Study type

Study topic

Disease /health condition
Human medicinal product

Study type

Non-interventional study

Scope of the study

Assessment of risk minimisation measure implementation or effectiveness
Drug utilisation
Effectiveness study (incl. comparative)
Safety study (incl. comparative)

Data collection methods

Combined primary data collection and secondary use of data
Non-interventional study

Non-interventional study design

Cohort
Other

Non-interventional study design, other

Post-authorisation, prospective and retrospective, single cohort, uncontrolled, multi-centre European and US study
Study drug and medical condition

Name of medicine

OBIZUR

Medical condition to be studied

Acquired haemophilia
Population studied

Short description of the study population

A subject must be prescribed Obizur for the treatment of a bleeding episode by a physician, independent of and prior to the decision to enrol the subject in the study.

Inclusion Criteria
Subjects who meet ALL of the following criteria are eligible for this study:
1. Adult subject or legal authorised representative is willing to provide informed consent, unless informed consent is not required (e.g. subjects who are deceased),
as local regulations allow.
2. Subject is being treated or was treated with Obizur in routine clinical practice

Exclusion Criteria
Subjects who meet ANY of the following criteria are not eligible for this study:
1. Subject has participated in a clinical study involving a medicinal product or device within 30 days prior to enrolment or is scheduled to participate in a clinical study involving a medicinal product or device at study entry
2. Subject has known anaphylactic reactions to the active substance, hamster protein, or to any of the excipients of Obizur listed in the SPC /PL
 The following list of excipients can be found in the SPC/PL:
o Powder:
 Polysorbate 80
 Sodium chloride
 Calcium chloride dihydrate
 Sucrose
 Tris Base
 Tris HCl
 Tri-sodium citrate dihydrate
o Solvent:
 Sterilised water for injections
3. US Subject who has participated in the post-marketing study, NCT2610127

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)

Estimated number of subjects

50
Study design details

Main study objective

The primary objective of the study is to document the safety of subjects treated with Obizur in real-life clinical practice by recording any AEs, including but not limited to: - Hypersensitivity reactions - Thromboembolic events - Dose dispensing medication errors

Outcomes

The primary endpoint is safety, which will be assessed by adverse event (AE)/ serious adverse event (SAE) frequency, seriousness, severity and outcome (subject recovered/ recovered with sequelae, not recovered/ fatal). Particular attention will be given to the following adverse event of special interest (AESI): hypersensitivity reactions, thromboembolic events and dose dispensing medication errors, - Immunogenicity: newly recognised anti-pFVIII inhibitors or increase in titre of anti-pFVIII inhibitors and evolution of titre over time - Clinical characteristics of the treated subject population - Overall effectiveness assessment for resolution of bleeding determined as either bleeding stopped or did not stop - Time and dosage administered to achieve bleeding control

Data analysis plan

Categorical variables will be summarised by absolute and relative frequencies (number of valid and missing observations and percentages). Continuous variables will be summarised by descriptive statistics (number of valid and missing observations, mean, standard deviation, median, interquartile range, minimum, and maximum). Two-sided 95% CI will be provided for the main statistical estimator.
Documents
Study results

241501-clinical-study-report-redact

English (196.35 KB - PDF)View document