Clinical Trial: A Pilot Study of Ruxolitinib in Secondary Hemophagocytic Syndrome
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: Pilot Study of Ruxolitinib in Secondary Hemophagocytic Syndrome
Brief Summary: This is a pilot study to determine the efficacy of Ruxolitinib in secondary hemophagocytic syndrome. The primary objective is to assess the efficacy of ruxolitinib 15 mg PO twice daily in patients with HPS. The primary endpoint is overall survival at two months.
Detailed Summary:
Hemophagocytic Syndrome (HPS) is a disorder characterized by pathological activation of the immune system resulting in a systemic disorder characterized by excessive cytokine production and macrophage activation, culminating in cytopenias and evidence of hemophagocytosis on tissue specimens. The disorder can be sporadic or familial due to one of several mutations and is primarily seen in the pediatric population, with a reported incidence of 1 case per 3000 admissions1. The actual incidence in adults is unknown and can be rarely sporadic, or secondary to viral infections, malignancy, or autoimmune disease.
HPS is a universally fatal disease if untreated. In adults, the median survival has been reported to be less than 2 months if diagnosis and treatment is delayed2. Adult patients are treated with pediatric protocols with early institution of etoposide and steroids and consolidation with allogeneic stem cell transplant in appropriately selected patients if a familial form is identified 3. Other treatment strategies have been attempted, including rituximab4, infliximab5, entaracept6, tocilizumab7, and alemtuzumab8. These anecdotal reports highlight the therapeutic potential of cytokine-targeted therapies in this disorder.
This is a pilot study to determine the efficacy of Ruxolitinib in secondary hemophagocytic syndrome. The primary objective is to assess the efficacy of ruxolitinib 15 mg PO twice daily in patients with HPS. The primary endpoint is overall survival at two months.
Patients will receive Ruxolitinib at 15 mg twice daily orally either on an empty stomach or with food for 3 weeks (21 days) in a 3 week (21 day) cycle. Ruxolitinib will be administered in continuous 21-day cycles.
In the absence of treatment delays o
Sponsor: University of Michigan Cancer Center
Current Primary Outcome: Number of Patients Alive at 2 Months [ Time Frame: 2 Months ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Percentage of Patients with a Response to Treatment with Ruxolitinib [ Time Frame: 2 Years ]Complete response is defined as complete normalization of all quantifiable symptoms and laboratory abnormalities. A partial response is defined as at least a 25% improvement in two or more quantifiable symptoms/laboratory markers 2 weeks following the initiation of treatment.
- Duration of Response [ Time Frame: 2 Years ]Duration will be calculated from the date of the determination of partial response or better until the date of progression, death, or additional non-protocol therapy. For patients lost to follow-up, duration will be censored at the time of last clinical assessment.
- Progression Free Survival Time [ Time Frame: 2 Years ]Progressive Disease is defined as at least a 50% worsening in two or more quantifiable laboratory markers.
Original Secondary Outcome: Same as current
Information By: University of Michigan Cancer Center
Dates:
Date Received: January 22, 2015
Date Started: September 2015
Date Completion: September 2018
Last Updated: March 2, 2017
Last Verified: March 2017
