Clinical trial NCT03932201: Evaluating Effectiveness and Long Term Safety of Damoctocog Alfa Pegol in Patients, Who Have Been Diagnosed With Hemophilia A
Official title: Observational Study Evaluating Effectiveness and Safety of Real-World Treatment With Damoctocog Alfa Pegol in Previously Treated Patients With Hemophilia A
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Hemophilia A
Lead sponsor: Bayer
Sponsor class: INDUSTRY
First posted: 2019-04-30
Enrollment: 371
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Previously treated patients with hemophilia A receiving damoctocog alfa pegol with any kind of treatment modality (on-demand, prop hylaxis, or intermittent prophylaxis).

Brief summary: The aim of the HEM-POWR study is to understand better how Damoctocog alfa pegol (Jivi) is used to treat people with Hemophilia A in day-to-day life, how well the treatment is tolerated and how satisfied patients and physicians are with the treatment.


Eligibility criteria: Inclusion Criteria:

* Diagnosis of hemophilia A.
* Patients previously treated for Hemophilia A.
* Patients without previous history of inhibitors or patients with previous history of inhibitors on standard prophylaxis therapy for at least 1 year prior to study entry.
* No current evidence of FVIII inhibitor or clinical suspicion of FVIII inhibitor.
* Initiation of or currently on damoctocog alfa pegol with any kind of treatment modality (on-demand, prophylaxis, or intermittent prophylaxis).
* Signed informed consent/assent.

Exclusion Criteria:

* Concurrent participation in an investigational program with interventions outside of routine clinical practice.
* Diagnosis of any other bleeding/coagulation disorder other than hemophilia A.
* Contra-indications according to the local marketing authorization.
* Patient on immune tolerance induction (ITI) treatment at the time of enrollment.

Primary outcomes:
measure: Mean annualized number of reported total bleeds in patients with hemophilia A
timeFrame: Up to 36 months
measure: Median annualized number of reported total bleeds in patients with hemophilia A
timeFrame: Up to 36 months
Secondary outcomes:
measure: Occurrence of AEs.
timeFrame: Up to 36 months
measure: Duration of AEs.
timeFrame: Up to 36 months
measure: Treatment of AEs.
timeFrame: Up to 36 months
measure: Severity of AEs.
timeFrame: Up to 36 months
measure: Outcome of AEs.
timeFrame: Up to 36 months
measure: Number of infusions and FVIII consumption to achieve hemostasis during surgery
timeFrame: Up to 36 months
measure: Change in joint scores (HJHS)
timeFrame: From baseline to 12 months, 24 months and 36 months
measure: Joint status evaluation by ultrasound (HEAD-US score), if available or part of standard clinical practice.
timeFrame: Up to 36 months
measure: Change of number of affected joints by patient
timeFrame: From baseline to 12 months, 24 months and 36 months
measure: Annualized number of spontaneous, joint, and trauma bleeds
timeFrame: Up to 36 months
measure: Number of reported bleeds during the study compared with number of reported bleeds for previous FVIII products in the 12 months prior to enrollment into the study.
timeFrame: Up to 36 months
measure: Proportion of patients with 0 bleeds, and the difference in proportion comparing to previous prophylaxis treatment.
timeFrame: Up to 36 months
measure: AUC for previous FVIII products versus damoctocog alfa pegol.
timeFrame: Up to 36 months
measure: Half-life [t½] for previous FVIII products versus damoctocog alfa pegol.
timeFrame: Up to 36 months
measure: FVIII trough for previous FVIII products versus damoctocog alfa pegol.
timeFrame: Up to 36 months
measure: FVIII peak levels for previous FVIII products versus damoctocog alfa pegol.
timeFrame: Up to 36 months
measure: In-vivo recovery for previous FVIII products versus damoctocog alfa pegol.
timeFrame: Up to 36 months
measure: Number of infusions to control for a bleed
timeFrame: Up to 36 months
measure: Changes of Hemo-SAT A score
timeFrame: From baseline to 12 months, 24 months and 36 months
measure: Changes of Hemo-QoL (A and SF) score
timeFrame: From baseline to 12 months, 24 months and 36 months
measure: Changes of WPAI score
timeFrame: From baseline to 12 months, 24 months and 36 months
Other outcomes:
Locations:
city: Mobile
country: United States
facility: South Alabama Medical Science Foundation
state: Alabama
city: Phoenix
country: United States
facility: Banner MD Anderson Cancer Center
state: Arizona
city: Davis
country: United States
facility: University California Davis
state: California
city: Los Angeles
country: United States
facility: Orthopaedic Hospital DBA Orthopaedic Institute for Children
state: California
city: Orange
country: United States
facility: The Center for Comprehensive Care and Diagnosis of Inherited Blood Disorders
state: California
city: Aurora
country: United States
facility: University of Colorado Hemophilia and Thrombosis Center
state: Colorado
city: Washington
country: United States
facility: Georgetown University
state: District of Columbia
city: New Orleans
country: United States
facility: Tulane University
state: Louisiana
city: Minneapolis
country: United States
facility: Regents of University of Minnesota
state: Minnesota
city: Greenville
country: United States
facility: East Carolina University - Brody School of Medicine
state: North Carolina
city: Multiple Locations
country: Belgium
facility: Many locations
city: Multiple Locations
country: Brazil
facility: Many locations
city: Multiple Locations
country: Canada
facility: Many locations
city: Multiple Locations
country: Colombia
facility: Many locations
city: Multiple Locations
country: Denmark
facility: Many locations
city: Multiple Locations
country: Germany
facility: Many locations
city: Multiple Locations
country: Greece
facility: Many locations
city: Multiple Locations
country: Italy
facility: Many locations
city: Multiple Locations
country: Japan
facility: Many locations
city: Multiple Locations
country: Kuwait
facility: Many locations
city: Multiple Locations
country: Netherlands
facility: Many locations
city: Multiple Locations
country: Norway
facility: Many locations
city: Multiple Locations
country: Saudi Arabia
facility: Many locations
city: Multiple Locations
country: Slovenia
facility: Many locations
city: Multiple Locations
country: Spain
facility: Many locations
city: Multiple Locations
country: Sweden
facility: Many locations
city: Multiple Locations
country: Switzerland
facility: Many locations
city: Multiple Locations
country: Taiwan
facility: Many locations
city: Multiple Locations
country: United Arab Emirates
facility: Many locations
Study officials:
References:
citation: Sanabria M, Alvarez Roman MT, Castaman G, Janbain M, Matsushita T, Meijer K, Oldenburg J, Friedl S, Reding MT. Design of the HEM-POWR study: a prospective, observational study of real-world treatment with damoctocog alfa pegol in patients with haemophilia A. BMJ Open. 2021 Sep 2;11(9):e044997. doi: 10.1136/bmjopen-2020-044997.