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
- Conditions
- Haemophagocytic LymphohistiocytosisMedDRA version: 18.0 Level: SOC Classification code 10010331 Term: Congenital, familial and genetic disorders System Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Immune System Diseases [C20]
- Registration Number
- EUCTR2012-003632-23-DE
- Lead Sponsor
- ovImmune SA
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 10
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 and approved by the SSC:
- 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: 10
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 1
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
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
- anti-CD20 as part of EBV infection treatment within the previous week
prior to screening
- any other biologic drug within 5 times their defined half-life period (a
list of some of the most commonly used biologic half-lives will be
included in the Study Specific Risk Management Plan).
4. Acute isolated or multiple organ failure(s) (heart, lung or kidney)
requiring aggressive therapy such as high dose of inotropic drugs,
circulatory assistance, hemofiltration or haemodialysis, artificial
ventilation.
5. Active mycobacteria, Shigella, Campylobacter, Leishmania or
Salmonella infections.
6. Evidence of history of tuberculosis or latent tuberculosis.
7. Positive serology for HIV antibodies, hepatitis B surface antigen or
hepatitis C antibodies.
8. History of malignancy.
9. Patient who have another concomitant disease or malformation
severely affecting the cardiovascular, pulmonary, liver or renal function.
10. History of hypersensitivity or allergy to any components of the study
regimen.
11. Receipt of a live or attenuated live (including BCG) vaccine within the last 12 weeks before screening.
12. 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