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Kinase Inhibitor

Avapritinib + Decitabine 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

No Placebo-Only Group

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.

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.

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

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 24 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Recommended phase 2 dose (RP2D)

Secondary study objectives

Incidence and frequency of AEs and changes in vital signs, ECGs, and laboratory tests

Overall Responsive Rate

Systemic mastocytosis overall response rate (ORR)

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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

2021

Completed Phase 4

~410

Decitabine

2011

Completed Phase 3

~2370