Study 1: Untreated Patient Study
Cerdelga is the ONLY first-line oral therapy indicated for the long-term
treatment of most adults with Gaucher disease type 1.
Tamara and Nate, USA,
patients with Gaucher disease type 1 treated with Cerdelga
Study 1 involved 40 adult patients with Gaucher disease type 1 who had not been on treatment or who were no longer receiving treatment. Spleen size, liver size, platelet levels, and hemoglobin levels were evaluated.
40 patients with Gaucher disease type 1 who were at least 16 years of age.
No study personnel, including the doctors, knew which patients were taking Cerdelga and which were taking placebo.
The duration of the primary analysis phase was 9 months. 20 patients were taking Cerdelga, and 20 taking placebo.
Some patients continued in the study for up to 2 years. After the 9-month primary analysis phase, patients in the placebo group had the opportunity to switch to Cerdelga. 39 of 40 patients continued in the study.
In the long-term, open-label extension phase, patients taking Cerdelga showed the following changes in spleen and liver size and in hemoglobin and platelet levels
The most common side effects
(occurring in ≥10% patients)
with Cerdelga (eliglustat)
| Side effect | Cerdelga (n=20) |
Placebo (n=20) |
|---|---|---|
| Number of patients (%) | ||
| Joint Pain | 9 (45%) | 2 (10%) |
| Headache | 8 (40%) | 6 (30%) |
| Migraine | 2 (10%) | 0 (0%) |
| Flatulence | 2 (10%) | 1 (5%) |
| Nausea | 2 (10%) | 1 (5%) |
| Mouth Pain | 2 (10%) | 1 (5%) |
