A Pilot Study of Ruxolitinib in Secondary Hemophagocytic Syndrome
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02400463|
Recruitment Status : Recruiting
First Posted : March 27, 2015
Last Update Posted : April 11, 2019
|Condition or disease||Intervention/treatment||Phase|
|Hemophagocytic Syndrome (HPS)||Drug: Ruxolitinib||Phase 2|
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 admissions. 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 delayed. 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. Other treatment strategies have been attempted, including rituximab, infliximab, entaracept, tocilizumab, and alemtuzumab. 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 4 weeks (28 days) in a 4 week (28 day) cycle. Ruxolitinib will be administered in continuous 28-day cycles.
In the absence of treatment delays or cessation due to adverse events, treatment may continue indefinitely or until one of the following criteria applies:
- Disease progression.
- Intercurrent illness that prevents further administration of treatment.
- The investigator considers it, for safety reasons, to be in the best interest of the patient.
- Unacceptable adverse events such as any toxicity or other issue that causes a delay of study drug administration by more than 4 weeks.
- General or specific changes in the patient's condition render the patient unacceptable for further treatment in the judgment of the investigator.
- Patient decision to withdraw from treatment (partial consent) or from the study (full consent.
Patients will be followed for toxicity for 30 days after treatment has been discontinued or until death, whichever occurs first.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Pilot Study of Ruxolitinib in Secondary Hemophagocytic Syndrome|
|Study Start Date :||September 2015|
|Estimated Primary Completion Date :||November 2019|
|Estimated Study Completion Date :||November 2020|
Ruxolitinib 15 mg by mouth twice daily.
For patients unable to ingest tablets, ruxolitinib suspended in water may be administered through a nasogastric (NG) or percutaneous endoscopy gastrostomy (PEG) tube.
- Number of Patients Alive at 2 Months [ Time Frame: 2 Months ]
- Percentage of Patients with a Response to Treatment with Ruxolitinib [ Time Frame: 2 Months ]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.
- Duration of Response [ Time Frame: Up to 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 Months ]Progressive Disease is defined as at least a 50% worsening in two or more quantifiable laboratory markers.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02400463
|Contact: Ryan Wilcox, M.D.||email@example.com|
|United States, Michigan|
|University of Michigan Comprehensive Cancer Center||Recruiting|
|Ann Arbor, Michigan, United States, 48109|
|Contact: Ryan Wilcox, M.D. 734-615-1482 firstname.lastname@example.org|
|Principal Investigator: Ryan Wilcox, M.D.|
|Principal Investigator:||Ryan Wilcox, M.D.||Int Med-Hematology/Oncology - Faculty and Staff|