Study type

Study type

Non-interventional study

Scope of the study

Assessment of risk minimisation measure implementation or effectiveness
Disease epidemiology
Drug utilisation
Effectiveness study (incl. comparative)
Non-interventional study

Non-interventional study design

Other

Non-interventional study design, other

Longitudinal observational registry
Study drug and medical condition

Name of medicine

Strensiq

Medical condition to be studied

Hypophosphatasia
Population studied

Age groups

Preterm newborn infants (0 – 27 days)
Term newborn infants (0 – 27 days)
Infants and toddlers (28 days – 23 months)
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)

Estimated number of subjects

1100
Study design details

Main study objective

To collect information on the natural history of hypophosphatasia (HPP) from patients of all ages, including infants, children, and adults with HPP, regardless of age at onset. To characterize the epidemiology of the HPP population. To collect and evaluate long-term safety and effectiveness data in HPP patients who have/are receiving treatment with asfotase alfa.

Data analysis plan

Prior to the conduct of data analysis each year, details of planned analyses and patient cohorts will be prespecified in an a priori Epidemiological and Statistical Analysis Plan (ESAP). Categorical variables will be described using frequencies and percentages and modeled using logistic regression, while continuous variables will be described using means, standard deviations, medians, and inter-quartile ranges with modeling accomplished through generalized linear models, where appropriate. Study results will be summarized and reviewed at appropriate intervals based on patient enrollment, scientific considerations, and regulatory requirements. At a minimum, study results will be summarized annually and reported, and may include patient and clinical characteristics, as well as safety and effectiveness outcomes. Following termination of the Registry, a final analysis and report will also be prepared.
Documents
Study publications
Martos-Moreno GÁ, Linglart A, Petryk A, Kishnani PS, Rockman-Greenberg C, Dahir…
Seefried L, Dahir K, Petryk A, Högler W, Linglart A, Martos‐Moreno GÁ, Ozono K,…
Högler W, Langman C, Gomes da Silva H, Fang S, Linglart A, Ozono K, Petryk A, R…
Linglart et al. Frequency of ectopic calcifications among patients with hypopho…
Dahir KM, Angel MM, Linglart A, Petryk A, Kishnani PS, Rockman-Greenberg C, Mar…