Study type

Study type

Non-interventional study

Scope of the study

Effectiveness study (incl. comparative)
Other

If ‘other’, further details on the scope of the study

Evaluation of long-term safety and effectiveness of taliglucerase alfa, including effects on pregnancy and fetal outcomes, and newborns and infants who breastfeed from mothers treated with taliglucerase alfa
Non-interventional study

Non-interventional study design

Cohort
Study drug and medical condition

Medicinal product name, other

Elelyso

Medical condition to be studied

Gaucher's disease
Population studied

Age groups

  • Children (2 to < 12 years)
  • Adolescents (12 to < 18 years)
  • Adults (18 to < 46 years)
  • Adults (46 to < 65 years)
  • Adults (65 to < 75 years)
  • Adults (75 to < 85 years)
  • Adults (85 years and over)

Special population of interest

Pregnant women

Estimated number of subjects

1
Study design details

Main study objective

The main objectives of the registry are to characterize the safety profile of taliglucerase alfa through the solicited collection and summary of non serious and serious adverse event data and to characterize the effectiveness of taliglucerase alfa through the collection and analysis of Gaucher disease measures

Outcomes

Reports of all serious adverse events and non serious adverse events that occur during follow up and Gaucher disease measures including hematologic (hemoglobin and platelet count) and organ volume (spleen and liver) assessments, Sub-study outcomes that include reports of all relevant pregnancy, fetal, neonatal and infant outcomes

Data analysis plan

Data will be analyzed using descriptive statistics. For outcomes of interest, summary statistics, including counts and frequencies will be calculated. Crude cumulative incidence, and crude incidence rates per person-time will be calculated as appropriate. Depending on the outcome of interest, stratified analyses may be performed. Further exploratory analyses will be developed as necessary