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Ruxolitinib + Fostamatinib for Chronic Graft-versus-Host Disease

Phase 1

Waitlist Available

Led By Chenyu Lin, MD

Research Sponsored by Stefanie Sarantopoulos, MD, PhD.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Have undergone allogeneic hematopoietic cell transplantation (HCT) from any donor source (matched unrelated donor, sibling, haploidentical) using bone marrow, peripheral blood, or cord blood stem cells

Patients with clinically diagnosed cGvHD staging of mild to severe according to NIH Consensus Criteria prior to Cycle 1 Day 1 and with confirmed steroid refractoriness or steroid dependence irrespective of the concomitant use of a calcineurin inhibitor

Must not have

History of progressive multifocal leuko-encephalopathy (PML)

Evidence of active and clinically significant viral disease including HIV, CMV, HHV-6, HBV, HCV, or BK virus

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 year

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a combination of two medications, fostamatinib and ruxolitinib, for the treatment of chronic GvHD. The goal is to find the safest and most effective

Who is the study for?

This trial is for individuals with chronic Graft-versus-Host Disease (cGVHD) who haven't responded well to steroids. Participants should have specific conditions like Bronchiolitis Obliterans Syndrome and be able to receive both Ruxolitinib and Fostamatinib.

What is being tested?

The study tests the safety and effectiveness of combining two drugs, Ruxolitinib and Fostamatinib, for cGVHD patients not responding to steroids. It starts by finding a safe dose before expanding to more patients who will help determine the best dose for future studies.

What are the potential side effects?

Possible side effects from Ruxolitinib may include infections, low blood counts, dizziness, or headaches. Fostamatinib might cause high blood pressure, diarrhea, nausea, or liver enzyme elevations. The combination's side effects will be closely monitored.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have received a stem cell transplant from a donor.

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I have chronic GvHD that's not responding well to steroids, regardless of other treatments.

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I am 18 years old or older.

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My blood tests show enough neutrophils, platelets, and hemoglobin.

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I can take care of myself and am up and about more than half of my waking hours.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have had progressive multifocal leukoencephalopathy in the past.

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I do not have active significant viral infections like HIV or hepatitis.

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I do not have serious heart problems like recent heart attacks or severe heart failure.

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My blood pressure is not higher than 160/100 mmHg.

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My kidney function is low, but I am not on dialysis.

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My cancer returned after a stem cell transplant.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 year

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~1 year

This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Minimum safe and biologically effective dose

Secondary study objectives

1-year cGvHD-free survival

Duration of Response

Overall response rate

+1 more

Awards & Highlights

No Placebo-Only Group

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

Trial Design

6Treatment groups

Experimental Treatment

Group I: Dose escalation phase: Dose level 3Experimental Treatment2 Interventions

Fostamatinib at dose level 3 (dose of 100mg BID) in combination with standard of care ruxolitinib 10mg BID

Group II: Dose escalation phase: Dose level 2Experimental Treatment2 Interventions

Fostamatinib at dose level 2 (dose of 150mg QAM) in combination with standard of care ruxolitinib 10mg BID

Group III: Dose escalation phase: Dose level 1Experimental Treatment2 Interventions

Fostamatinib at dose level 1 (dose of 100mg QAM) in combination with standard of care ruxolitinib 10mg BID

Group IV: Dose escalation phase: Dose level 0Experimental Treatment2 Interventions

Fostamatinib at dose level 0 (dose of 50mg QAM) in combination with standard of care ruxolitinib 10mg BID

Group V: Candidate Dose #2Experimental Treatment2 Interventions

In the safety expansion cohort, subjects will be randomized to one of two candidate doses of fostamatinib (identified from the dose escalation phase) in combination with ruxolitinib 10mg BID.

Group VI: Candidate Dose #1Experimental Treatment2 Interventions

In the safety expansion cohort, subjects will be randomized to one of two candidate doses of fostamatinib (identified from the dose escalation phase) in combination with ruxolitinib 10mg BID.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Ruxolitinib

2018

Completed Phase 3

~1170

Fostamatinib

2021

Completed Phase 3

~1520