Ruxolitinib as a Salvage Therapy for Hemophagocytic Lymphohistiocytosis

• Sponsor: Beijing Friendship Hospital (Other)
• Overall Status: Unknown status
• CT.gov ID: NCT04120090
• Collaborator: (none)
• Enrollment: 80
• Location: 1
• Arms: 2
• Anticipated Duration (Months): 12
• Patients Per Site Per Month: 6.7

Study Details

Study Description

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).

Condition or Disease	Intervention/Treatment	Phase
Hemophagocytic Lymphohistiocytosis	Drug: low dose ruxolitinib; Drug: high dose ruxolitinib	Phase 3

Study Design

• Study Type: Interventional
• Anticipated Enrollment: 80 participants
• Allocation: Non-Randomized
• Intervention Model: Parallel Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Clinical Study of Different Doses of Ruxolitinib as a Salvage Therapy for Refractory/Relapsed Hemophagocytic Lymphohistiocytosis
• Actual Study Start Date: Jul 1, 2019
• Anticipated Primary Completion Date: Jul 1, 2020
• Anticipated Study Completion Date: Jul 1, 2020

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures

1. Response rate [1 years]

   complete response (CR) and partial response (PR) rates

2. Progression Free Survival [1 years]

   from date of inclusion to date of progression, relapse, or death from any cause Overall Survival

Secondary Outcome Measures

1. Overall Survival [1 years]

   from the date of inclusion to date of death, irrespective of cause Adverse Events

2. Adverse events [1 year]

   Adverse events including myelosuppression, infection, bleeding and so on.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 1 Year to 75 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Beijing Friendship Hospital, Capital Medical University	Beijing	Beijing	China	100050

Sponsors and Collaborators

• Beijing Friendship Hospital

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Zhao Wang, Clinical Professor, Beijing Friendship Hospital
• ClinicalTrials.gov Identifier: NCT04120090
• Other Study ID Numbers: Ruxolitinib-HLH
• First Posted: Oct 9, 2019
• Last Update Posted: Oct 29, 2019
• Last Verified: Oct 1, 2019

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Lymphohistiocytosis, Hemophagocytic

Study Results

No Results Posted as of Oct 29, 2019