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出境医 / 临床实验 / rFVIIIFc (Elocta®) ITI Chart Review in Patients With Haemophilia A

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

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
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
Layout table for study information
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
Arms and Interventions
Group/Cohort Intervention/treatment
Hemophili A patients
Patients treated with rFVIIIFc for ITI
 Other: rFVIIIFc
Drug according to prescription
Other Name: Elocta
Outcome Measures
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
Layout table for eligibility information
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

Locations
Layout table for location information
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
Layout table for investigator information
Study Director: Stefan Lethagen Swedish Orphan Biovitrum
Tracking Information
First Submitted Date September 19, 2018
First Posted Date May 15, 2019
Last Update Posted Date October 30, 2020
Actual Study Start Date November 7, 2018
Estimated Primary Completion Date September 30, 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: October 29, 2020)
  • ITI with rFVIIIFc: Main dose [ Time Frame: From 2018 to 2022 ]
    Main dose will be assessed on the prescribed dose (IU/kg)
  • ITI with rFVIIIFc: Main injection frequency [ Time Frame: From 2018 to 2022 ]
    Main injection frequency will be assessed on the prescribed frequency
  • ITI with rFVIIIFc: Duration [ Time Frame: From 2018 to 2022 ]
    Number of treatment months
  • 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.
  • 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.
  • 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)
  • 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)
  • Outcome of ITI with rFVIIIFc: Time to success [ Time Frame: From 2018 to 2022 ]
    Treatment time to reach success (see outcome #5)
  • Outcome of ITI with rFVIIIFc: Inhibitor titer levels [ Time Frame: From 2018 to 2022 ]
    BU/ml
  • Outcome of ITI with rFVIIIFc: Half-life [ Time Frame: From 2018 to 2022 ]
    FVIII half-life (hours)
  • Outcome of ITI with rFVIIIFc: Recovery level [ Time Frame: From 2018 to 2022 ]
    FVIII recovery level (%)
  • Outcome of ITI with rFVIIIFc: Bleeds [ Time Frame: From 2018 to 2022 ]
    Number of bleeds per month during ITI-treatment
  • 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.
  • Long-term outcome after ITI with rFVIIIFc: Time to relapse [ Time Frame: From 2018 to 2022 ]
    Time to occurrence of relapse (see outcome #13)
  • 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.
  • Long-term outcome after ITI with rFVIIIFc: Bleeds [ Time Frame: From 2018 to 2022 ]
    Number of bleeds per month.
Original Primary Outcome Measures
 (submitted: May 13, 2019)
  • ITI with rFVIIIFc: Main dose [ Time Frame: From 2018 to 2023 ]
    Main dose will be assessed on the prescribed dose (IU/kg)
  • ITI with rFVIIIFc: Main injection frequency [ Time Frame: From 2018 to 2023 ]
    Main injection frequency will be assessed on the prescribed frequency
  • ITI with rFVIIIFc: Duration [ Time Frame: From 2018 to 2023 ]
    Number of treatment months
  • ITI with rFVIIIFc: Concomitant by-passing agents [ Time Frame: From 2018 to 2023 ]
    Product name and main dose will be used to describe any concomitant use of by-passing agents.
  • Outcome of ITI with rFVIIIFc: Overall outcome [ Time Frame: From 2018 to 2023 ]
    The Investigator will assess overall outcome as: success, partial success, failure, early withdrawal or other.
  • Outcome of ITI with rFVIIIFc: Time to undetectable inhibitor titer [ Time Frame: From 2018 to 2023 ]
    Treatment time to reach undetectable inhibitor levels (<0.6 BU/ml)
  • Outcome of ITI with rFVIIIFc: Time to normal recovery [ Time Frame: From 2018 to 2023 ]
    Treatment time to reach normal recovery levels (≥66% of the expected value)
  • Outcome of ITI with rFVIIIFc: Time to success [ Time Frame: From 2018 to 2023 ]
    Treatment time to reach success (see outcome #5)
  • Outcome of ITI with rFVIIIFc: Inhibitor titer levels [ Time Frame: From 2018 to 2023 ]
    BU/ml
  • Outcome of ITI with rFVIIIFc: Half-life [ Time Frame: From 2018 to 2023 ]
    FVIII half-life (hours)
  • Outcome of ITI with rFVIIIFc: Recovery level [ Time Frame: From 2018 to 2023 ]
    FVIII recovery level (%)
  • Outcome of ITI with rFVIIIFc: Bleeds [ Time Frame: From 2018 to 2023 ]
    Number of bleeds per month during ITI-treatment
  • Long-term outcome after ITI with rFVIIIFc: Occurrence of relapse [ Time Frame: From 2018 to 2023 ]
    Occurrence of relapse (Yes/No) will be assessed by the investigator.
  • Long-term outcome after ITI with rFVIIIFc: Time to relapse [ Time Frame: From 2018 to 2023 ]
    Time to occurrence of relapse (see outcome #13)
  • Long-term outcome after ITI with rFVIIIFc: Treatment regimen [ Time Frame: From 2018 to 2023 ]
    Treatment regimen will be described as: ITI, prophylaxis or on-demand; and also by product used.
  • Long-term outcome after ITI with rFVIIIFc: Bleeds [ Time Frame: From 2018 to 2023 ]
    Number of bleeds per month.
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title rFVIIIFc (Elocta®) ITI Chart Review in Patients With Haemophilia A
Official Title A Chart Review Study of Patients With Haemophilia A With Inhibitors Treated With rFVIIIFc (Elocta®) for Immune Tolerance Induction
Brief Summary A chart review study of patients with haemophilia A with inhibitors treated with rFVIIIFc (Elocta®) for immune tolerance induction.
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
Study Design Observational Model: Cohort
Time Perspective: Other
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
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.
Condition Hemophilia A With Inhibitor
Intervention Other: rFVIIIFc
Drug according to prescription
Other Name: Elocta
Study Groups/Cohorts Hemophili A patients
Patients treated with rFVIIIFc for ITI
Intervention: Other: rFVIIIFc
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Active, not recruiting
Estimated Enrollment
 (submitted: October 29, 2020) 		45
Original Estimated Enrollment
 (submitted: May 13, 2019) 		50
Estimated Study Completion Date December 30, 2022
Estimated Primary Completion Date September 30, 2022   (Final data collection date for primary outcome measure)
Eligibility 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.
Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries France,   Germany,   Ireland,   Italy,   Kuwait,   Norway,   Saudi Arabia,   Switzerland
Removed Location Countries United Arab Emirates
 
Administrative Information
NCT Number NCT03951103
Other Study ID Numbers Sobi.Elocta-004
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: No
Responsible Party Swedish Orphan Biovitrum
Study Sponsor Swedish Orphan Biovitrum
Collaborators Kantar Health
Investigators
Study Director: Stefan Lethagen Swedish Orphan Biovitrum
PRS Account Swedish Orphan Biovitrum
Verification Date October 2020

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