Open-label Early Phase 2 Study With a Single Arm of Interferon Gamma-1b Treatment of Osteopetrosis

21549-01

This study evaluates the effects of ACTIMMUNE (IFN-γ1b) in children and adults with intermediate osteoporosis. All participants will receive treatment with ACTIMMUNE for 12 months. The investigators hypothesize that ACTIMMUNE will be tolerated by participants for the full 12 months and result in decreased disease severity.

2016-02-22

2019-03-11

2019-04-11

Completed

Early phase I

5

Inclusion Criteria: * Diagnosis of osteopetrosis; and * Anemia (Hemoglobin \<12 g/dL) not related to iron deficiency, or * Neutropenia (Neutrophil count \<1000 neutrophils/ul unsupported with cytokines), or * Thrombocytopenia (Platelet count \<50,000 cells x 109/L), or * History of impaired bone healing, or * ≥ 1 serious infection over prior year defined as requiring hospitalization and/or IV antibiotics, and * Age \> 1 year; and * Ability to travel to a study center for every 3-6 month study visits; and * Patient or parent/legal guardian is able and willing to provide informed consent. For patients 7 to 17 years of age, assent must also be provided. Exclusion Criteria: * 12 months or fewer following HCT; * Pregnancy or breastfeeding; * Known or suspected allergy to interferon gamma-1b or related products; * Participation in simultaneous therapeutic study that involves an investigational study drug or agent within 4 weeks of study enrollment; * ALT greater than 3 fold higher than normal; or * Any other social or medical condition that the Investigator believes would pose a significant hazard to the subject if the investigational therapy were initiated or be detrimental to the study.

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 5, 'type': 'ACTUAL'}}

2023-10-11