Clinical trial NCT03248141: Understanding Hemophilia A and B Drug Dosage Administration Patterns
Official title: UNDERSTANDING HEMOPHILIA A AND B DRUG DOSAGE ADMINISTRATION PATTERNS
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Hemophilia A
Hemophilia B
Keywords: Hemophilia A Factor VIII
Hemophilia B Factor IX
Cross-sectional
Epidemiological
Clinical practice
United States
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2017-08-14
Enrollment: 11
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
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.

Brief summary: 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.

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.

Eligibility 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

Primary outcomes:
measure: Resource Utilization Pattern
timeFrame: From baseline up to end of study (6 months)
measure: Dosing Pattern
timeFrame: From baseline up to end of study (6 months)
Secondary outcomes:
Other outcomes:
Locations:
city: Gainesville
country: United States
facility: University of Florida College of Medicine
state: Florida
city: Las Vegas
country: United States
facility: Alliance for Childhood Diseases, dba Hemophilia Treatment Center of Nevada
state: Nevada
city: Fort Worth
country: United States
facility: Cook Children's Medical Center
state: Texas
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: