Understanding Hemophilia A and B Drug Dosage Administration Patterns

Pfizer

Status: Terminated

Conditions: Hemophilia B; Hemophilia A

Treatments: Drug: Hemophilia B standard half-life; Drug: Hemophilia B extended half-life; Drug: Hemophilia A extended half-life; Drug: Hemophilia A standard half-life

Study type: Observational

Funder types: Industry

Identifiers

NCT03248141

HEMOBAFS (Other Identifier)

B1821056

Details and patient eligibility

About

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.

Full 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.

Enrollment: 11 patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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

Trial design

11 participants in 2 patient groups

Hemophilia B

Description:

real world administration patterns and resource utilization implications

Treatment:

Drug: Hemophilia B standard half-life

Drug: Hemophilia B extended half-life

Hemophilia A

Description:

real world administration patterns and resource utilization implications

Treatment:

Drug: Hemophilia A extended half-life

Drug: Hemophilia A standard half-life