Understanding Hemophilia A and B Drug Dosage Administration Patterns

UNDERSTANDING HEMOPHILIA A AND B DRUG DOSAGE ADMINISTRATION PATTERNS

Study Design

A prospective observational, cross-sectional epidemiological study in U.S. site-based clinical practice settings.

30 sites will enroll approximately 300 patients

Participating patients - or their caregiver in the case of patients under the age of 18 - will be consented to participate.

Physicians complete a retrospective chart review on each enrolled patient.

Patients will complete a one-time study questionnaire.

Study Overview

• Status: Terminated
• Conditions: Hemophilia A; Hemophilia B
• Intervention / Treatment: Drug: Hemophilia B standard half-life; Drug: Hemophilia B extended half-life; Drug: Hemophilia A standard half-life; Drug: Hemophilia A extended half-life

Detailed Description

Study recruitment was stopped due to difficulty in enrolling the targeted number of patients on March 21, 2018. Subjects currently enrolled into the study have completed the study as per protocol. There were no safety concerns involved in the decision to stop enrollment.

• Study Type: Observational
• Enrollment (Actual): 11

Contacts and Locations

Study Locations

• United States
  • Florida
    • Gainesville, Florida, United States, 32610
      • University of Florida College of Medicine
  • Nevada
    • Las Vegas, Nevada, United States, 89109
      • Alliance for Childhood Diseases, dba Hemophilia Treatment Center of Nevada
  • Texas
    • Fort Worth, Texas, United States, 76104
      • Cook Children's Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: ADULT; OLDER_ADULT; CHILD
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Patients diagnosed with either hemophilia A or B who present for a routine Clinical visit will be asked to participate in the study by the treating physician at participating hemophilia treatment centers in the United States of America.

Description

Physician/Clinician Participants:

• Must be a healthcare provider
• Currently manages at least 10 hemophilia A and/or B patients

Patient Participants:

• Willing and able to provide informed consent
• Diagnosed with hemophilia A or B
• Current disease severity is either moderately severe or severe with a clotting factor level of ≤5%
• If suffering from hemophilia A, must be currently taking moroctocog alfa (or another standard half-life treatment), rurioctocog alfa or efraloctocog alfa for at least six months.

(If currently taking rurioctocog alfa or efraloctocog alfa, must have been switched from a standard half-life treatment and had been on that prior treatment for at least six months).

• If suffering from hemophilia B, must be currently taking nonacog alfa or eftrenonacog alfa for at least six months (If currently taking eftrenonacog alfa, must have switched from nonacog alfa and had been on that prior treatment for at least six months).
• Infuse at least 3 times per month

Exclusion criteria:

• Female with hemophilia A or B
• Mild Haemophilia A or B

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Hemophilia B; real world administration patterns and resource utilization implications	Drug: Hemophilia B standard half-life; Benefix; Drug: Hemophilia B extended half-life; Alprolix
Hemophilia A; real world administration patterns and resource utilization implications	Drug: Hemophilia A standard half-life; Xyntha and other standard half-life agents; Drug: Hemophilia A extended half-life; Eloctate and Adynovate

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Resource Utilization Pattern	From baseline up to end of study (6 months)
Dosing Pattern	From baseline up to end of study (6 months)

Collaborators and Investigators

• Sponsor: Pfizer

Publications and helpful links

Helpful Links

• To obtain contact information for a study center near you, click here.

Study record dates

Study Major Dates

• Study Start (ACTUAL): September 1, 2017
• Primary Completion (ACTUAL): March 14, 2018
• Study Completion (ACTUAL): March 14, 2018

Study Registration Dates

• First Submitted: May 31, 2017
• First Submitted That Met QC Criteria: August 9, 2017
• First Posted (ACTUAL): August 14, 2017

Study Record Updates

• Last Update Posted (ACTUAL): May 20, 2019
• Last Update Submitted That Met QC Criteria: February 22, 2019
• Last Verified: February 1, 2019

More Information

Terms related to this study

• Keywords: Epidemiological; Cross-sectional; Clinical practice; United States; Hemophilia A Factor VIII; Hemophilia B Factor IX
• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Blood Coagulation Disorders; Hemophilia A; Hemophilia B; Coagulants; Factor VIII
• Other Study ID Numbers: B1821056; HEMOBAFS (OTHER: Alias Study Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No