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

NCT03951103 | Completed

• 1 other identifier: Sobi.Elocta-004
• Study Type: observational
• Target: 44
• Locations: 8 countries
• Sites: 18

Brief Summary

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

Conditions

Hemophilia A With Inhibitor

Keywords

Elocta; rFVIIIFc

Outcome Measures

Primary Outcomes (16)

• ITI with rFVIIIFc: Main dose

  Main dose will be assessed on the prescribed dose (IU/kg)

  From 2018 to 2022

• ITI with rFVIIIFc: Main injection frequency

  Main injection frequency will be assessed on the prescribed frequency

  From 2018 to 2022

• ITI with rFVIIIFc: Duration

  Number of treatment months

  From 2018 to 2022

• 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

• 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

• 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

• 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

• Outcome of ITI with rFVIIIFc: Time to success

  Treatment time to reach success (see outcome #5)

  From 2018 to 2022

• Outcome of ITI with rFVIIIFc: Inhibitor titer levels

  BU/ml

  From 2018 to 2022

• Outcome of ITI with rFVIIIFc: Half-life

  FVIII half-life (hours)

  From 2018 to 2022

• Outcome of ITI with rFVIIIFc: Recovery level

  FVIII recovery level (%)

  From 2018 to 2022

• Outcome of ITI with rFVIIIFc: Bleeds

  Number of bleeds per month during ITI-treatment

  From 2018 to 2022

• 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

• Long-term outcome after ITI with rFVIIIFc: Time to relapse

  Time to occurrence of relapse (see outcome #13)

  From 2018 to 2022

• 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

• Long-term outcome after ITI with rFVIIIFc: Bleeds

  Number of bleeds per month.

  From 2018 to 2022

Study Arms (1)

Hemophili A patients

Patients treated with rFVIIIFc for ITI

Other: rFVIIIFc

Interventions

rFVIIIFc OTHER

Drug according to prescription

Also known as: Elocta

Hemophili A patients

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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.

You may not qualify if:

• Current participation in any investigational medicinal product trial.

Sponsors & Collaborators

• Swedish Orphan Biovitrum: lead

Study Sites (18)

Swedish Orphan Biovitrum Research Site

Paris, France

Location

Swedish Orphan Biovitrum Research Site

Rennes, France

Location

Swedish Orphan Biovitrum Research Site

Tours, France

Location

Swedish Orphan Biovitrum Research Site

Frankfurt, Germany

Location

Swedish Orphan Biovitrum Research Site

Friedrichshain, Germany

Location

Swedish Orphan Biovitrum Research Site

Hanover, Germany

Location

Swedish Orphan Biovitrum Research Site

Mitte, Germany

Location

Swedish Orphan Biovitrum Research Site

Dublin, Ireland

Location

Swedish Orphan Biovitrum Research Site

Catania, Italy

Location

Swedish Orphan Biovitrum Research Site

Catanzaro, Italy

Location

Swedish Orphan Biovitrum Research Site

Genova, Italy

Location

Swedish Orphan Biovitrum Research Site

Napoli, Italy

Location

Swedish Orphan Biovitrum Research Site

Kuwait City, Kuwait

Location

Swedish Orphan Biovitrum Research Site

Oslo, Norway

Location

Swedish Orphan Biovitrum Research Site

Riyadh, 12233, Saudi Arabia

Location

Swedish Orphan Biovitrum Research Site (a)

Riyadh, 12713, Saudi Arabia

Location

Swedish Orphan Biovitrum Research Site (p)

Riyadh, 12713, Saudi Arabia

Location

Swedish Orphan Biovitrum Research Site

Bern, Switzerland

Location

Related Publications (1)

• Klamroth R, Al Saleh M, Glosli H, Schiavulli M, Guillet B, Bystricka L, Schonstein A, Lethagen S. Immune Tolerance Induction With a Recombinant Factor VIII Fc in Haemophilia A: Data From a Chart Review Study. Eur J Haematol. 2025 Aug;115(2):134-141. doi: 10.1111/ejh.14427. Epub 2025 Apr 27.

  PMID: 40289300 DERIVED

MeSH Terms

Interventions

factor VIII-Fc fusion protein

Study Officials

• Stefan Lethagen

  Swedish Orphan Biovitrum

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 19, 2018
• First Posted: May 15, 2019
• Study Start: November 7, 2018
• Primary Completion: September 30, 2022
• Study Completion: September 30, 2022
• Last Updated: September 19, 2024

Record last verified: 2024-09

Data Sharing

• IPD Sharing: Will not share

Locations

CH (1); DE (4); FR (3); SA (3); KU (1); IE (1); IT (4); NO (1)