Clinical study results
Results were seen across multiple clinical end points studied in adult and pediatric patients.
In adults: 9-month study
Based on a study of previously untreated adults with Type 1 Gaucher disease, adults taking ELELYSO® (taliglucerase alfa) for injection showed improvement after 9 months of treatment across a range of symptoms.* This included a significant improvement in the main goal of the study, reduction in spleen size. It also included a significant reduction in liver size and increased hemoglobin level and platelet count.
*Data are from a 9-month pivotal trial of 31 patients with Type 1 Gaucher disease. The trial compared the effectiveness and safety of 2 different doses of ELELYSO. Patients were previously untreated and randomly chosen to receive either 30 or 60 Units of ELELYSO per kilogram of body weight every other week. The recommended dosage in treatment-naïve adult patients is 60 Units per kilogram of body weight every other week. ELELYSO 30 Units per kilogram of body weight is not a recommended dosage.
Adults taking 60 Units of ELELYSO per kilogram of body weight after 9 months experienced, on average:
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Mean Spleen Size (main study goal)
Decreased by 35%
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Mean Hemoglobin Level
Increased by 19%
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Mean Liver SizeDecreased by 12%
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Mean Platelet CountIncreased by 64%
In adults: extension study
In an extension of the initial 9-month study of ELELYSO, 26 previously untreated adult patients were followed for an additional 15 months in double-blind fashion. They showed continued improvement on treatment with ELELYSO after the initial study for a total treatment duration of 24 months.
Adults taking 60 Units of ELELYSO per kilogram of body weight after a total of 24 months experienced, on average:
-
Mean Spleen Size (main study goal)Decreased by 55%
-
Mean Hemoglobin LevelIncreased by 21%
-
Mean Liver SizeDecreased by 19%
-
Mean Platelet CountIncreased by 104%
In adults:continuation of the extension study
12 Months: Following the double-blind extension, 23 of the 26 patients continued open-label treatment with ELELYSO for an additional 12 months for a treatment duration of 36 months and demonstrated stability in the above clinical parameters.
24 Months: In a second extension trial, 19 of the 23 patients continued open-label treatment with ELELYSO for another 24 months resulting in a total treatment duration of 60 months.
Double-blind means that neither the researchers nor the patients knew which dose (30 or 60 Units of ELELYSO per kilogram of body weight) the patients were receiving.
Open label means that both the researchers and the patients knew which dose (30 or 60 Units of ELELYSO per kilogram of body weight) the patients were receiving.
In children: 12-month study
In a 12-month study in children with Type 1 Gaucher disease who were previously untreated, children taking ELELYSO showed improvements in the clinical end points of the study, which included a reduction of spleen and liver size and an increase in hemoglobin level and platelet count.†
†Data are from a 12-month clinical trial of 9 children ages 2-13 with Type 1 Gaucher disease. The trial compared the effectiveness and safety of 2 different doses of ELELYSO. Patients were previously untreated and randomly chosen to receive either 30 or 60 Units of ELELYSO per kilogram of body weight every other week. The recommended dosage in treatment-naïve pediatric patients 4 years of age and older is 60 Units per kilogram of body weight every other week. ELELYSO 30 Units per kilogram of body weight is not a recommended dosage. There are insufficient data to inform dosing in patients less than 4 years of age.
Children taking 60 Units of ELELYSO per kilogram of body weight after 12 months experienced, on average:
-
Median Hemoglobin Level Increased by 14%
-
Median Spleen SizeDecreased by 33%
-
Median Liver SizeDecreased by 16%
-
Median Platelet CountIncreased by 64%
In children: extension study
In an extension of the initial 12-month study of ELELYSO, all 9 of the previously untreated pediatric patients were followed for an additional 12 months in double-blind fashion. They showed continued improvement on treatment with ELELYSO after the initial study for a treatment duration of 24 months.
Children taking 60 Units of ELELYSO per kilogram of body weight after a total of 24 months experienced, on average:
-
Median Hemoglobin LevelIncreased by 22%
-
Median Spleen SizeDecreased by 61%
-
Median Liver SizeDecreased by 21%
-
Median Platelet CountIncreased by 87%
In children: 12-month continuation of the extension study
Following the 12 months of blinded treatment in the extension study, all 9 patients continued treatment in an open-label fashion through 36 months.
In adults and children: 9-month switch study
A 9-month study was done to assess the safety and effectiveness of ELELYSO in 26 adults and 5 children switching from Cerezyme®‡ (imiglucerase for injection) to ELELYSO. People in the study were switched from Cerezyme to ELELYSO at the same dosage.
Eighteen of the 26 adult patients who completed the 9-month clinical trial continued treatment with ELELYSO in an open-label extension trial for an additional 27 months (total treatment 36 months). Patients maintained stability in clinical parameters (spleen volume, liver volume, platelet count, and hemoglobin); however, only 10 of 18 adult patients completed 27 months of ELELYSO treatment in the extension trial and only 7 patients had their spleen and liver volumes assessed at 36 months.
‡Cerezyme is a registered trademark of Genzyme Corporation.
§Based on a 9-month, multicenter, open-label clinical trial of 26 adults and 5 children switching from Cerezyme to ELELYSO. All patients were clinically stable at the time of the clinical trial and had a stable biweekly dosage of imiglucerase for at least 6 months prior to enrollment.