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Trial record 2 of 4 for:    swedish orphan | oslo, Norway | Child
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rFVIIIFc (Elocta®) ITI Chart Review in Patients With Haemophilia A

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03951103
Recruitment Status : Active, not recruiting
First Posted : May 15, 2019
Last Update Posted : May 12, 2022
Sponsor:
Collaborator:
Kantar Health
Information provided by (Responsible Party):
Swedish Orphan Biovitrum

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

Condition or disease Intervention/treatment
Hemophilia A With Inhibitor Other: rFVIIIFc

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 Design
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Study Type : Observational
Estimated Enrollment : 45 participants
Observational Model: Cohort
Time Perspective: Other
Official Title: A Chart Review Study of Patients With Haemophilia A With Inhibitors Treated With rFVIIIFc (Elocta®) for Immune Tolerance Induction
Actual Study Start Date : November 7, 2018
Estimated Primary Completion Date : September 30, 2022
Estimated Study Completion Date : December 30, 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Groups and Cohorts
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Group/Cohort Intervention/treatment
Hemophili A patients
Patients treated with rFVIIIFc for ITI
 Other: rFVIIIFc
Drug according to prescription
Other Name: Elocta


Outcome Measures
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Primary Outcome Measures :
  1. ITI with rFVIIIFc: Main dose [ Time Frame: From 2018 to 2022 ]
    Main dose will be assessed on the prescribed dose (IU/kg)

  2. ITI with rFVIIIFc: Main injection frequency [ Time Frame: From 2018 to 2022 ]
    Main injection frequency will be assessed on the prescribed frequency

  3. ITI with rFVIIIFc: Duration [ Time Frame: From 2018 to 2022 ]
    Number of treatment months

  4. 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.

  5. 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.

  6. 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)

  7. 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)

  8. Outcome of ITI with rFVIIIFc: Time to success [ Time Frame: From 2018 to 2022 ]
    Treatment time to reach success (see outcome #5)

  9. Outcome of ITI with rFVIIIFc: Inhibitor titer levels [ Time Frame: From 2018 to 2022 ]
    BU/ml

  10. Outcome of ITI with rFVIIIFc: Half-life [ Time Frame: From 2018 to 2022 ]
    FVIII half-life (hours)

  11. Outcome of ITI with rFVIIIFc: Recovery level [ Time Frame: From 2018 to 2022 ]
    FVIII recovery level (%)

  12. Outcome of ITI with rFVIIIFc: Bleeds [ Time Frame: From 2018 to 2022 ]
    Number of bleeds per month during ITI-treatment

  13. 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.

  14. Long-term outcome after ITI with rFVIIIFc: Time to relapse [ Time Frame: From 2018 to 2022 ]
    Time to occurrence of relapse (see outcome #13)

  15. 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.

  16. Long-term outcome after ITI with rFVIIIFc: Bleeds [ Time Frame: From 2018 to 2022 ]
    Number of bleeds per month.


Eligibility Criteria
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Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
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.
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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03951103


Locations
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France
Swedish Orphan Biovitrum Research Site
Paris, France
Swedish Orphan Biovitrum Research Site
Rennes, France
Swedish Orphan Biovitrum Research Site
Tours, France
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, Germany
Ireland
Swedish Orphan Biovitrum Research Site
Dublin, Ireland
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, Italy
Kuwait
Swedish Orphan Biovitrum Research Site
Kuwait City, Kuwait
Norway
Swedish Orphan Biovitrum Research Site
Oslo, Norway
Saudi Arabia
Swedish Orphan Biovitrum Research Site
Riyadh, Saudi Arabia, 12233
Swedish Orphan Biovitrum Research Site (a)
Riyadh, Saudi Arabia, 12713
Swedish Orphan Biovitrum Research Site (p)
Riyadh, Saudi Arabia, 12713
Switzerland
Swedish Orphan Biovitrum Research Site
Bern, Switzerland
Sponsors and Collaborators
Swedish Orphan Biovitrum
Kantar Health
Investigators
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Study Director: Stefan Lethagen Swedish Orphan Biovitrum
More Information
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Responsible Party: Swedish Orphan Biovitrum
ClinicalTrials.gov Identifier: NCT03951103    
Other Study ID Numbers: Sobi.Elocta-004
First Posted: May 15, 2019    Key Record Dates
Last Update Posted: May 12, 2022
Last Verified: May 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Swedish Orphan Biovitrum:
Elocta
rFVIIIFc
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
 Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn