Pfizer Registry of Outcomes in Growth hormone RESearch (PROGRES): A multi country, non-interventional prospective cohort study among patients with human growth hormone (hGH) treatments under routine clinical care

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Disease epidemiology

  Effectiveness study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  NGENLA
• Anatomical Therapeutic Chemical (ATC) code:
  (H01AC01) somatropin

  somatropin

  (H01AC08) somatrogon

  somatrogon
• Medical condition to be studied:
  Growth hormone deficiency

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:
  600

Study design details

• Main study objective:
  The purpose of this multi-country, non interventional prospective cohort study will be to assess the long term safety and effectiveness of Genotropin, other daily hGH treatments, and Ngenla, a long-acting hGH, once granted marketing authorization and is commercially available, all of which is at the discretion of the treating physician under routine clinical care.
• Outcomes:
  Primary Objectives: ; - To estimate incidence rates (IRs) of safety events of interest among patients on Genotropin, other daily hGH treatments and Ngenla. ; - To describe and compare effectiveness of Genotropin, other daily hGH treatments and Ngenla. ; ; Secondary Objectives: ; - To evaluate treatment adherence and compliance of Genotropin, other daily hGH treatments and Ngenla. ; - To evaluate the health-related quality of life (HRQoL) and treatment experience of patients on Genotropin, other daily hGH treatments and Ngenla. ; - To compare incidence rates of safety events in patients on Genotropin, other daily hGH and Ngenla by exploratory analysis
• Data analysis plan:
  Descriptive analyses will be performed to gain an understanding of the qualitative and quantitative nature of the data collected and the characteristics of the sample studied. ; Descriptive statistics for continuous variables include the number of observations, mean, median, standard deviation, interquartile range, and range. Categorical variables will be presented as number of observations, frequency, and percentage, 2-sided 95% confidence intervals (CIs) will be included as appropriate. ; All medical history, comorbidities and AEs will be recorded and coded using the most recent version of Medical Dictionary for Regulatory Activities. ; All exposures and concomitant medications will be recorded and coded using World Health Organization (WHO) Drug Dictionary. ; Safety data will be presented in tabular and/or listing format and summarized descriptively, where appropriate. Event count and AE frequency will be reported. Number (and %) with AEs, SAEs, and discontinuations will be reported.

Documents

Study report

c0311015-report-body.pdf