Back to resultsRuxolitinib Combined With Dexamethasone for HLH
Primary Purpose
Hemophagocytic Lymphohistiocytosis
Status
Unknown status
Phase
Phase 1
Locations
China
Study Type
Interventional
Intervention
Ruxolitinib
Dexamethasone
Sponsored by
About this trial
This is an interventional treatment trial for Hemophagocytic Lymphohistiocytosis
Eligibility Criteria
Inclusion Criteria:
- Secondary and refractory HLH.
Exclusion Criteria:
- Family HLH.
Sites / Locations
- Yan YueRecruiting
- Yan YueRecruiting
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Ruxolitinib and Placebo
Placebo and Ruxolitinib
Arm Description
Ruxolitinib 2.5 mg twice daily by oral
Sugar pill 2.5 mg twice daily by oral
Outcomes
Primary Outcome Measures
Severity of disease
Serum ferritin>2000ng/ml, disease actvie; serum ferritin<2000ng/ml, disease control;
Secondary Outcome Measures
Axillary temperature
36<Axillary temperature<37.2, disease control; 37.2<Axillary temperature<38.2, disease partly control; Axillary temperature>38.2, disease active
Full Information
NCT ID
NCT03795909
First Posted
January 3, 2019
Last Updated
January 5, 2019
Sponsor
Capital Research Institute of Pediatrics
1. Study Identification
Unique Protocol Identification Number
NCT03795909
Brief Title
Ruxolitinib Combined With Dexamethasone for HLH
Official Title
Ruxolitinib Combined With Dexamethasone for Pediatric Refractory and Secondary Hemophagocytic Lymphohistiocytosis
Study Type
Interventional
2. Study Status
Record Verification Date
January 2019
Overall Recruitment Status
Unknown status
Study Start Date
March 1, 2017 (Actual)
Primary Completion Date
March 1, 2020 (Anticipated)
Study Completion Date
March 1, 2022 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Capital Research Institute of Pediatrics
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
A protocol named as "HLH-DR" for patients with refractory and secondary hemophagocytic lymphohistiocytosismay.
Detailed Description
A modified protocol about ruxolitinib combined with dexamethasone which includes oral ruxolitinib (2.5 mg twice daily for patients if the age<14 years and the weight <25kg，5 mg twice daily for patients if the age<14 years and the weight≥25kg, 10mg twice daily for patients if the age ≥14 years and<18 years）. Dexamethasone (10mg/m2.d) delayes for the first 2 weeks （form week 1 to 2），(5mg/m2.d) from week 3 to 4，(2.5mg/m2.d) from week 5 to 6），1.25mg/m2.d for week 7，and dropped off on week 8. Every 2 weeks, patients can be evaluated based the diagnosed index
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophagocytic Lymphohistiocytosis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Sequential Assignment
Masking
Participant
Allocation
Randomized
Enrollment
50 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Ruxolitinib and Placebo
Arm Type
Experimental
Arm Description
Ruxolitinib 2.5 mg twice daily by oral
Arm Title
Placebo and Ruxolitinib
Arm Type
Placebo Comparator
Arm Description
Sugar pill 2.5 mg twice daily by oral
Intervention Type
Drug
Intervention Name(s)
Ruxolitinib
Other Intervention Name(s)
Jakafi
Intervention Description
Ruxolitinib （2.5 mg twice daily for patients if the age<14 years and the weight <25kg，5 mg twice daily for patients if the age<14 years and the weight≥25kg, 10mg twice daily for patients if the age ≥14 years and<18 years）
Intervention Type
Drug
Intervention Name(s)
Dexamethasone
Other Intervention Name(s)
Hormone
Intervention Description
Dexamethasone (10mg/m2.d) delayes for the first 2 weeks（form week 1 to 2),(5mg/m2.d) from week 3 to 4，(2.5mg/m2.d) from week 5 to 6），1.25mg/m2.d for week 7，and dropped off on week 8.
Primary Outcome Measure Information:
Title
Severity of disease
Description
Serum ferritin>2000ng/ml, disease actvie; serum ferritin<2000ng/ml, disease control;
Time Frame
2 weeks
Secondary Outcome Measure Information:
Title
Axillary temperature
Description
36<Axillary temperature<37.2, disease control; 37.2<Axillary temperature<38.2, disease partly control; Axillary temperature>38.2, disease active
Time Frame
2 weeks
10. Eligibility
Sex
All
Gender Based
Yes
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Secondary and refractory HLH.
Exclusion Criteria:
Family HLH.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Yan Yue, MD
Phone
8618515238169
Ext
8618515238169
Email
adaile123@sina.com
First Name & Middle Initial & Last Name or Official Title & Degree
xiaodong d Shi, MD
Phone
8613911601076
Ext
8618515238169
Email
18611196921@163.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Yan Yue, MD
Organizational Affiliation
Capital Institute of Pediatrics, China
Official's Role
Principal Investigator
Facility Information:
Facility Name
Yan Yue
City
Beijing
State/Province
Chaoyang District
ZIP/Postal Code
100020
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Yan Yue, MD
Phone
8618515238169
Ext
8618515238169
Email
18611196921@163.com
First Name & Middle Initial & Last Name & Degree
Yan Yue, MD
Phone
8618515238169
Ext
8618515238169
Email
adaile123@sina.com
First Name & Middle Initial & Last Name & Degree
Jia Y Qin, MD
Facility Name
Yan Yue
City
Beijing
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
xiaodong Shi, MD
Phone
008613911601076
Ext
8618515238169
Email
adaile123@sina.com
First Name & Middle Initial & Last Name & Degree
Yan Yue, MD
Phone
8618515238169
Ext
8618515238169
Email
adaile123@sina.com
12. IPD Sharing Statement
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Ruxolitinib Combined With Dexamethasone for HLH
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