A Pilot Study of Ruxolitinib in Secondary Hemophagocytic Syndrome

NCT02400463 | Completed Phase 2 Results DMC

• 2 other identifiers: UMCC 2014.112HUM00092921
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

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.

Conditions

Hemophagocytic Syndrome (HPS)

Outcome Measures

Primary Outcomes (1)

• Overall Survival at 2 Months

  Number of Patients Alive at 2 Months after the first administration of ruxolitinib.

  2 Months

Secondary Outcomes (4)

• Percentage of Patients With a Response to Treatment With Ruxolitinib

  2 Months

• Duration of Response

  Up to 3 years (Due to funding and other constraints, participant follow-up was discontinued in 2020. Thus, not all participants were followed for a full three years.)

• Progression Free Survival Time

  Up to 3 years (Due to funding and other constraints, participant follow-up was discontinued in 2020. Thus, not all participants were followed for a full three years.)

• Regimen Related Toxicities

  Up to 30 days after last treatment administration

Study Arms (1)

Ruxolitinib

EXPERIMENTAL

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.

Drug: Ruxolitinib

Interventions

Ruxolitinib DRUG

Ruxolitinib

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients, or their legally authorized representative, must voluntarily provide written IRB-approved informed consent.
• Males and females, 18 years of age or older at the time of enrollment.
• Patients must meet the diagnostic criteria for HPS (at least 5 of the following): fever, splenomegaly, cytopenia involving ≥2 cell lines (Hemoglobin \<9 g/dL; platelets \<100,000/μL; absolute neutrophil count \<1000/μL), hypertriglyceridemia or hypofibrinogenemia, tissue demonstration of hemophagocytosis, low or absent NK (Natural Killer) cell activity, serum ferritin ≥3000 ug/L, soluble IL-2 receptor (CD25) \>2400 U/mL.
• In the investigator's opinion, the patient has the ability to participate fully in the study, and comply with all its requirements.

You may not qualify if:

• CNS (Central Nervous System) involvement
• Malabsorption
• Known secondary HPS (Hemophagocytic Syndrome) that is otherwise treatable (e.g. non-Hodgkin's lymphoma).
• Pregnant or lactating female: all females of child-bearing potential must have a negative serum pregnancy test within 7 days of treatment; lactating females must discontinue breast feeding.
• Estimated creatinine clearance \<15mL/min
• Has received any prior systemic therapy, excluding corticosteroids, within 7 days (or 5 half-lives) of treatment.
• No active malignancy at the time of enrollment, except nonmelanoma skin cancers or carcinoma in situ. Patients with a prior history of malignancy are eligible if their malignancy has been definitely treated or is in remission and does not require ongoing adjuvant or cancer-directed therapies.
• Active hepatitis B or hepatitis C or known HIV infection
• Known (and biopsy-confirmed) liver cirrhosis; or, a reported history of liver cirrhosis with a Model for End-stage Liver Disease (MELD) score \>20.

Sponsors & Collaborators

• University of Michigan Rogel Cancer Center: lead

Study Sites (1)

University of Michigan Comprehensive Cancer Center

Ann Arbor, Michigan, 48109, United States

Location

Related Publications (1)

• Ahmed A, Merrill SA, Alsawah F, Bockenstedt P, Campagnaro E, Devata S, Gitlin SD, Kaminski M, Cusick A, Phillips T, Sood S, Talpaz M, Quiery A, Boonstra PS, Wilcox RA. Ruxolitinib in adult patients with secondary haemophagocytic lymphohistiocytosis: an open-label, single-centre, pilot trial. Lancet Haematol. 2019 Dec;6(12):e630-e637. doi: 10.1016/S2352-3026(19)30156-5. Epub 2019 Sep 16.

  PMID: 31537486 DERIVED

Related Links

• The Lancet Haematology, VOLUME 6, ISSUE 12, E630-E637, DECEMBER 01, 2019

MeSH Terms

Conditions

Lymphohistiocytosis, Hemophagocytic

Interventions

ruxolitinib

Condition Hierarchy (Ancestors)

Histiocytosis, Non-Langerhans-Cell; Histiocytosis; Lymphatic Diseases; Hemic and Lymphatic Diseases

Results Point of Contact

• Title: Ryan Wilcox
• Organization: University of Michigan Rogel Cancer Center

rywilcox@umich.edu

734-615-1482

Study Officials

• Ryan Wilcox, M.D.

  Int Med-Hematology/Oncology - Faculty and Staff

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 22, 2015
• First Posted: March 27, 2015
• Study Start: February 5, 2016
• Primary Completion: October 10, 2019
• Study Completion: January 7, 2020
• Last Updated: January 25, 2021
• Results First Posted: November 18, 2020

Record last verified: 2021-01

Locations

US (1)