rFVIIIFc (Elocta®) ITI Chart Review in Patients With Haemophilia A

Hemophilia A With Inhibitor Clinical Trial

Official title:

A Chart Review Study of Patients With Haemophilia A With Inhibitors Treated With rFVIIIFc (Elocta®) for Immune Tolerance Induction

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03951103
• Other study ID #: Sobi.Elocta-004
• Status: Completed
• Start date: November 7, 2018
• Est. completion date: September 30, 2022

Study information

• Verified date: January 2024
• Source: Swedish Orphan Biovitrum
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 44
• Est. completion date: September 30, 2022
• Est. primary completion date: September 30, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Hemophilia A
• Hemophilia A With Inhibitor

Intervention

Other:
• rFVIIIFc
Drug according to prescription

Locations

Country	Name	City	State
France	Swedish Orphan Biovitrum Research Site	Paris
France	Swedish Orphan Biovitrum Research Site	Rennes
France	Swedish Orphan Biovitrum Research Site	Tours
Germany	Swedish Orphan Biovitrum Research Site	Berlin-Friedrichshain
Germany	Swedish Orphan Biovitrum Research Site	Berlin-Mitte
Germany	Swedish Orphan Biovitrum Research Site	Frankfurt
Germany	Swedish Orphan Biovitrum Research Site	Hannover
Ireland	Swedish Orphan Biovitrum Research Site	Dublin
Italy	Swedish Orphan Biovitrum Research Site	Catania
Italy	Swedish Orphan Biovitrum Research Site	Catanzaro
Italy	Swedish Orphan Biovitrum Research Site	Genova
Italy	Swedish Orphan Biovitrum Research Site	Napoli
Kuwait	Swedish Orphan Biovitrum Research Site	Kuwait City
Norway	Swedish Orphan Biovitrum Research Site	Oslo
Saudi Arabia	Swedish Orphan Biovitrum Research Site	Riyadh
Saudi Arabia	Swedish Orphan Biovitrum Research Site (a)	Riyadh
Saudi Arabia	Swedish Orphan Biovitrum Research Site (p)	Riyadh
Switzerland	Swedish Orphan Biovitrum Research Site	Bern

Sponsors (2)

Lead Sponsor	Collaborator
Swedish Orphan Biovitrum	Cerner Enviza

Countries where clinical trial is conducted

France,  Germany,  Ireland,  Italy,  Kuwait,  Norway,  Saudi Arabia,  Switzerland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	ITI with rFVIIIFc: Main dose	Main dose will be assessed on the prescribed dose (IU/kg)	From 2018 to 2022
Primary	ITI with rFVIIIFc: Main injection frequency	Main injection frequency will be assessed on the prescribed frequency	From 2018 to 2022
Primary	ITI with rFVIIIFc: Duration	Number of treatment months	From 2018 to 2022
Primary	ITI with rFVIIIFc: Concomitant by-passing agents	Product name and main dose will be used to describe any concomitant use of by-passing agents.	From 2018 to 2022
Primary	Outcome of ITI with rFVIIIFc: Overall outcome	The Investigator will assess overall outcome as: success, partial success, failure, early withdrawal or other.	From 2018 to 2022
Primary	Outcome of ITI with rFVIIIFc: Time to undetectable inhibitor titer	Treatment time to reach undetectable inhibitor levels (<0.6 BU/ml)	From 2018 to 2022
Primary	Outcome of ITI with rFVIIIFc: Time to normal recovery	Treatment time to reach normal recovery levels (=66% of the expected value)	From 2018 to 2022
Primary	Outcome of ITI with rFVIIIFc: Time to success	Treatment time to reach success (see outcome #5)	From 2018 to 2022
Primary	Outcome of ITI with rFVIIIFc: Inhibitor titer levels	BU/ml	From 2018 to 2022
Primary	Outcome of ITI with rFVIIIFc: Half-life	FVIII half-life (hours)	From 2018 to 2022
Primary	Outcome of ITI with rFVIIIFc: Recovery level	FVIII recovery level (%)	From 2018 to 2022
Primary	Outcome of ITI with rFVIIIFc: Bleeds	Number of bleeds per month during ITI-treatment	From 2018 to 2022
Primary	Long-term outcome after ITI with rFVIIIFc: Occurrence of relapse	Occurrence of relapse (Yes/No) will be assessed by the investigator.	From 2018 to 2022
Primary	Long-term outcome after ITI with rFVIIIFc: Time to relapse	Time to occurrence of relapse (see outcome #13)	From 2018 to 2022
Primary	Long-term outcome after ITI with rFVIIIFc: Treatment regimen	Treatment regimen will be described as: ITI, prophylaxis or on-demand; and also by product used.	From 2018 to 2022
Primary	Long-term outcome after ITI with rFVIIIFc: Bleeds	Number of bleeds per month.	From 2018 to 2022