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

September 17, 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

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Swedish Orphan Biovitrum

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Cerner Enviza

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • 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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
November 7, 2018

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
September 30, 2022

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
September 30, 2022

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
September 19, 2018

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 13, 2019

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
May 15, 2019

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
September 19, 2024

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 17, 2024

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
September 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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