A Study to Investigate the Safety and Efficacy of an Anti-IFNγ mAb in Children Affected by Primary Haemophagocytic Lymphohistiocytosis



Status:Active, not recruiting
Healthy:No
Age Range:Any - 18
Updated:2/28/2019
Start Date:January 2013

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A Phase 2/3, Open-label, Single Arm, Multicentre Study to Assess Safety, Tolerability, Pharmacokinetics and Efficacy of Intravenous Multiple Administrations of NI-0501, an Anti-interferon Gamma (Anti-IFNγ) Monoclonal Antibody, in Paediatric Patients With Primary Haemophagocytic Lymphohistiocytosis

The purpose of this study is to assess the safety, tolerability and efficacy of a new drug
aimed at controlling disease activity in patients diagnosed with primary haemophagocytic
lymphohistiocytosis. The new drug can be administered as the first-line therapy, to patients
not previously treated with the current standard of care, or can be given to patients who
have either failed or were unable to tolerate the current standard of care. Administration
will be on top of a glucocorticosteroid, which is usually part of the current recommended
treatment.


Inclusion Criteria:

- Gender: male and female

- Age: up to and including 18 years at diagnosis of Haemophagocytic Lymphohistiocytosis

- Primary HLH patients

- Patient (if ≥ 18 years old), or patient's legal representative(s) must have signed
informed consent

Exclusion Criteria:

- Diagnosis of secondary Haemophagocytic Lymphohistiocytosis consequent to a proven
rheumatic or neoplastic disease.

- Body weight < 3 kg.

- Patients treated with biologics within a specific timeframe

- Active Mycobacteria, Histoplasma Capsulatum, Shigella, Salmonella, Campylobacter and
Leishmania infections.

- Presence of malignancy.

- Concomitant disease or malformation severely affecting the cardiovascular, pulmonary,
liver or renal function
We found this trial at
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sites
Salt Lake City, Utah 84143
Principal Investigator: Mark N Fluchel, MD
Phone: 801-662-4710
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Chapel Hill, North Carolina 27599
(919) 962-2211
Principal Investigator: Kimberly Kasow, MD
Phone: 919-962-8733
University of North Carolina at Chapel Hill Carolina’s vibrant people and programs attest to the...
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1405 Clifton Road NE
Atlanta, Georgia 30322
404-785-6000
Principal Investigator: Shanmuganathan Chandrakasan, MD
Phone: 404-727-8877
Children's Healthcare of Atlanta Whether treating a toddler in an emergency or supporting a teen...
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13123 E 16th Ave
Aurora, Colorado 80045
(720) 777-1234
Principal Investigator: Timothy Garrington, MD
Phone: 720-777-6740
Children's Hospital Colorado At Children's Hospital Colorado, we see more, treat more and heal more...
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Boston, Massachusetts 02115
Principal Investigator: Barbara A Degar, MD
Phone: 617-632-5186
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Boston, MA
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Cincinnati, Ohio 45229
Principal Investigator: Michael Jordan, MD
Phone: 513-636-7287
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Cincinnati, OH
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Houston, Texas 77030
Principal Investigator: Carl Allen, MD, PhD
Phone: 832-824-4312
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Houston, TX
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1 Albert-Schweitzer-Campus
Münster, 48149
Principal Investigator: Claudia Rössig, Prof. Dr. Med.
Phone: 0049 2518347742
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Münster,
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Wilmington, Delaware 19803
Principal Investigator: Jonathan Powell, MD
Phone: 302-651-5505
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Wilmington, DE
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