Osteopetrosis Clinical Trial
Official title:
Post-Marketing Surveillance Study of Actimmune (Interferon Gamma-1b) in Patients With Severe Malignant Osteopetrosis
The purpose of this study is to establish a registry of all children with severe, malignant osteopetrosis who are treated with Actimmune (IFN-g 1b or Interferon gamma-1b) to monitor the effects of IFN-g 1b on preventing progression of this disease and to follow the safety of patients receiving it on a long-term basis. In addition, evaluation of the possible effect of Actimmune therapy on the humoral response to normal childhood vaccinations in this same patient population will be examined.Interferon gamma is a substance that the body makes naturally.
It is made by white blood cells and appears to be involved in regulating the body's ability
to fight off infection. Actimmune is a synthetic form of Interferon gamma which is similar
to that normally made by white blood cells.
IFN-g 1b (Actimmune®) is currently approved by the United States Food and Drug
Administration (FDA) for the treatment of patients with chronic granulomatous disease (CGD)
to reduce the frequency and severity of serious infections. It is also approved in patients
with severe, malignant osteopetrosis to delay the time to disease progression. In research
trials, IFN-g 1b has been given to over 2,000 patients in diseases such as CGD,
osteopetrosis, atopic dermatitis, pulmonary fibrosis, atypical mycobacteria and various
cancers.
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Time Perspective: Retrospective
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