AN OBSERVATIONAL, LONGITUDINAL, PROSPECTIVE, LONG-TERM REGISTRY OF PATIENTS WITH HYPOPHOSPHATASIA (ALX-HPP-501)

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.