search
Back to results

(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

Primary Purpose

Indolent Systemic Mastocytosis

Status
Active
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Avapritinib
Placebo
Sponsored by
Blueprint Medicines Corporation
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Indolent Systemic Mastocytosis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Key Inclusion Criteria:

  • 1. Patient must have SM, confirmed by Central Pathology Review of BM biopsy, and central review of B- and C-findings by WHO diagnostic criteria.
  • 2. Patient must have moderate-to-severe symptoms based on minimum mean total symptom score (TSS) of the ISM Symptom Assessment Form (ISM-SAF) over the 14-day eligibility screening period.
  • 3. Patient must have failed to achieve adequate symptom control for 1 or more Baseline symptoms.
  • 4. For patients receiving corticosteroids, the dose must be ≤ 20 mg/d prednisone or equivalent, and the dose must be stable for ≥ 14 days.
  • 5. Patient must have an Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0 to 2.

Key Exclusion Criteria:

  • 1. Patient has been diagnosed with any of the following WHO SM subclassifications: cutaneous mastocytosis only, smoldering SM, SM with associated hematologic neoplasm, aggressive SM, mast cell leukemia, or mast cell sarcoma.
  • 2. Patient must not have received prior treatment with avapritinib.
  • 3. Patient must not have had any cytoreductive therapy including but not limited to masitinib and midostaurin, or investigational agent for < 14 days or 5 half-lives of the drug (whichever is longer), and for cladribine, interferon alpha, pegylated interferon, or antibody therapy < 28 days or 5 half-lives of the drug (whichever is longer), before beginning the 14-day ISM-SAF eligibility TSS assessment.
  • 4. Patient must not have received radiotherapy or psoralen and ultraviolet A (PUVA) therapy < 14 days before beginning the 14-day ISM-SAF eligibility TSS assessment.
  • 5. Patient must not have received any hematopoietic growth factor the preceding 14 days before beginning the 14-day ISM-SAF eligibility TSS assessment.
  • 6. Patient must not have a QT interval corrected using Fridericia's formula (QTcF) of > 480 msec.

Sites / Locations

  • University of Alabama at Birmingham
  • Mayo Clinic Hospital
  • Stanford Cancer Institute
  • Mayo Clinic Florida
  • H. Lee Moffitt Cancer Center
  • Winship Cancer Institute, Emory University
  • Rush University Medical Center
  • University of Kansas Hospital
  • Brigham & Women's Hospital
  • Dana Farber Cancer Institute
  • Michigan Medicine, University of Michigan
  • Mayo Clinic
  • Washington University School of Medicine
  • Herbert Irving Comprehensive Cancer Center
  • Duke University Health System (DUHS)
  • University Hospitals Cleveland Medical Center
  • University of Texas, MD Anderson Cancer Center
  • Huntsman Cancer Institute
  • Virginia Commonwealth University Medical Center
  • University Hospital Antwerp
  • Tom Baker Cancer Centre
  • University of Alberta Hospital
  • St. Michael's Hospital
  • Odense Universitetshospital, ORCA/Allergicentret, Hudafdeling I og Allergicenter
  • Hôpital de la Timone, Service de dermatologie
  • Hôpital Pitié-Salpêtrière, Service de Dermatologie
  • CHU Toulouse Larrey, CEREMAST, Service de Dermatologie et Allergologie cutanée
  • Uniklinik RWTH Aachen
  • Charité Universitätsmedizin Berlin
  • University Clinic Hamburg Eppendorf, University Cancer Center Hamburg (UCCH)
  • Universitätsklinikum Schleswig-Holstein, Hämatologie/Onkologie
  • Universitätsklinik Mainz, Universitäts-Hautklinik, Clinical Research Center
  • Universitätsmedizin Mannheim, III. Medizinische Klinik
  • Klinikum rechts der Isar, Technische Universität München
  • A.O.U di Bologna - IRCCS, Istituto di Ematologia Lorenzo e Ariosto Seragnoli, Ematologia
  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Poloclinico, UOC Ematologia
  • A.O. OO.RR. S.Giovanni di Dio e Ruggi d'Aragona, University of Salerno
  • Azienda Ospedaliera Universitaria Integrata di Verona
  • University Medical Center Groningen (UMCG)
  • Erasmus Medical Center
  • Oslo Universitetssykehus, Rikshospitalet, Department of Hematology
  • Hospital Universitari Vall d'Hebron
  • lnstituto de Estudios de Mastocitosis de Castilla la Mancha, Hospital Virgen del Valle - Complejo Hospitalario de Toledo
  • Karolinska University Hospital, Hematologimottagningen R51
  • Akademiska sjukhuset, Hematologmottagningen/101A
  • University Hospital Basel
  • NHS Greater Glasgow and Clyde, Beatson West of Scotland Cancer Centre
  • Guy's and St Thomas' NHS Foundation Trust - Guy's Hospital
  • Clatterbridge Cancer Centre NHS Foundation Trust

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm 6

Arm 7

Arm Type

Experimental

Experimental

Experimental

Placebo Comparator

Experimental

Placebo Comparator

Experimental

Arm Label

(Part 1) Avapritinib Dose 1 + BSC

(Part 1) Avapritinib Dose 2 + BSC

(Part 1) Avapritinib Dose 3 + BSC

(Part 1) Placebo + BSC

(Part 2) Avapritinib RP2D + BSC

(Part 2) Placebo + BSC

(Part 3) Avapritinib RP2D + BSC

Arm Description

Avapritinib will be administered orally in continuous 28-day cycles

Avapritinib will be administered orally in continuous 28-day cycles

Avapritinib will be administered orally in continuous 28-day cycles

Placebo will be administered orally in continuous 28-day cycles

Avapritinib will be administered orally in continuous 28-day cycles

Placebo will be administered orally in continuous 28-day cycles

Avapritinib will be administered orally in continuous 28-day cycles

Outcomes

Primary Outcome Measures

Part 1: Recommended Phase 2 dose (RP2D) in patients with ISM
Part 2: Mean change in ISM Symptom Assessment Form (ISM-SAF) total symptom score (TSS) as compared to placebo
0 - 110 points (higher value represents worse symptom outcomes)
Part 3: Number of Participants with Adverse Events

Secondary Outcome Measures

Part 2: Proportion of patients with a ≥50% reduction in serum tryptase
Part 2: Proportion of patients with a ≥50% reduction in peripheral blood V-kit Hardy-Zuckerman 4 feline sarcoma viral oncogene homolog aspartate 816 valine (KIT D816V) allele fraction or undetectable for patients with detectable mutation at Baseline
Part 2: Proportion of patients with ≥50% reduction in ISM-SAF TSS
Part 2: Proportion of patients with ≥30% reduction in ISM-SAF TSS
Part 2: Proportion of patients with a ≥50% reduction in bone marrow mast cells or no aggregates for patients with aggregates at Baseline
Parts 1, 2, and 3: Change in serum tryptase
Parts 1, 2, and 3: Change in KIT D816V allele burden in blood
Parts 1, 2, and 3: Change in bone marrow mast cells
Parts 1, 2, and 3: Change in best supportive care (BSC) concomitant medication usage
Parts 1, 2, and 3: Change from Baseline in ISM-SAF Score
Parts 1, 2, and 3: Change in Mastocytosis Quality of Life Questionnaire (MC-QoL)
Parts 1, 2, and 3: Change in Patient's Global Impression of Symptom Severity (PGIS)
Parts 1, 2, and 3: Change in 12-item Short Form Health Survey (SF-12)
0 - 100 points (higher value represents better symptom outcomes)
Parts 1, 2, and 3: Change in Patients' Global Impression of Change (PGIC)
1 - 7 (higher value represents worse symptom outcomes)
Parts 1, 2, and 3: Change in EuroQuol 5 Dimensions 5 Levels (EQ 5D-5L)
0 - 100 (higher value represents better symptom outcomes)
Parts 1, 2, and 3: Safety of avapritinib as assessed by number of adverse events
CTCAE version 5.0

Full Information

First Posted
October 30, 2018
Last Updated
June 14, 2023
Sponsor
Blueprint Medicines Corporation
search

1. Study Identification

Unique Protocol Identification Number
NCT03731260
Brief Title
(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
Official Title
A 3-Part, Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Evaluate Safety and Efficacy of Avapritinib (BLU-285), a Selective KIT Mutation-Targeted Tyrosine Kinase Inhibitor, in Indolent and Smoldering Systemic Mastocytosis With Symptoms Inadequately Controlled With Standard Therapy
Study Type
Interventional

2. Study Status

Record Verification Date
June 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
April 16, 2019 (Actual)
Primary Completion Date
June 23, 2027 (Anticipated)
Study Completion Date
January 31, 2028 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Blueprint Medicines Corporation

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
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 symptoms are not adequately controlled by BSC. The study will be conducted in 3 parts. All patients will receive treatment with avapritinib during Part 3 including those rolling over from the placebo group.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Indolent Systemic Mastocytosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Model Description
In Part 1 of the study, patients will be randomly assigned to 1 of 3 doses of avapritinib or to placebo + BSC. Once the recommended phase 2 dose (RP2D) of avapritinib is identified in Part 1, patients in Part 2 will be randomly assigned to receive avapritinib at the RP2D + BSC or matching placebo + BSC. In Part 3, patients who have completed treatment in Part 1 or Part 2 of the study (including those initially randomized to placebo) may participate in a long-term open-label extension, receiving avapritinib at the RP2D + BSC.
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
251 (Actual)

8. Arms, Groups, and Interventions

Arm Title
(Part 1) Avapritinib Dose 1 + BSC
Arm Type
Experimental
Arm Description
Avapritinib will be administered orally in continuous 28-day cycles
Arm Title
(Part 1) Avapritinib Dose 2 + BSC
Arm Type
Experimental
Arm Description
Avapritinib will be administered orally in continuous 28-day cycles
Arm Title
(Part 1) Avapritinib Dose 3 + BSC
Arm Type
Experimental
Arm Description
Avapritinib will be administered orally in continuous 28-day cycles
Arm Title
(Part 1) Placebo + BSC
Arm Type
Placebo Comparator
Arm Description
Placebo will be administered orally in continuous 28-day cycles
Arm Title
(Part 2) Avapritinib RP2D + BSC
Arm Type
Experimental
Arm Description
Avapritinib will be administered orally in continuous 28-day cycles
Arm Title
(Part 2) Placebo + BSC
Arm Type
Placebo Comparator
Arm Description
Placebo will be administered orally in continuous 28-day cycles
Arm Title
(Part 3) Avapritinib RP2D + BSC
Arm Type
Experimental
Arm Description
Avapritinib will be administered orally in continuous 28-day cycles
Intervention Type
Drug
Intervention Name(s)
Avapritinib
Other Intervention Name(s)
BLU-285
Intervention Description
Avapritinib tablet
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo tablet
Primary Outcome Measure Information:
Title
Part 1: Recommended Phase 2 dose (RP2D) in patients with ISM
Time Frame
9 months
Title
Part 2: Mean change in ISM Symptom Assessment Form (ISM-SAF) total symptom score (TSS) as compared to placebo
Description
0 - 110 points (higher value represents worse symptom outcomes)
Time Frame
6 months
Title
Part 3: Number of Participants with Adverse Events
Time Frame
Up to 5 years
Secondary Outcome Measure Information:
Title
Part 2: Proportion of patients with a ≥50% reduction in serum tryptase
Time Frame
6 months
Title
Part 2: Proportion of patients with a ≥50% reduction in peripheral blood V-kit Hardy-Zuckerman 4 feline sarcoma viral oncogene homolog aspartate 816 valine (KIT D816V) allele fraction or undetectable for patients with detectable mutation at Baseline
Time Frame
6 months
Title
Part 2: Proportion of patients with ≥50% reduction in ISM-SAF TSS
Time Frame
6 months
Title
Part 2: Proportion of patients with ≥30% reduction in ISM-SAF TSS
Time Frame
6 months
Title
Part 2: Proportion of patients with a ≥50% reduction in bone marrow mast cells or no aggregates for patients with aggregates at Baseline
Time Frame
6 months
Title
Parts 1, 2, and 3: Change in serum tryptase
Time Frame
Up to 5 years
Title
Parts 1, 2, and 3: Change in KIT D816V allele burden in blood
Time Frame
Up to 5 years
Title
Parts 1, 2, and 3: Change in bone marrow mast cells
Time Frame
Up to 5 years
Title
Parts 1, 2, and 3: Change in best supportive care (BSC) concomitant medication usage
Time Frame
Up to 5 years
Title
Parts 1, 2, and 3: Change from Baseline in ISM-SAF Score
Time Frame
Up to 5 years
Title
Parts 1, 2, and 3: Change in Mastocytosis Quality of Life Questionnaire (MC-QoL)
Time Frame
Up to 5 years
Title
Parts 1, 2, and 3: Change in Patient's Global Impression of Symptom Severity (PGIS)
Time Frame
Up to 5 years
Title
Parts 1, 2, and 3: Change in 12-item Short Form Health Survey (SF-12)
Description
0 - 100 points (higher value represents better symptom outcomes)
Time Frame
Up to 5 years
Title
Parts 1, 2, and 3: Change in Patients' Global Impression of Change (PGIC)
Description
1 - 7 (higher value represents worse symptom outcomes)
Time Frame
Up to 5 years
Title
Parts 1, 2, and 3: Change in EuroQuol 5 Dimensions 5 Levels (EQ 5D-5L)
Description
0 - 100 (higher value represents better symptom outcomes)
Time Frame
Up to 5 years
Title
Parts 1, 2, and 3: Safety of avapritinib as assessed by number of adverse events
Description
CTCAE version 5.0
Time Frame
Up to 5 years

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria: 1. Patient must have SM, confirmed by Central Pathology Review of BM biopsy, and central review of B- and C-findings by WHO diagnostic criteria. 2. Patient must have moderate-to-severe symptoms based on minimum mean total symptom score (TSS) of the ISM Symptom Assessment Form (ISM-SAF) over the 14-day eligibility screening period. 3. Patient must have failed to achieve adequate symptom control for 1 or more Baseline symptoms. 4. For patients receiving corticosteroids, the dose must be ≤ 20 mg/d prednisone or equivalent, and the dose must be stable for ≥ 14 days. 5. Patient must have an Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0 to 2. Key Exclusion Criteria: 1. Patient has been diagnosed with any of the following WHO SM subclassifications: cutaneous mastocytosis only, smoldering SM, SM with associated hematologic neoplasm, aggressive SM, mast cell leukemia, or mast cell sarcoma. 2. Patient must not have received prior treatment with avapritinib. 3. Patient must not have had any cytoreductive therapy including but not limited to masitinib and midostaurin, or investigational agent for < 14 days or 5 half-lives of the drug (whichever is longer), and for cladribine, interferon alpha, pegylated interferon, or antibody therapy < 28 days or 5 half-lives of the drug (whichever is longer), before beginning the 14-day ISM-SAF eligibility TSS assessment. 4. Patient must not have received radiotherapy or psoralen and ultraviolet A (PUVA) therapy < 14 days before beginning the 14-day ISM-SAF eligibility TSS assessment. 5. Patient must not have received any hematopoietic growth factor the preceding 14 days before beginning the 14-day ISM-SAF eligibility TSS assessment. 6. Patient must not have a QT interval corrected using Fridericia's formula (QTcF) of > 480 msec.
Facility Information:
Facility Name
University of Alabama at Birmingham
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35294
Country
United States
Facility Name
Mayo Clinic Hospital
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85054
Country
United States
Facility Name
Stanford Cancer Institute
City
Stanford
State/Province
California
ZIP/Postal Code
94305
Country
United States
Facility Name
Mayo Clinic Florida
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32224
Country
United States
Facility Name
H. Lee Moffitt Cancer Center
City
Tampa
State/Province
Florida
ZIP/Postal Code
33612
Country
United States
Facility Name
Winship Cancer Institute, Emory University
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Rush University Medical Center
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
University of Kansas Hospital
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
Brigham & Women's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Dana Farber Cancer Institute
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02215
Country
United States
Facility Name
Michigan Medicine, University of Michigan
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48106
Country
United States
Facility Name
Mayo Clinic
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Facility Name
Washington University School of Medicine
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Herbert Irving Comprehensive Cancer Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
Duke University Health System (DUHS)
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
University Hospitals Cleveland Medical Center
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44106
Country
United States
Facility Name
University of Texas, MD Anderson Cancer Center
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Huntsman Cancer Institute
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84112
Country
United States
Facility Name
Virginia Commonwealth University Medical Center
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23298
Country
United States
Facility Name
University Hospital Antwerp
City
Edegem
ZIP/Postal Code
2650
Country
Belgium
Facility Name
Tom Baker Cancer Centre
City
Calgary
State/Province
Alberta
ZIP/Postal Code
T2N 4N2
Country
Canada
Facility Name
University of Alberta Hospital
City
Edmonton
State/Province
Alberta
ZIP/Postal Code
T6G 2B7
Country
Canada
Facility Name
St. Michael's Hospital
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5B 1W8
Country
Canada
Facility Name
Odense Universitetshospital, ORCA/Allergicentret, Hudafdeling I og Allergicenter
City
Odense
ZIP/Postal Code
DK-5000
Country
Denmark
Facility Name
Hôpital de la Timone, Service de dermatologie
City
Marseille
ZIP/Postal Code
13385
Country
France
Facility Name
Hôpital Pitié-Salpêtrière, Service de Dermatologie
City
Paris
ZIP/Postal Code
75013
Country
France
Facility Name
CHU Toulouse Larrey, CEREMAST, Service de Dermatologie et Allergologie cutanée
City
Toulouse
ZIP/Postal Code
31059
Country
France
Facility Name
Uniklinik RWTH Aachen
City
Aachen
ZIP/Postal Code
52074
Country
Germany
Facility Name
Charité Universitätsmedizin Berlin
City
Berlin
ZIP/Postal Code
10117
Country
Germany
Facility Name
University Clinic Hamburg Eppendorf, University Cancer Center Hamburg (UCCH)
City
Hamburg
ZIP/Postal Code
20246
Country
Germany
Facility Name
Universitätsklinikum Schleswig-Holstein, Hämatologie/Onkologie
City
Lübeck
ZIP/Postal Code
23538
Country
Germany
Facility Name
Universitätsklinik Mainz, Universitäts-Hautklinik, Clinical Research Center
City
Mainz
ZIP/Postal Code
55131
Country
Germany
Facility Name
Universitätsmedizin Mannheim, III. Medizinische Klinik
City
Mannheim
ZIP/Postal Code
68167
Country
Germany
Facility Name
Klinikum rechts der Isar, Technische Universität München
City
Munich
ZIP/Postal Code
80802
Country
Germany
Facility Name
A.O.U di Bologna - IRCCS, Istituto di Ematologia Lorenzo e Ariosto Seragnoli, Ematologia
City
Bologna
ZIP/Postal Code
40138
Country
Italy
Facility Name
Fondazione IRCCS Ca' Granda Ospedale Maggiore Poloclinico, UOC Ematologia
City
Milan
ZIP/Postal Code
20122
Country
Italy
Facility Name
A.O. OO.RR. S.Giovanni di Dio e Ruggi d'Aragona, University of Salerno
City
Salerno
ZIP/Postal Code
84131
Country
Italy
Facility Name
Azienda Ospedaliera Universitaria Integrata di Verona
City
Verona
ZIP/Postal Code
37126
Country
Italy
Facility Name
University Medical Center Groningen (UMCG)
City
Groningen
ZIP/Postal Code
9713 GZ
Country
Netherlands
Facility Name
Erasmus Medical Center
City
Rotterdam
ZIP/Postal Code
3015 GD
Country
Netherlands
Facility Name
Oslo Universitetssykehus, Rikshospitalet, Department of Hematology
City
Oslo
ZIP/Postal Code
0372
Country
Norway
Facility Name
Hospital Universitari Vall d'Hebron
City
Barcelona
ZIP/Postal Code
08035
Country
Spain
Facility Name
lnstituto de Estudios de Mastocitosis de Castilla la Mancha, Hospital Virgen del Valle - Complejo Hospitalario de Toledo
City
Toledo
ZIP/Postal Code
45071
Country
Spain
Facility Name
Karolinska University Hospital, Hematologimottagningen R51
City
Stockholm
ZIP/Postal Code
141 86
Country
Sweden
Facility Name
Akademiska sjukhuset, Hematologmottagningen/101A
City
Uppsala
ZIP/Postal Code
751 85
Country
Sweden
Facility Name
University Hospital Basel
City
Basel
ZIP/Postal Code
4031
Country
Switzerland
Facility Name
NHS Greater Glasgow and Clyde, Beatson West of Scotland Cancer Centre
City
Glasgow
ZIP/Postal Code
G12 OXL
Country
United Kingdom
Facility Name
Guy's and St Thomas' NHS Foundation Trust - Guy's Hospital
City
London
ZIP/Postal Code
SE1 9RT
Country
United Kingdom
Facility Name
Clatterbridge Cancer Centre NHS Foundation Trust
City
Wirral
ZIP/Postal Code
CH63 4JY
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
34663404
Citation
Padilla B, Shields AL, Taylor F, Li X, Mcdonald J, Green T, Boral AL, Lin HM, Akin C, Siebenhaar F, Mar B. Psychometric evaluation of the Indolent Systemic Mastocytosis Symptom Assessment Form (ISM-SAF) in a phase 2 clinical study. Orphanet J Rare Dis. 2021 Oct 18;16(1):434. doi: 10.1186/s13023-021-02037-3.
Results Reference
derived
Links:
URL
http://PioneerTrial.com
Description
More information about the study

Learn more about this trial

(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

We'll reach out to this number within 24 hrs