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
English (44 KB - PDF)View document