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

January 31, 2024 updated by: Swedish Orphan Biovitrum

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

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Observational

Enrollment (Actual)

44

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Paris, France
        • Swedish Orphan Biovitrum Research Site
      • Rennes, France
        • Swedish Orphan Biovitrum Research Site
      • Tours, France
        • Swedish Orphan Biovitrum Research Site
  • Germany
      • Berlin-Friedrichshain, Germany
        • Swedish Orphan Biovitrum Research Site
      • Berlin-Mitte, Germany
        • Swedish Orphan Biovitrum Research Site
      • Frankfurt, Germany
        • Swedish Orphan Biovitrum Research Site
      • Hannover, Germany
        • Swedish Orphan Biovitrum Research Site
  • Ireland
      • Dublin, Ireland
        • Swedish Orphan Biovitrum Research Site
  • Italy
      • Catania, Italy
        • Swedish Orphan Biovitrum Research Site
      • Catanzaro, Italy
        • Swedish Orphan Biovitrum Research Site
      • Genova, Italy
        • Swedish Orphan Biovitrum Research Site
      • Napoli, Italy
        • Swedish Orphan Biovitrum Research Site
  • Kuwait
      • Kuwait City, Kuwait
        • Swedish Orphan Biovitrum Research Site
  • Norway
      • Oslo, Norway
        • Swedish Orphan Biovitrum Research Site
  • Saudi Arabia
      • Riyadh, Saudi Arabia, 12233
        • Swedish Orphan Biovitrum Research Site
      • Riyadh, Saudi Arabia, 12713
        • Swedish Orphan Biovitrum Research Site (a)
      • Riyadh, Saudi Arabia, 12713
        • Swedish Orphan Biovitrum Research Site (p)
  • Switzerland
      • Bern, Switzerland
        • Swedish Orphan Biovitrum Research Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

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.

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Other

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Hemophili A patients
Patients treated with rFVIIIFc for ITI
Other: rFVIIIFc
Drug according to prescription
Other Names:
  • Elocta

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
ITI with rFVIIIFc: Main dose
Time Frame: From 2018 to 2022
Main dose will be assessed on the prescribed dose (IU/kg)
From 2018 to 2022
ITI with rFVIIIFc: Main injection frequency
Time Frame: From 2018 to 2022
Main injection frequency will be assessed on the prescribed frequency
From 2018 to 2022
ITI with rFVIIIFc: Duration
Time Frame: From 2018 to 2022
Number of treatment months
From 2018 to 2022
ITI with rFVIIIFc: Concomitant by-passing agents
Time Frame: From 2018 to 2022
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
Time Frame: From 2018 to 2022
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
Time Frame: From 2018 to 2022
Treatment time to reach undetectable inhibitor levels (<0.6 BU/ml)
From 2018 to 2022
Outcome of ITI with rFVIIIFc: Time to normal recovery
Time Frame: From 2018 to 2022
Treatment time to reach normal recovery levels (≥66% of the expected value)
From 2018 to 2022
Outcome of ITI with rFVIIIFc: Time to success
Time Frame: From 2018 to 2022
Treatment time to reach success (see outcome #5)
From 2018 to 2022
Outcome of ITI with rFVIIIFc: Inhibitor titer levels
Time Frame: From 2018 to 2022
BU/ml
From 2018 to 2022
Outcome of ITI with rFVIIIFc: Half-life
Time Frame: From 2018 to 2022
FVIII half-life (hours)
From 2018 to 2022
Outcome of ITI with rFVIIIFc: Recovery level
Time Frame: From 2018 to 2022
FVIII recovery level (%)
From 2018 to 2022
Outcome of ITI with rFVIIIFc: Bleeds
Time Frame: From 2018 to 2022
Number of bleeds per month during ITI-treatment
From 2018 to 2022
Long-term outcome after ITI with rFVIIIFc: Occurrence of relapse
Time Frame: From 2018 to 2022
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 Frame: From 2018 to 2022
Time to occurrence of relapse (see outcome #13)
From 2018 to 2022
Long-term outcome after ITI with rFVIIIFc: Treatment regimen
Time Frame: From 2018 to 2022
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
Time Frame: From 2018 to 2022
Number of bleeds per month.
From 2018 to 2022

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Swedish Orphan Biovitrum

Collaborators

Cerner Enviza

Investigators

  • Study Director: Stefan Lethagen, Swedish Orphan Biovitrum

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 7, 2018

Primary Completion (Actual)

September 30, 2022

Study Completion (Actual)

September 30, 2022

Study Registration Dates

First Submitted

September 19, 2018

First Submitted That Met QC Criteria

May 13, 2019

First Posted (Actual)

May 15, 2019

Study Record Updates

Last Update Posted (Estimated)

February 1, 2024

Last Update Submitted That Met QC Criteria

January 31, 2024

Last Verified

January 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • Sobi.Elocta-004

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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