSM-SAF is a 10-item questionnaire that assesses symptoms and functional domains specific to AdvSM. Individual symptom scores are generated based on average scores for each 7-day period. Two symptoms (vomiting and diarrhea) are scored for number of episodes. An increase in score represents a worse symptoms outcome. Participants completed the AdvSM-SAF daily using an eDiary. | Safety Population: all participants who received ≥1 dose of avapritinib. Here, 'Number Analyzed' signifies those participants evaluable for this outcome measure at the specified timepoint. | Posted | Mean | Standard Deviation | Number of Episodes | Baseline, Month 10 |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Patient's Global Impression of Symptom Severity (PGIS) | The PGIS is a single-item scale that assesses a participant's perception of disease symptoms at a point in time. Scores range from 0 to 4 points, with higher values representing worse symptom outcomes. It is widely used to evaluate a participant's overall sense of whether a treatment has been beneficial. Each cycle is 28 days long. | Safety Population: all participants who received ≥1 dose of avapritinib. Here, 'Number Analyzed' signifies those participants evaluable for this outcome measure at the specified timepoint. | Posted | Mean | Standard Deviation | Score on a Scale | Baseline, Cycle 1, Day 15 and Day 1 of Cycle 2, Cycle 3, Cycle 5, Cycle 7, Cycle 9, Cycle 11, Cycle 14, and Cycle 17 (28 days/cycle) |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30) | Quality of life was assessed using EORTC QLQ-C30, which is a 30-item questionnaire that includes 5 functional domains (physical, cognitive, role, emotional, and social) and a global health status scale. Twenty-eight questions are scored from 1 (not at all) to 4 (very much), while the other 2 are scored from 1 (very poor) to 7 (excellent). The calculated average is standardized using a linear transformation to a standardized scale of 0 to 100, with a decrease in score representing a decrease in quality of life. Each cycle is 28 days long. | Safety Population: all participants who received ≥1 dose of avapritinib. Here, 'Number Analyzed' signifies those participants evaluable for this outcome measure at the specified timepoint. | Posted | Mean | Standard Deviation | Score on a Scale | Baseline, Cycle 1, Day 15 and Day 1 of Cycle 2, Cycle 3, Cycle 5, Cycle 7, Cycle 9, Cycle 11, Cycle 14, and Cycle 17 (28 days/cycle) |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants Experiencing Treatment-emergent Adverse Events (TEAE) | A TEAE was defined as any adverse events that occurred between the first dose of a study drug through 30 days after the last dose of any study drug. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. | Safety Population: all participants who received ≥1 dose of avapritinib. | Posted | Count of Participants | Participants | Baseline through Month 65 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Maximum Plasma Concentration (Cmax) of a Single Dose of Avapritinib | Blood samples were collected at specified timepoints. Results reported as nanograms/milliliter (ng/mL). | Pharmacokinetic Population: all participants who received ≥1 dose of avapritinib and had had >3 post-dose concentrations collected. | Posted | Mean | Full Range | ng/mL | Day 1 of Cycle 1 (1 hour postdose) (28 days/cycle) |
|
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| Secondary | Correlation Between TSS and Serum Tryptase | The change in baseline for AdvSM-SAF TSS was correlated with the change in baseline for serum tryptase. Spearman correlation coefficients were performed with corresponding scatter plots on change from baseline. | Safety Population: all participants who received ≥1 dose of avapritinib. | Posted | Number | Spearman Coefficient | Baseline, Month 10 |
|
|
Day 1 through Month 65
All reported safety data based upon the Safety Population: all participants who received ≥1 dose of avapritinib regardless of the starting avapritinib dose (N=107). All participants were treated with a starting dose of 200 mg with the exception of 2 participants who were treated with a starting dose of 100 mg.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Avapritinib (100 mg) | Avapritinib was administered once daily as an immediate-release tablet, orally, in 28-day cycles. | 1 | 2 | 2 | 2 | 2 | 2 |
| EG001 | Avapritinib (200 mg) | Avapritinib was administered once daily as an immediate-release tablet, orally, in 28-day cycles. | 33 | 105 | 67 | 105 | 104 | 107 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Haemorrhagic diathesis | Blood and lymphatic system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Leukocytosis | Blood and lymphatic system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Mast cell activation syndrome | Blood and lymphatic system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Cardiac failure | Cardiac disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Acute myocardial infarction | Cardiac disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Cardiac failure congestive | Cardiac disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Coronary artery dissection | Cardiac disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Myocardial infarction | Cardiac disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Vertigo | Ear and labyrinth disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Ascites | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Intra-abdominal haemorrhage | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Small intestinal obstruction | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Duodenal ulcer haemorrhage | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Gastritis erosive | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Haematemesis | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Ileus paralytic | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Inguinal hernia | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Lower gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Lymphangiectasia intestinal | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Pancreatic fistula | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Pancreatitis acute | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Disease progression | General disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 27.0 | Systematic Assessment |
| |
| General physical health deterioration | General disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Oedema | General disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Acute cholecystitis necrotic | Hepatobiliary disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Budd-Chiari syndrome | Hepatobiliary disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Cholecystitis | Hepatobiliary disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA 27.0 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Appendicitis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| COVID-19 pneumonia | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Diverticulitis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Diverticulitis intestinal perforated | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Erysipelas | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Infection | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Peritonitis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Appendiceal abscess | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Clostridium difficile infection | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Cystitis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Device related infection | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Endocarditis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Escherichia sepsis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Fournier's gangrene | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Gastroenteritis astroviral | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Groin abscess | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Herpes zoster | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Intervertebral discitis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Parapharyngeal space infection | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Paraspinal abscess | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Pneumonia aspiration | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Septic endocarditis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Septic shock | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Spontaneous bacterial peritonitis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Stoma site cellulitis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Submandibular abscess | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Urinary tract infection bacterial | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Urosepsis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Procedural haemorrhage | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Subdural haematoma | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Upper limb fracture | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Cystitis radiation | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Femur fracture | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Post procedural haematuria | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Post procedural haemorrhage | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Post procedural hypotension | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Procedural nausea | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Radiation proctitis | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Rib fracture | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Spinal compression fracture | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Haemoglobin decreased | Investigations | MedDRA 27.0 | Systematic Assessment |
| |
| Hepatic enzyme increased | Investigations | MedDRA 27.0 | Systematic Assessment |
| |
| Neutrophil count decreased | Investigations | MedDRA 27.0 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Hypocalcaemia | Metabolism and nutrition disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Intervertebral disc protrusion | Metabolism and nutrition disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Colon cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 27.0 | Systematic Assessment |
| |
| Angiosarcoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 27.0 | Systematic Assessment |
| |
| Basal cell carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 27.0 | Systematic Assessment |
| |
| Benign neoplasm | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 27.0 | Systematic Assessment |
| |
| Bladder cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 27.0 | Systematic Assessment |
| |
| Lung adenocarcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 27.0 | Systematic Assessment |
| |
| Malignant melanoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 27.0 | Systematic Assessment |
| |
| Papillary thyroid cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 27.0 | Systematic Assessment |
| |
| Cognitive disorder | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Haemorrhage intracranial | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Transient ischaemic attack | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Dementia | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Encephalopathy | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Migraine | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Nervous system disorder | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Neuropathy peripheral | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Polyneuropathy | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Bipolar disorder | Psychiatric disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Confusional state | Psychiatric disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Mental status changes | Psychiatric disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Chronic kidney disease | Renal and urinary disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Renal failure | Renal and urinary disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Hypertonic bladder | Renal and urinary disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Obstructive nephropathy | Renal and urinary disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Bronchial haemorrhage | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Haemothorax | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Pulmonary mass | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Thoracic haemorrhage | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Skin lesion | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Angiodysplasia | Vascular disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Embolism | Vascular disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Lymphangiectasia | Vascular disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Shock haemorrhagic | Vascular disorders | MedDRA 27.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Leukopenia | Blood and lymphatic system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Periorbital oedema | Eye disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Eyelid oedema | Eye disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Lacrimation increased | Eye disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Ascites | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Face oedema | General disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Swelling face | General disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Oedema | General disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Hyperbilirubinaemia | Hepatobiliary disorders | MedDRA 27.0 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Herpes zoster | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Blood alkaline phosphatase increased | Investigations | MedDRA 27.0 | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA 27.0 | Systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA 27.0 | Systematic Assessment |
| |
| Weight increased | Investigations | MedDRA 27.0 | Systematic Assessment |
| |
| Neutrophil count decreased | Investigations | MedDRA 27.0 | Systematic Assessment |
| |
| Platelet count decreased | Investigations | MedDRA 27.0 | Systematic Assessment |
| |
| White blood cell count decreased | Investigations | MedDRA 27.0 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 27.0 | Systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA 27.0 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 27.0 | Systematic Assessment |
| |
| Blood lactate dehydrogenase increased | Investigations | MedDRA 27.0 | Systematic Assessment |
| |
| Hyperuricaemia | Metabolism and nutrition disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Hypophosphataemia | Metabolism and nutrition disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Hypocalcaemia | Metabolism and nutrition disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Folate deficiency | Metabolism and nutrition disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Hypomagnesaemia | Metabolism and nutrition disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Bone pain | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Cognitive disorder | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Dysgeusia | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Memory impairment | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Disturbance in attention | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Taste disorder | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Confusional state | Psychiatric disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Hair colour changes | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Night sweats | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Petechiae | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Flushing | Vascular disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Leukocytosis | Blood and lymphatic system disorders | MedDRA 27.0 | Systematic Assessment |
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| Cytopenia | Blood and lymphatic system disorders | MedDRA 27.0 | Systematic Assessment |
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| Lymphopenia | Blood and lymphatic system disorders | MedDRA 27.0 | Systematic Assessment |
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| Neutrophilia | Blood and lymphatic system disorders | MedDRA 27.0 | Systematic Assessment |
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| Cataract | Eye disorders | MedDRA 27.0 | Systematic Assessment |
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| Toothache | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
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| Gastric polyps | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
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| Rectal tenesmus | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
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| Clostridium difficile colitis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
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| Peritonitis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
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| Varicella zoster virus infection | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
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| Immunisation reaction | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
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| Hypoalbuminaemia | Metabolism and nutrition disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Skin papilloma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 27.0 | Systematic Assessment |
| |
| Adenocarcinoma gastric | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 27.0 | Systematic Assessment |
| |
| Aphasia | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
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| Polyneuropathy | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
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| Post herpetic neuralgia | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
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| Haematuria | Renal and urinary disorders | MedDRA 27.0 | Systematic Assessment |
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Compared to investigator-assessed response with its challenging interpretations of any differences in ORR by central adjudication, ORR by centrally adjudicated mIWG-MRT-ECNM criteria as assessed by the study steering committee is the most accurate and reliable evaluation of response to treatment in AdvSM.
After (a) sponsor publication of the comprehensive multi-center clinical trial results; (b) sponsor notification that the multi-center clinical trial submission is no longer planned; or (c) the 18-month anniversary of the completion or early termination of the multi-center clinical trial, the PI (institution/investigator) may publish or publicly present the study data in accordance with the provisions set forth by the sponsor.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Blueprint Medicines Medical Information | Blueprint Medicines | 1-888-258-7768 | medinfo@blueprintmedicines.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jun 29, 2020 | Dec 17, 2025 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D034721 | Mastocytosis, Systemic |
| D007946 | Leukemia, Mast-Cell |
| ID | Term |
|---|---|
| D008415 | Mastocytosis |
| D009372 | Neoplasms, Connective Tissue |
| D018204 | Neoplasms, Connective and Soft Tissue |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D000090362 | Mast Cell Activation Disorders |
| D007154 | Immune System Diseases |
| D007938 | Leukemia |
| D015470 | Leukemia, Myeloid, Acute |
| D007951 | Leukemia, Myeloid |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C000707147 | avapritinib |
Not provided
Not provided
Not provided
| Unknown or Not Reported |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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