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.ALT greater than 3 fold higher than normal or.You should also change the site with every injection. Look for places like the thighs, back of the upper arms, or abdomen (stomach area). Participation in simultaneous therapeutic study that involves an investigational study drug or agent within 4 weeks of study enrollment When injecting ACTIMMUNE (Interferon gamma-1b), choose a site that’s padded by some fat below the skin.Known or suspected allergy to interferon gamma-1b or related products.Patient or parent/legal guardian is able and willing to provide informed consent.įor patients 7 to 17 years of age, assent must also be provided. Ability to travel to a study center for every 3-6 month study visits and.Change in immunologic and hematologic function, bone mineral density and osteoclast function, physical function and quality of life.Specifically, i) the ability to enroll patients, and ii) continued treatment throughout the 1-year observational period. The feasibility and tolerability of interferon gamma-1b treatment for 1 year in patients with intermediate osteopetrosis.Therefore, the investigators will conduct an early phase 2, multi-center, open-label, 12-month clinical trial of ACTIMMUNE (IFN-γ1b) treatment of patients with intermediate osteopetrosis to determine the following: Two previous studies of IFN-γ1b in a small group of individuals with osteopetrosis found a decrease in trabecular bone area, an increase in marrow space, a decrease in the number of severe infections requiring antibiotic therapy, and an increase in superoxide generation by granulocyte-macrophage colonies. Interferon gamma (IFN-γ) is a naturally occurring cytokine that has been shown to have anti-microbial and anti-viral immunomodulatory effects, and is a potent stimulator of superoxide anion production which in turn promotes the formation and activation of osteoclasts. Thus additional treatments for osteopetrosis are needed both for individuals who are not candidates for HCT and to prolong the time until HCT is needed. Therefore, this treatment is only indicated in select individuals with life-threatening complications of their disease. In patients with severe disease, this high bone mass compromises bone marrow space leading to marrow failure and frequent infections, along with hepatosplenomegaly from extramedullary hematopoiesis.Ĭurrently, the only treatment for individuals with severe forms of osteopetrosis is hematopoietic cell transplantation (HCT), however survival in patients with osteopetrosis treated with HCT is only around 55%. Owing to the size and duration of the trial, a clinically significant survival benefit could not be ruled out.Osteopetrosis is a rare inherited metabolic bone disease characterized by impaired osteoclast function resulting in defective bone resorption and generalized high bone mass and mineral density (BMD). In a well-defined population of patients with idiopathic pulmonary fibrosis, interferon gamma-1b did not affect progression-free survival, pulmonary function, or the quality of life. More pneumonias were reported among patients in the interferon gamma-1b group, but the incidence of severe or life-threatening respiratory tract infections was similar in the two groups. However, the rates of treatment adherence and premature discontinuation of treatment were similar in the two groups. Treatment with interferon gamma-1b was associated with more frequent constitutional symptoms. Ten percent of patients in the interferon gamma-1b group died, as compared with 17 percent of patients in the placebo group (P=0.08). Over a median of 58 weeks, interferon gamma-1b therapy did not significantly affect the primary end point of progression-free survival, defined as the time to disease progression or death, and no significant treatment effect was observed on measures of lung function, gas exchange, or the quality of life. In a double-blind, multinational trial, we randomly assigned 330 patients with idiopathic pulmonary fibrosis that was unresponsive to corticosteroid therapy to receive subcutaneous interferon gamma-1b or placebo. Any interferon in breastmilk is probably destroyed in the infant's gastrointestinal tract and not absorbed, except perhaps in neonates. However, the amounts of the similar drugs, interferon alfa and interferon beta-1a, excreted into milk are very low. No data are available on the use of exogenous interferon gamma 1b during breastfeeding. Idiopathic pulmonary fibrosis is a progressive, fatal disease with no known efficacious therapy. Interferon gamma is a normal component of human milk.
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