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CompletedNCT03405337PARkERUpdated Sep 27, 2018

Evaluation of Patient and Physician Reported Reasons for Switching Factor VIII Replacement Therapies

An observational study in Hemophilia A, sponsored by Bayer. Completed at 1 site in United States. Open to participants aged 12 Years and older. Per ClinicalTrials.gov, last updated 2018-09-27.

Sponsored by Bayer · Observational

Study type
Observational
Model
Cohort
Time perspective
Other
Enrollment
160
Ages
12 Years and older
Sex
All
01

Study summary

This US study will assess hemophilia A patient characteristics, health history and reasons for switching or not switching from both patient/caregiver and physician perspectives. For this purpose, this research study will include hemophilia A: 1) patients who have switched from conventional therapy to new FVIII products with an improved PK profile. 2) patients who remain on conventional therapy (who have never switched) but have considered switching, including those patients who switched from conventional therapy to new FVIII products with improved pharmacokinetics and then subsequently "switched back" to conventional replacement therapy. In doing so, real world evidence will be obtained from both patient and physician perspectives offering key insights for effective therapeutic management of patients with hemophilia A and to more fully understand what drives patient switching from a patient perspective and a physician perspective.

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Conditions studied

  • Hemophilia A

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Who can participate

Ages eligible
12 Years and older
Sexes eligible
All
Accepts healthy volunteers
No
Sampling method
Non-probability sample

Study population

Study Population for the Patient/Caregiver Study:

Hemophilia A patients/caregivers will be recruited via opt-in patient panels and/or research databases.

Patients with hemophilia A (≥ 18 years of age) or caregivers of children with hemophilia A (≥12-\<18 years of age)

Study Population for the Physician Chart Study:

The study population for the physician chart review study will be patients, who initiated FVIII products with improved half-life for the treatment of hemophilia A during the eligibility period. In order to obtain a broad patient population that is representative of real world prescribing; all eligible patients will be included in the study irrespective of the FVIII product with improved half-life received.

Eligibility criteria

Inclusion Criteria:

Inclusion criteria for patients/caregivers in the Patient/Caregiver Study:

  • Patients with hemophilia A (≥ 18 years of age) or caregivers of children with hemophilia A (≥12-\<18 years of age)
  • Group 1: Hemophilia A patients/caregivers who have switched to FVIII products with improved half-life for the treatment of hemophilia A during the eligibility period. These patients can also include those who have switched back from FVIII products with improved half-life to conventional FVIII replacement therapy within the Data Collection Period
  • Group 2: Hemophilia A patients/caregivers who are considering switching to FVIII products with improved half-life within 12 months of the Start of the Documentation period and have been prescribed prophylaxis regimen of at least 2x/week
  • Able to understand, read, write and speak English
  • Provide electronic informed consent
  • Able to access the Internet for at least 20 minutes per day during the Data Collection Period

Inclusion criteria for physicians:

  • At least 60% of time spent in direct patient care
  • Board-certified or eligible with a Specialty in Hematology or Hematology-Oncology
  • Physicians with a specialty in Hematology-Oncology must have at least 10% of their practice dedicated to treatment of hemophilia
  • A minimum of 2 years' experience treating hemophilia A patients

Inclusion criteria for patients in the physician chart study:

  • Hemophilia A patients age 12 year and over
  • Prior treatment with one of the following FVIII replacement products: Adynovate, Afstyla, Eloctate, or Kovaltry
  • Patients that have 12 months of medical chart data available; 6 months on conventional therapy and 6 months after switching to FVIII products with improved half-life.

Exclusion Criteria:

Exclusion criteria for patients/caregivers in the Patient/Caregiver Study:

  • Hemophilia A patient initiated FVIII products with improved half-life for the treatment at time of diagnosis with hemophilia A.

Exclusion criteria for physicians:

  • Unwilling to comply with the study protocol
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Study design

Observational model
Cohort
Time perspective
Other
Enrollment
160 participants (actual)
Patient registry
No

Groups and cohorts

  • FVIII products (prospective)

    Qualitative patient/caregiver study: Hemophilia A patients/caregivers (N=30) having initiated a FVIII products with improved half-life

    Drug: FVIII products · Drug: Conventional FVIII replacement therapies

  • Conventional FVIII replacement therapies

    Qualitative patient/caregiver study: Hemophilia A patients/caregivers (N=30) receiving "conventional" FVIII replacement therapy for at least 6 months who are considering switching to a FVIII product with improved half-life within the next 1 year

    Drug: Conventional FVIII replacement therapies

  • FVIII products (retrospective)

    Quantitative physician interview/ chart review study: Hemophilia A patients (N=100) who have switched from "conventional" FVIII replacement therapy to FVIII products with improved half-life.

    Drug: FVIII products · Drug: Conventional FVIII replacement therapies

Interventions

  • DrugFVIII products

    Adynovate, Eloctate, Afstyla, Kovaltry

  • DrugConventional FVIII replacement therapies

    Advate, Kogenate FS, Helixate, Novoeight, Nuwiq, Recombinate, Xyntha

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What researchers measure

Primary outcomes

  1. The reasons of patients/caregivers "switch" from conventional FVIII replacement therapy to FVIII products with improved half-life

    These patients/caregivers will participate in an asynchronous online discussion forum consisting of predetermined open ended and close ended questions for a series of 6 consecutive days completing approximately 20 minutes' worth of questions per day.

    Time frame: Up to 6 days

  2. The obstacles of switching among hemophilia A patients who did not switch from conventional therapy to FVIII products with improved half-life but who are open to switching

    These patients/caregivers will participate in an asynchronous online discussion forum consisting of predetermined open ended and close ended questions for a series of 6 consecutive days completing approximately 20 minutes' worth of questions per day.

    Time frame: Up to 6 days

  3. The clinical characteristics of patients who switch from conventional FVIII replacement therapy to FVIII products with improved half-life

    A retrospective patient medical chart review

    Time frame: Up to 4.5 months

  4. The changes of treatment characteristics from 6 months prior to switching compared to 6 months after switching from conventional FVIII replacement therapy to FVIII products with improved half-life

    A retrospective patient medical chart review

    Time frame: Up to 4.5 months

  5. The changes of bleeding related outcomes from 6 months prior to switching compared to 6 months after switching from conventional FVIII replacement therapy to FVIII products with improved half-life

    A retrospective patient medical chart review

    Time frame: Up to 4.5 months

  6. The reasons for switching from conventional FVIII replacement therapy to FVIII products with improved half-life, from the physician perspective

    A retrospective patient medical chart review

    Time frame: Up to 4.5 months

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Study locations

1 site
  • Many Locations
    Whippany, New Jersey 07981, United States
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Registry details

Key details

Study ID
NCT03405337
Lead sponsor
Bayer
Responsible party
Sponsor
First posted
Jan 23, 2018
Start date
Feb 14, 2018
Primary completion
Jun 13, 2018
Completion
Jun 13, 2018
Last update
Sep 27, 2018

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
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