Fostamatinib as a Single Agent or in Combination With Ruxolitinib for Treatment of Patients With Myelofibrosis With Severe Thrombocytopenia

Part of paid clinical trials in St Louis, Missouri.

Sponsor
Washington University School of Medicine
Study ID
NCT04543279
Phase
PHASE2
Status
Terminated

Conditions

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Fostamatinib — DRUG
    Fostamatinib will be supplied by Rigel Pharmaceuticals.
  • Ruxolitinib — DRUG
    Ruxolitinib is commercially available.

Study Details

Fostamatinib may improve thrombocytopenia in myelofibrosis patients with severe thrombocytopenia (platelet \<50,000/microL) and allow them to initiate treatment with a JAK2 inhibitor, ruxolitinib. Additionally, fostamatinib monotherapy may also improve myelofibrosis related symptoms and splenomegaly.

Key Dates

Start date
May 3, 2021
Status verified
Dec 2023
Primary completion
Jul 1, 2022
Completion
Jul 30, 2022

Study Design

Enrollment
3 participants (actual)
Allocation
NON_RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT

Arms

  • Experimental: Part A: Fostamatinib
    The starting dose of fostamatinib is 100 mg twice daily (BID). After the first cycle, if no major dose related safety issue is observed and the platelet count is less than 50K/microL, then the fostamatinib dose will be increased to 150 mg BID for the next 2 cycles; otherwise the dose may be continued at 100 mg BID.
  • Experimental: Part B: Fostamatinib + Ruxolitinib
    After 3 cycles of fostamatinib monotherapy, all patients with a sustained platelet count ≥ 50K/microL, will continue on the current fostamatinib dose plus ruxolitinib at the recommended dose per standard prescribing guidelines for an additional 9 cycles. Patients who do not reach platelet count of at least 50K/microL but who achieve clinical benefit per the treating provider may continue on single agent fostamatinib for up to 12 total treatment cycles. If these patients achieve a sustained platelet count of ≥ 50K/microL at any point prior to Cycle 10 Day 1, then they may be eligible to enroll in Part B of the study and continue treatment with fostamatinib and ruxolitinib for the remainder of the study.

Primary Outcome Measure

Number of Participants With a Platelet Response (Part A) [ Time Frame: Week 12 ]

Locations (1)

FacilityCityStateZIPSite coordinators
Washington University School of MedicineSt LouisMissouri63110-

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By research site
Washington University School of Medicine· St Louis, MO

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