EURO-HIT-HLH European cooperative pilot study for testing Hybrid ImmunoTherapy for Hemophagocytic LymphoHistiocytosis

Conditions: Familial hemophagoytic lymphohistiocytosis
MedDRA version: 14.1Level: SOCClassification code 10005329Term: Blood and lymphatic system disordersSystem Organ Class: 10005329 - Blood and lymphatic system disorders
Therapeutic area: Diseases [C] - Immune System Diseases [C20]

Registration Number: EUCTR2011-002052-14-IT

Lead Sponsor: AZIENDA OSPEDALIERA MEYER

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 50

Inclusion Criteria

Diagnosis: The diagnosis of hemophagocytic lymphohistiocytosis must be met according to the HLH-2004 criteria. Age: Patients <18 years of age. Disease Status: The patient must have active disease at the time of enrollment. If the diagnosis is made by genetic criteria, the patient must also meet at least 4 out of 8 clinical criteria. Clinical and/or laboratory observations establishing the diagnosis of active HLH (except NK function) must occur no more than 1 week prior to study entry.
Are the trial subjects under 18? yes
Number of subjects for this age range: 50
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

- Recent treatment, within 3 months, with another therapeutic regimen for HLH. -Known active malignancy -Known rheumatologic diagnosis which may be the underlying cause of HLH, such as systemic onset juvenile idiopathic arthritis or systemic lupus erythematosus - Pregnancy (as determined by serum or urine test) or active breast feeding - Failure to provide signed informed consent

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To achieve an early disease control thus allowing the patient to undergo HSCT when appropriate, in the shortest time. Primary endopoint: survival at 8 weeks;Secondary Objective: - Response to therapy at 2, 4 and 8 weeks - Reactivation during and after initial treatment - proportion of patients who may undergo HSCT;Primary end point(s): Primary endopoint: survival at 8 weeks;Timepoint(s) of evaluation of this end point: Eight weeks from treatment start

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): - Response to therapy at 2, 4 and 8 weeks - Reactivation during and after initial treatment - proportion of patients who may undergo HSCT;Timepoint(s) of evaluation of this end point: All within day 100 from treatment start.