A study to investigate the safety and efficacy of a new drug, NI-0501, in children with a disease that is called Primary Haemophagocytic Lymphohistiocytosis

Recruitment & Eligibility

Status: ot Recruiting

Sex: Not specified

Target Recruitment: 38

Inclusion Criteria

1. Primary HLH patients of both genders, up to and including 18 years at diagnosis of HLH, or at an age appropriate to be treated in the investigator's practice. The diagnosis must be made on the following criteria (as per HLH-2004 protocol):
a. A molecular diagnosis or familial history consistent with primary HLH OR
b. 5 out of 8 criteria below are fulfilled:
- Fever
- Splenomegaly
- Cytopenias affecting 2 of 3 lineages in the peripheral blood (hemoglobin <90g/L; platelets <100x10^9/L; neutrophils <1x10^9/L)
- Hypertriglyceridemia (fasting triglycerides >3mmol/L or >265mg/dL) and/or hypofibrinogenemia (=1.5g/L)
- Hemophagocytosis in bone marrow, spleen or lymph nodes, with no evidence of malignancy
- Low or absent natural killer (NK)-cell activity
- Ferritin =500µg/L
- Soluble CD25 (sCD25; i.e. soluble IL-2 receptor) =2400U/mL
2. Presence of active disease as assessed by treating physician
3. Patients having already received HLH conventional therapy must fulfill one of the following criteria as assessed by the treating physician:
- Having not responded
- Having not achieved a satisfactory response
- Having not maintained a satisfactory response
- Showing intolerance of conventional HLH treatment
At the time of enrollment, eligible patients might still be receiving treatment (induction or maintenance) or might have discontinued it.
4. Informed consent signed by the patient (if =18 years old) or by their legally authorized representative(s) with the assent of patients who are legally capable of providing it.
5. Having received guidance on contraception for both male and female patients sexually active and having reached puberty:
Females of child-bearing potential, having a negative pregnancy test at screening and unless true abstinence is in line with the preferred and usual lifestyle of the patient, must agree to use adequate method(s) of birth control from screening until 6 months after receiving last dose of the study drug. Males with partner(s) of child-bearing potential must agree to take appropriate precautions to avoid fathering a child from screening until 6 months after receiving last dose of the study drug.
Are the trial subjects under 18? yes
Number of subjects for this age range: 38
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 3
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Diagnosis of secondary HLH consequent to a proven rheumatic or neoplastic disease.
2. Body weight < 3 kg.
3. Patients treated with:
- any T-cell depleting agents (such as anti-thymocyte globulin [ATG], anti-CD52) during the previous 2 weeks prior to screening
- any other biologic drug within 5 times their defined half-life period, expect for rituximab in case of documented B-cell EBV infection
4. Active mycobacteria, Histoplasma Capsulatum, Shigella, Campylobacter, Leishmania or Salmonella infections.
5. Evidence of history of tuberculosis or latent tuberculosis.
6. Positive serology for HIV antibodies, hepatitis B surface antigen or hepatitis C antibodies.
7. Presence of malignancy.
8. Patient who have another concomitant disease or malformation severely affecting the cardiovascular, pulmonary, liver or renal function.
9. History of hypersensitivity or allergy to any components of the study regimen.
10. Receipt of a live or attenuated live (including BCG) vaccine within the last 12 weeks before screening.
11. Pregnant or lactating female patients.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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