Ruxolitinib as a Salvage Therapy for Hemophagocytic Lymphohistiocytosis

Clinical Study of Different Doses of Ruxolitinib as a Salvage Therapy for Refractory/Relapsed Hemophagocytic Lymphohistiocytosis

This study aimed to investigate the efficacy and safety of different doses of ruxolitinib as a salvage therapy for refractory/relapsed hemophagocytic lymphohistiocytosis(HLH).

Study Overview

• Status: Unknown
• Conditions: Hemophagocytic Lymphohistiocytosis
• Intervention / Treatment: Drug: low dose ruxolitinib; Drug: high dose ruxolitinib

• Study Type: Interventional
• Enrollment (Anticipated): 80
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: jingshi wang; Phone Number: 86-010-63139862; Email: wangjingshi987@126.com

Study Locations

• China
  • Beijing
    • Beijing, Beijing, China, 100050
      • Recruiting
      • Beijing Friendship Hospital, Capital Medical University
      • Contact: Zhao Wang

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 75 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. meet hemophagocytic lymphohistiocytosis (HLH)-04 diagnostic criteria;
2. treated with HLH-94 no less than 2 weeks before enrollment and did not achieve at least PR; or relapsed patients after remission；
3. Life expectancy exceeds 1 month;
4. Age≥1 year old and ≤75 years old, gender is not limited；
5. Before the start of the study, total bilirubin ≤ 10 times the upper limit of normal; serum creatinine ≤ 1.5 times normal；
6. Serum human immunodeficiency virus(HIV) antigen or antibody negative；
7. Hepatitis C virus (HCV) antibody is negative, or HCV antibody is positive, but HCV RNA is negative；
8. Both hepatitis B virus (HBV) surface antigen and HBV core antibody were negative. If any of the above is positive, peripheral blood hepatitis B virus DNA titer should be detected, and less than 1×103 copies/ml can enter the group；
9. Informed consent.

Exclusion Criteria:

1. Pregnancy or lactating Women；
2. Allergic to ruxolitinib；
3. Active bleeding of the internal organs；
4. uncontrollable infection；
5. Serious mental illness;
6. Non-melanoma skin cancer history;
7. Patients unable to comply during the trial and/or follow-up phase;
8. Participate in other clinical research at the same time.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Low dose	Drug: low dose ruxolitinib; The dose for adult patients(Age>=14 years) is generally 10 mg twice daily. For children(Age<14 years ,weight >=25kg),the dose was generally 5mg twice daily. For children(Age<14 years, weight <25kg), the dose was generally 2.5mg twice daily.
Experimental: High dose	Drug: high dose ruxolitinib; The dose for adult patients(Age>=14 years) is generally 20mg twice daily. For children(Age<14 years ,weight >=25kg),the dose was generally 10mg twice daily. For children(Age<14 years, weight <25kg), the dose was generally 5mg twice daily

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Response rate	complete response (CR) and partial response (PR) rates	1 years
Progression Free Survival	from date of inclusion to date of progression, relapse, or death from any cause Overall Survival	1 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall Survival	from the date of inclusion to date of death, irrespective of cause Adverse Events	1 years
Adverse events	Adverse events including myelosuppression, infection, bleeding and so on.	1 year

Collaborators and Investigators

• Sponsor: Beijing Friendship Hospital

Study record dates

Study Major Dates

• Study Start (Actual): July 1, 2019
• Primary Completion (Anticipated): July 1, 2020
• Study Completion (Anticipated): July 1, 2020

Study Registration Dates

• First Submitted: October 6, 2019
• First Submitted That Met QC Criteria: October 6, 2019
• First Posted (Actual): October 9, 2019

Study Record Updates

• Last Update Posted (Actual): October 29, 2019
• Last Update Submitted That Met QC Criteria: October 27, 2019
• Last Verified: October 1, 2019

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Lymphatic Diseases; Histiocytosis, Non-Langerhans-Cell; Histiocytosis; Lymphohistiocytosis, Hemophagocytic
• Other Study ID Numbers: Ruxolitinib-HLH

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No