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

Sponsor
Swedish Orphan Biovitrum (Industry)
Overall Status
Active, not recruiting
CT.gov ID
NCT03951103
Collaborator
Kantar Health (Industry)
45
Enrollment
18
Locations
49.7
Anticipated Duration (Months)
2.5
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

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 Phase
  • 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

Study Type:
Observational
Anticipated 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 :
Nov 7, 2018
Anticipated Primary Completion Date :
Sep 30, 2022
Anticipated Study Completion Date :
Dec 30, 2022

Arms and Interventions

Arm Intervention/Treatment
Hemophili A patients

Patients treated with rFVIIIFc for ITI

Other: rFVIIIFc
Drug according to prescription
Other Names:
  • Elocta

    		•

    Outcome Measures

    Primary Outcome Measures

    1. ITI with rFVIIIFc: Main dose [From 2018 to 2022]

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

    2. ITI with rFVIIIFc: Main injection frequency [From 2018 to 2022]

      Main injection frequency will be assessed on the prescribed frequency

    3. ITI with rFVIIIFc: Duration [From 2018 to 2022]

      Number of treatment months

    4. ITI with rFVIIIFc: Concomitant by-passing agents [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 [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 [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 [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 [From 2018 to 2022]

      Treatment time to reach success (see outcome #5)

    9. Outcome of ITI with rFVIIIFc: Inhibitor titer levels [From 2018 to 2022]

      BU/ml

    10. Outcome of ITI with rFVIIIFc: Half-life [From 2018 to 2022]

      FVIII half-life (hours)

    11. Outcome of ITI with rFVIIIFc: Recovery level [From 2018 to 2022]

      FVIII recovery level (%)

    12. Outcome of ITI with rFVIIIFc: Bleeds [From 2018 to 2022]

      Number of bleeds per month during ITI-treatment

    13. Long-term outcome after ITI with rFVIIIFc: Occurrence of relapse [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 [From 2018 to 2022]

      Time to occurrence of relapse (see outcome #13)

    15. Long-term outcome after ITI with rFVIIIFc: Treatment regimen [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 [From 2018 to 2022]

      Number of bleeds per month.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    N/A and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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

    Locations

    Site City State Country Postal Code
    1 Swedish Orphan Biovitrum Research Site Paris France
    2 Swedish Orphan Biovitrum Research Site Rennes France
    3 Swedish Orphan Biovitrum Research Site Tours France
    4 Swedish Orphan Biovitrum Research Site Berlin-Friedrichshain Germany
    5 Swedish Orphan Biovitrum Research Site Berlin-Mitte Germany
    6 Swedish Orphan Biovitrum Research Site Frankfurt Germany
    7 Swedish Orphan Biovitrum Research Site Hannover Germany
    8 Swedish Orphan Biovitrum Research Site Dublin Ireland
    9 Swedish Orphan Biovitrum Research Site Catania Italy
    10 Swedish Orphan Biovitrum Research Site Catanzaro Italy
    11 Swedish Orphan Biovitrum Research Site Genova Italy
    12 Swedish Orphan Biovitrum Research Site Napoli Italy
    13 Swedish Orphan Biovitrum Research Site Kuwait City Kuwait
    14 Swedish Orphan Biovitrum Research Site Oslo Norway
    15 Swedish Orphan Biovitrum Research Site Riyadh Saudi Arabia 12233
    16 Swedish Orphan Biovitrum Research Site (a) Riyadh Saudi Arabia 12713
    17 Swedish Orphan Biovitrum Research Site (p) Riyadh Saudi Arabia 12713
    18 Swedish Orphan Biovitrum Research Site Bern Switzerland

    Sponsors and Collaborators

    • Swedish Orphan Biovitrum
    • Kantar Health

    Investigators

    • Study Director: Stefan Lethagen, Swedish Orphan Biovitrum

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Swedish Orphan Biovitrum
    ClinicalTrials.gov Identifier:
    NCT03951103
    Other Study ID Numbers:
    • Sobi.Elocta-004
    First Posted:
    May 15, 2019
    Last Update Posted:
    May 12, 2022
    Last Verified:
    May 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    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:
    Hemophilia A

    Study Results

    No Results Posted as of May 12, 2022