A Study to Investigate the Safety and Efficacy of an Anti-IFNÎ³ mAb in Children Affected by Primary Haemophagocytic Lymphohistiocytosis
Phase: Phase 2/Phase 3
DFCI Protocol ID: 14-018
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.
Dana-Farber Cancer Institute, Children's Hospital Boston
Barbara Degar, MD,
Dana-Farber Cancer Institute
Dana-Farber Cancer Institute:
Childrens Hospital Pediatric Clinical Translation Investigation Program CTIP, email@example.com
- Gender: male and female
- Age: up to and including 18 years at diagnosis of Haemophagocytic Lymphohistiocytosis
- Primary HLH patients
- Patient (if â‰¥ 18 years old), or patient's legal representative(s) must have signed
- 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
- Presence of malignancy.
- Concomitant disease or malformation severely affecting the cardiovascular, pulmonary,
liver or renal function