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

NCT03405337 | Completed

• 1 other identifier: 19529
• Study Type: observational
• Target: 160
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (6)

• 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.

  Up to 6 days

• 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.

  Up to 6 days

• 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

  Up to 4.5 months

• 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

  Up to 4.5 months

• 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

  Up to 4.5 months

• 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

  Up to 4.5 months

Study Arms (3)

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

FVIII products DRUG

Adynovate, Eloctate, Afstyla, Kovaltry

FVIII products (prospective); FVIII products (retrospective)

Conventional FVIII replacement therapies DRUG

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

Conventional FVIII replacement therapies; FVIII products (prospective); FVIII products (retrospective)

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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
• 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
• 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.

You may not qualify if:

• \- Hemophilia A patient initiated FVIII products with improved half-life for the treatment at time of diagnosis with hemophilia A.
• \- Unwilling to comply with the study protocol

Sponsors & Collaborators

• Bayer: lead

Study Sites (1)

Many Locations

Whippany, New Jersey, 07981, United States

Location

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 15, 2018
• First Posted: January 23, 2018
• Study Start: February 14, 2018
• Primary Completion: June 13, 2018
• Study Completion: June 13, 2018
• Last Updated: September 27, 2018

Record last verified: 2018-09

Locations

US (1)