Ruxolitinib as a Salvage Therapy for Hemophagocytic Lymphohistiocytosis

NCT04120090 | Unknown Phase 3 DMC

• 1 other identifier: Ruxolitinib-HLH
• Study Type: interventional
• Target: 80
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Hemophagocytic Lymphohistiocytosis

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (2)

• Overall Survival

  1 years

• Adverse events

  1 year

Study Arms (2)

Low dose

ACTIVE COMPARATOR

Drug: low dose ruxolitinib

High dose

EXPERIMENTAL

Drug: high dose ruxolitinib

Interventions

low dose ruxolitinib DRUG

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.

Low dose

high dose ruxolitinib DRUG

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

High dose

Eligibility Criteria

• Age: 1 Year - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• meet hemophagocytic lymphohistiocytosis (HLH)-04 diagnostic criteria;
• treated with HLH-94 no less than 2 weeks before enrollment and did not achieve at least PR; or relapsed patients after remission；
• Life expectancy exceeds 1 month;
• Age≥1 year old and ≤75 years old, gender is not limited；
• Before the start of the study, total bilirubin ≤ 10 times the upper limit of normal; serum creatinine ≤ 1.5 times normal；
• Serum human immunodeficiency virus(HIV) antigen or antibody negative；
• Hepatitis C virus (HCV) antibody is negative, or HCV antibody is positive, but HCV RNA is negative；
• 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；
• Informed consent.

You may not qualify if:

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

Sponsors & Collaborators

• Beijing Friendship Hospital: lead

Study Sites (1)

Beijing Friendship Hospital, Capital Medical University

Beijing, Beijing Municipality, 100050, China

RECRUITING

MeSH Terms

Conditions

Lymphohistiocytosis, Hemophagocytic

Interventions

ruxolitinib

Condition Hierarchy (Ancestors)

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

Central Study Contacts

jingshi wang

CONTACT

86-010-63139862; wangjingshi987@126.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Clinical Professor

Study Record Dates

• First Submitted: October 6, 2019
• First Posted: October 9, 2019
• Study Start: July 1, 2019
• Primary Completion: July 1, 2020
• Study Completion: July 1, 2020
• Last Updated: October 29, 2019

Record last verified: 2019-10

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)