Ruxolitinib as a Salvage Therapy for Hemophagocytic Lymphohistiocytosis

Capital Medical University

Status and phase: Unknown; Phase 3

Conditions: Hemophagocytic Lymphohistiocytosis

Treatments: Drug: high dose ruxolitinib; Drug: low dose ruxolitinib

Study type: Interventional

Funder types: Other

Identifiers

NCT04120090

Ruxolitinib-HLH

Details and patient eligibility

About

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

Enrollment: 80 estimated patients

Sex: All

Ages: 1 to 75 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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80 participants in 2 patient groups

Low dose

Active Comparator group

Treatment:

Drug: low dose ruxolitinib

High dose

Experimental group

Treatment:

Drug: high dose ruxolitinib