![]() ![]() ![]() The primary endpoint will measure the change in neurological outcome and evaluate the effect of ACTIMMUNE versus placebo as measured by the modified Friedreich's Ataxia Rating Scale (mFARS), focused on objective neurologic measures such as upper and lower extremity coordination improvement from baseline. Approximately 90 patients will be enrolled at four sites in the United States. This Phase 3 trial (NCT02415127) is a randomized, multi-center, double-blind, placebo-controlled study with patients randomized 1:1 to receive subcutaneous doses of either ACTIMMUNE or placebo three times a week for a total of 26 weeks. We look forward to continuing to work with the FDA with the goal of providing a potential treatment option to the FA community." "The Phase 3 study represents a significant next step in evaluating ACTIMMUNE in this population, which is approximately four thousand people in the United States. ![]() Walbert, chairman, president and chief executive officer, Horizon Pharma plc. "As a rare disease with no currently approved treatments, Friedreich's Ataxia represents an area of significant unmet medical need," said Timothy P. Horizon Pharma plc (NASDAQ: HZNP), a biopharmaceutical company focused on improving patients' lives by identifying, developing, acquiring and commercializing differentiated and accessible medicines that address unmet medical needs, today announced it has initiated the Phase 3 Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia study (" STEADFAST") of ACTIMMUNE (interferon gamma-1b) for the treatment of people with Friedreich's Ataxia (FA), a degenerative neuro-muscular disorder. Horizon Pharma plc Initiates Phase 3 Trial of ACTIMMUNE(R) (interferon gamma-1b) for the Treatment of People With Friedreich's Ataxia 27 KBĭUBLIN, IRELAND - ( Marketwired) - 06/05/15. ![]()
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