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Kinase Inhibitor
Dose Escalation for Systemic Mastocytosis
Phase 1
Recruiting
Led By Andrew Kuykendall, MD
Research Sponsored by H. Lee Moffitt Cancer Center and Research Institute
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 24 months
Awards & highlights
Study Summary
"This trial aims to study a new treatment for a challenging disease called systemic mastocytosis with an associated hematologic neoplasm. Current treatments only target one aspect of the disease, leading to progression
Who is the study for?
This trial is for patients with a condition called SM-AHN, which includes systemic mastocytosis and an associated blood cancer. Participants should have this specific diagnosis to be eligible.Check my eligibility
What is being tested?
The study tests the combination of two drugs, Avapritinib and Decitabine, to see if they can better control both systemic mastocytosis and the related blood cancer compared to current treatments.See study design
What are the potential side effects?
Possible side effects may include fatigue, nausea, bleeding issues due to low platelet counts, infections from low white blood cell counts, liver problems, and potential heart issues.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 24 months
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 24 months
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Recommended phase 2 dose (RP2D)
Secondary outcome measures
Incidence and frequency of AEs and changes in vital signs, ECGs, and laboratory tests
Overall Responsive Rate
Systemic mastocytosis overall response rate (ORR)
Trial Design
2Treatment groups
Experimental Treatment
Group I: Dose ExpansionExperimental Treatment3 Interventions
Participants with baseline platelet count (cycle 1, day 1) ≥ 75 x 10^9/L will begin the combination of Avapritinib at the dose determined by the dose-finding portion of the study and decitabine or Decitabine/Cedazuridine will be initiated during the first cycle. Participants with a baseline platelet count between 25 x 10^9/L and 74 x 10^9/L will receive decitabine or Decitabine/Cedazuridine (choice of investigator) as a single-agent for at least the first two cycles. Starting with cycle 3 and continuing with each subsequent cycle, patients will be eligible to receive Avapritinib in combination with decitabine or Decitabine/Cedazuridine if the platelet count on day 1 of the cycle is ≥ 75 x 10^9. If the platelet count is < 75 x 10^9/L on day 1 of the cycle, patient will receive single-agent Decitabine or Decitabine/Cedazuridine. In the absence of clear disease progression, patients will be treated for at least 6 cycles before being judged to have not responded.
Group II: Dose EscalationExperimental Treatment3 Interventions
Participants will be enrolled in cohorts of size 1-3. Participants will take a combination of Avapritinib and Decitabine. Decitabine will be administered in one of the following forms: I) Decitabine: starting IV dose of 20 mg/m2 on days 1-5 of a 28-day treatment cycle. II) Decitabine/Cedazuridine: starting dose of 35mg/100 mg oral tablet on days 1-5 of a 28-day treatment cycle. The starting dose of Avapritinib for the first cohort of patients will begin at dose level 1 with dose modifications to be made according to a Bayesian design.
Avapritnib Dose levels Level -1: 50mg Level 1: 100mg Level 2: 150mg Level 3: 200mg
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Avapritinib
2015
Completed Phase 1
~350
Decitabine
2004
Completed Phase 3
~1680
Find a Location
Who is running the clinical trial?
H. Lee Moffitt Cancer Center and Research InstituteLead Sponsor
543 Previous Clinical Trials
135,448 Total Patients Enrolled
Blueprint Medicines CorporationIndustry Sponsor
29 Previous Clinical Trials
6,175 Total Patients Enrolled
Andrew Kuykendall, MDPrincipal InvestigatorMoffitt Cancer Center
1 Previous Clinical Trials
25 Total Patients Enrolled
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
To what extent does the process of increasing medication dosage pose risk to patients?
"Since this trial is at the Phase 1 stage, there is limited data available to support both safety and efficacy. Therefore, our team at Power rates the safety of Dose Escalation as a 1 on a scale from 1 to 3."
Answered by AI
How large is the sample size for this particular investigation?
"Indeed, the details on clinicaltrials.gov indicate that this particular research study is actively seeking eligible participants. The trial was initially listed on March 1st, 2024 and its latest update was made on March 18th, 2024. The study aims to enroll a total of 34 participants distributed among seven designated sites."
Answered by AI
In the United States, are there multiple locations conducting this research study?
"Recruitment for this study is ongoing at various esteemed institutions including Mayo Clinic - Arizona in Phoenix, Stanford University Medical Center in Palo Alto, and Memorial Sloan Kettering Cancer Center in New york among 7 additional sites."
Answered by AI
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