Clinical trial NCT03951103: rFVIIIFc (Elocta®) ITI Chart Review in Patients With Haemophilia A
Official title: A Chart Review Study of Patients With Haemophilia A With Inhibitors Treated With rFVIIIFc (Elocta®) for Immune Tolerance Induction
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hemophilia A With Inhibitor
Keywords: Elocta
rFVIIIFc
Lead sponsor: Swedish Orphan Biovitrum
Sponsor class: INDUSTRY
First posted: 2019-05-15
Enrollment: 44
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: All haemophilia A patients who have been, or who are currently treated with rFVIIIFc for ITI meeting the inclusion and not meeting the exclusion criteria will be invited to participate. Approximately 45 patients from Europe and the Middle East are expected to be included in the study.

Brief summary: A chart review study of patients with haemophilia A with inhibitors treated with rFVIIIFc (Elocta®) for immune tolerance induction.

Detailed description: A multicenter, international, non- interventional, retrospective and prospective medical chart review study. Data will be collected from medical records for patients diagnosed with haemophilia A who have been, or who are currently, treated with rFVIIIFc for ITI . The study will be descriptive in nature and report on baseline characteristics, treatment and outcomes for patients who have been, or who are currently, treated with rFVIIIFc for ITI.

Eligibility criteria: Inclusion Criteria:

* Patients diagnosed with haemophilia A who have been, or who are currently, treated with rFVIIIFc for ITI.
* Signed and dated informed consent provided by the patient, or the patient's legally acceptable representative for patients under the legal age, before any study-related data collection are undertaken. Assent should be obtained from paediatric patients according to local regulations.

Exclusion Criteria:

* Current participation in any investigational medicinal product trial.

Primary outcomes:
measure: ITI with rFVIIIFc: Main dose
timeFrame: From 2018 to 2022
measure: ITI with rFVIIIFc: Main injection frequency
timeFrame: From 2018 to 2022
measure: ITI with rFVIIIFc: Duration
timeFrame: From 2018 to 2022
measure: ITI with rFVIIIFc: Concomitant by-passing agents
timeFrame: From 2018 to 2022
measure: Outcome of ITI with rFVIIIFc: Overall outcome
timeFrame: From 2018 to 2022
measure: Outcome of ITI with rFVIIIFc: Time to undetectable inhibitor titer
timeFrame: From 2018 to 2022
measure: Outcome of ITI with rFVIIIFc: Time to normal recovery
timeFrame: From 2018 to 2022
measure: Outcome of ITI with rFVIIIFc: Time to success
timeFrame: From 2018 to 2022
measure: Outcome of ITI with rFVIIIFc: Inhibitor titer levels
timeFrame: From 2018 to 2022
measure: Outcome of ITI with rFVIIIFc: Half-life
timeFrame: From 2018 to 2022
measure: Outcome of ITI with rFVIIIFc: Recovery level
timeFrame: From 2018 to 2022
measure: Outcome of ITI with rFVIIIFc: Bleeds
timeFrame: From 2018 to 2022
measure: Long-term outcome after ITI with rFVIIIFc: Occurrence of relapse
timeFrame: From 2018 to 2022
measure: Long-term outcome after ITI with rFVIIIFc: Time to relapse
timeFrame: From 2018 to 2022
measure: Long-term outcome after ITI with rFVIIIFc: Treatment regimen
timeFrame: From 2018 to 2022
measure: Long-term outcome after ITI with rFVIIIFc: Bleeds
timeFrame: From 2018 to 2022
Secondary outcomes:
Other outcomes:
Locations:
city: Paris
country: France
facility: Swedish Orphan Biovitrum Research Site
city: Rennes
country: France
facility: Swedish Orphan Biovitrum Research Site
city: Tours
country: France
facility: Swedish Orphan Biovitrum Research Site
city: Berlin-Friedrichshain
country: Germany
facility: Swedish Orphan Biovitrum Research Site
city: Berlin-Mitte
country: Germany
facility: Swedish Orphan Biovitrum Research Site
city: Frankfurt
country: Germany
facility: Swedish Orphan Biovitrum Research Site
city: Hannover
country: Germany
facility: Swedish Orphan Biovitrum Research Site
city: Dublin
country: Ireland
facility: Swedish Orphan Biovitrum Research Site
city: Catania
country: Italy
facility: Swedish Orphan Biovitrum Research Site
city: Catanzaro
country: Italy
facility: Swedish Orphan Biovitrum Research Site
city: Genova
country: Italy
facility: Swedish Orphan Biovitrum Research Site
city: Napoli
country: Italy
facility: Swedish Orphan Biovitrum Research Site
city: Kuwait City
country: Kuwait
facility: Swedish Orphan Biovitrum Research Site
city: Oslo
country: Norway
facility: Swedish Orphan Biovitrum Research Site
city: Riyadh
country: Saudi Arabia
facility: Swedish Orphan Biovitrum Research Site
city: Riyadh
country: Saudi Arabia
facility: Swedish Orphan Biovitrum Research Site (a)
city: Riyadh
country: Saudi Arabia
facility: Swedish Orphan Biovitrum Research Site (p)
city: Bern
country: Switzerland
facility: Swedish Orphan Biovitrum Research Site
Study officials:
affiliation: Swedish Orphan Biovitrum
name: Stefan Lethagen
role: STUDY_DIRECTOR
References: