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Tyrosine Kinase Inhibitor
Avapritinib for Systemic Mastocytosis
Phase 2
Waitlist Available
Research Sponsored by Blueprint Medicines Corporation
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Patient must have a diagnosis of aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematologic neoplasm (SM-AHN) or mast cell leukemia (MCL) based on World Health Organization diagnostic criteria. Before enrollment, the Study Steering Committee must confirm the diagnosis of AdvSM (based on Central Pathology Laboratory assessment of bone marrow).
Patient must have Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0 to 3.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up approximately 4 years after the first subjected enrolled
Awards & highlights
Study Summary
This trial is testing a new drug, avapritinib, for patients with advanced mastocytosis, which is a rare disease where mast cells accumulate in the body.
Who is the study for?
This trial is for adults with advanced systemic mastocytosis, including aggressive forms and mast cell leukemia. Participants must have a serum tryptase level of at least 20 ng/mL and an ECOG performance status of 0-3. They should not have had recent cytoreductive therapy or certain other treatments, nor should they have severe kidney issues, seizure disorders, brain cancer, low platelet counts, a history of intracranial bleeding, abnormal heart rhythm or liver enzymes due to causes other than mastocytosis.Check my eligibility
What is being tested?
The PATHFINDER study is testing the effectiveness and safety of Avapritinib (BLU-285), which targets specific mutations in tyrosine kinase involved in systemic mastocytosis. It's an open-label Phase 2 trial where all participants receive the drug without being compared to another treatment or placebo.See study design
What are the potential side effects?
Potential side effects from Avapritinib may include nausea, vomiting, swelling around the eyes or on the skin (periorbital edema), fatigue, muscle pain (myalgia), cognitive changes like memory problems or confusion (cognitive effects), hair color changes and fluid retention.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have been diagnosed with a severe form of mastocytosis confirmed by a specialized committee.
Select...
I can care for myself but may not be able to do active work.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ approximately 4 years after the first subjected enrolled
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~approximately 4 years after the first subjected enrolled
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Objective response rate (ORR) based on modified International Working Group-Myeloproliferative Neoplasms Research and Treatment and European Competence Network on Mastocytosis (IWG-MRT-ECNM) response criteria
Secondary outcome measures
Area Under Curve (0 to Tau) for Avapritinib
Change in EORTC QLQ-C30
Change in PGIS
+13 moreSide effects data
From 2021 Phase 1 trial • 250 Patients • NCT02508532100%
Decreased appetite
67%
Cough
67%
Weight decreased
67%
Dizziness
67%
Blood bilirubin increased
67%
Dysgeusia
33%
Peripheral sensory neuropathy
33%
Hypoxia
33%
Memory impairment
33%
Constipation
33%
Mood altered
33%
Periorbital oedema
33%
Influenza like illness
33%
Insomnia
33%
Anemia
33%
Rash
33%
Dry skin
33%
Hypertension
33%
Neutropenia
33%
Lacrimation increased
33%
Dyspepsia
33%
Dry mouth
33%
Hypokalaemia
33%
Back pain
33%
Myalgia
33%
Hair colour changes
33%
Alopecia
33%
Face oedema
33%
Dermatitis acneiform
33%
Contusion
33%
Leukopenia
33%
Hypophosphataemia
33%
Anxiety
100%
80%
60%
40%
20%
0%
Study treatment Arm
Experimental: Part 1 Avapritinib (Formerly BLU-285) 600 mg QD
Experimental: Part 1 and Part 2 Avapritinib (Formerly BLU-285) 300 or 400 mg QD
Experimental: Part 1 Avapritinib (Formerly BLU-285) 30 mg QD
Experimental: Part 1 Avapritinib (Formerly BLU-285) 60 mg QD
Experimental: Part 1 Avapritinib (Formerly BLU-285) 90 mg QD
Experimental: Part 1 Avapritinib (Formerly BLU-285) 135 mg QD
Experimental: Part 1 Avapritinib (Formerly BLU-285) 200 mg QD
Trial Design
1Treatment groups
Experimental Treatment
Group I: AvapritinibExperimental Treatment1 Intervention
Avapritinib will be administered as an immediate release tablet, orally, continuously, in 28-day cycles
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Avapritinib
2015
Completed Phase 1
~340
Find a Location
Who is running the clinical trial?
Blueprint Medicines CorporationLead Sponsor
29 Previous Clinical Trials
6,116 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- My kidney function is significantly reduced.I have been diagnosed with a severe form of mastocytosis confirmed by a specialized committee.Your blood test must show a level of tryptase higher than 20 ng/mL.I can care for myself but may not be able to do active work.I haven't taken any cancer-reducing drugs or experimental treatments within the last 14 days, or specific drugs like cladribine or antibody therapies within the last 28 days.I have high eosinophil levels and a positive FIP1L1-PDGFRA test, or no prior imatinib treatment failure.I have a history of brain bleeding or conditions that increase its risk within the last year.I have epilepsy or need medication to prevent seizures.I haven't had cancer treatment in the last 3 years, except for certain skin cancers, localized prostate cancer, or completely removed in-situ carcinoma.My platelet count is below 50,000 or I need platelet transfusions.I have a brain tumor or cancer that has spread to my brain.My bilirubin levels are not high due to liver disease or Gilbert's disease.My liver enzymes are high, but it might be due to my condition.Your heart's QT interval, when adjusted for your heart rate, is longer than 480 milliseconds.I have been treated with avapritinib before.
Research Study Groups:
This trial has the following groups:- Group 1: Avapritinib
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Are there any associated risks to taking Avapritinib?
"Assessing the safety of Avapritinib, our team at Power awarded it a score of 2. This rating is based on evidence that suggests its safe to use, though no clinical data has been provided in support of efficacy yet."
Answered by AI
What is the rate of recruitment for this clinical experiment?
"Unfortunately, this research has ceased recruitment and the last update to its information was on March 25th 2022. If you are still in search of studies, there exist 272 clinical trials actively seeking participants with systemic mastocytosis plus a hematologic neoplasm and 7 recruiting for Avapritinib users."
Answered by AI
Has anything of this nature been tested before in a clinical setting?
"Presently, Blueprint Medicines Corporation is managing 7 Avapritinib studies in 37 cities and 18 countries. The first trial began in 2018 with 103 participants and ultimately achieved Phase 2 approval status. Since then, an additional 3 experiments have been concluded."
Answered by AI
In what locations is this study currently being conducted?
"Potential participants have 13 different sites to choose from, including Rush University Medical Center of Chicago, St. Michael's Hospital in Toronto and Dana Farber Cancer Institute in Boston among others."
Answered by AI
Are there any current opportunities to join this experiment?
"This medical experiment is not currently open for enrollment. Initially advertised on November 21st, 2018 and last updated March 25th 2022, alternate opportunities can be found in 272 trials recruiting patients with systemic mastocytosis associated hematologic neoplasm or 7 studies offering Avapritinib treatments."
Answered by AI
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