Clinical Trial Results
The benefits of Soliris® (eculizumab) were studied in 3 clinical trials
Study 1 (TRIUMPH Study)
- To qualify for the TRIUMPH study, all patients were required to have at least 4 blood transfusions in the 12 months before the study began. A total of 87 PNH patients participated in this study
- In this study, 43 PNH patients received Soliris, and 44 PNH patients received a placebo (an infusion with no Soliris in it). Neither the patients nor the doctors knew which patients were receiving the drug
- The study lasted 26 weeks. All patients who received Soliris had a dramatic reduction in red blood cell destruction (hemolysis) compared to no change in hemolysis in the patients who received a placebo. The dramatic reduction with Soliris continued throughout the study
- Soliris treatment led to reduced need for transfusions. Approximately half of the patients treated with Soliris did not require a blood transfusion during the study (compared to all patients needing a transfusion in the placebo group)
- Soliris treatment was also associated with a significant improvement in fatigue that was assessed by scores of the FACIT-Fatigue instrument
- By the 3rd week of the study, patients on Soliris reported less fatigue and better health-related quality of life compared to the patients taking the placebo
- The most common side effects reported in the Soliris group were headache, runny nose (nasopharyngitis), back pain, and nausea. The number of headaches that occurred was similar in the Soliris and placebo groups after the first 2 weeks of therapy
Study 2 (SHEPHERD Study)
- The SHEPHERD study evaluated a broader PNH patient population including individuals who had lower platelet counts and required fewer transfusions
- To qualify for the SHEPHERD study, patients were required to have at least 1 transfusion in the past 2 years before the study began. A total of 97 patients participated in this study and were given Soliris for 1 year
- The study lasted for 52 weeks. Treatment with Soliris reduced hemolysis in all patients for the entire study. Soliris treatment also led to a reduction of blood clots (thrombosis), reduced need for blood transfusions, and a significant improvement in fatigue and overall quality of life
- In addition, Soliris was found to have a similar safety profile to Study 1 and was well tolerated over the study period of 1 year. The most common side effects reported in the Soliris group were headache, runny nose (nasopharyngitis), back pain, and nausea. The number of headaches that occurred was similar in the Soliris and placebo groups after the first 2 weeks of therapy
Study 3 (Extension Study)
- PNH patients who enrolled in TRIUMPH and SHEPHERD continued in a long-term extension study
- In total, almost 200 patients were observed and some of these PNH patients were taking Soliris for over 5 years
- These patients experienced a reduction in hemolysis for the length of the study (ranging from 10 to 54 months). In addition, patients had fewer blood clots during the study period than during the same period of time prior to treatment with Soliris