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

Status: Recruiting
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.

Conducting Institutions:

Dana-Farber Cancer Institute, Children's Hospital Boston

Overall PI:

Barbara Degar, MD, Dana-Farber Cancer Institute

Site-responsible Investigators:


Dana-Farber Cancer Institute: Childrens Hospital Pediatric Clinical Translation Investigation Program CTIP, ctip@partners.org

Eligibility Criteria

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

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