A cross-sectional study of patients with immune thrombocytopenic purpura and caregivers to estimate the proportion who administer romiplostim correctly after receipt of home administration training materials (20120269)

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Effectiveness study (incl. comparative)
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Cross-sectional

Study drug and medical condition

• Study drug International non-proprietary name (INN) or common name:
  ROMIPLOSTIM
• Medical condition to be studied:
  Immune thrombocytopenia

Population studied

• Short description of the study population:
  Adult Immune thrombocytopenic purpura (ITP) patient, treated per EU SmPC, or caregiver new (or at least a 3-month gap) to romiplostim administration; who had received Home Administration Training (HAT) pack training and were available at standard-of-care medical visit 4 weeks (range 2 to 8 weeks) after HAT pack training; and provided informed consent.
• Age groups:
  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:
  Immunocompromised
• Estimated number of subjects:
  40

Study design details

• Main study objective:
  The primary objective of this study is to estimate the proportion of adult subjects and caregivers who correctly adminster romiplostim after being trained with the home administration training pack.
• Outcomes:
  Successful self administration of romiplostim Time Frame: First Standard of Care visit post Home Administration Training (range 2-8 weeks) , Successful reconstitution of romiplostimAccuracy in administering the prescribed dose of romiplostimSuccessful injection of romiplostimSuccessful self administration of romiplostim at follow up visits
• Data analysis plan:
  The primary endpoint is a categorical yes/no indicator for whether the subject or caregiver administers romiplostim correctly at the 4 week visit. It is a composite endpoint based on a number of criteria and will be "yes" if all are met, and “no” if any of the criteria are not met. The data analysis for this study will be descriptive in nature. Endpoints/variables of a binary nature, such as the primary endpoint, will be summarized as the frequency and proportion (percentage). As a measure of precision, a 95% confidence interval (binomial exact) will be calculated around the point estimate (proportion). Endpoints/variables of a continuous nature will be summarized using the mean, standard deviation, range and median. The target sample size is 40 subjects. With this sample size, the 95% confidence interval around an observed proportion of 90% of subjects or caregivers correctly administering romiplostim is 77% to 96%.

Documents

Study results

01.09.01 Clinical Study Report 2016-04-04 20120269 Final report (abstract, pages 8-11)