Evaluating Effectiveness and Long Term Safety of Damoctocog Alfa Pegol in Patients, Who Have Been Diagnosed With Hemophilia A

Hemophilia A Clinical Trial

— HEM-POWR  

Official title:

Observational Study Evaluating Effectiveness and Safety of Real-World Treatment With Damoctocog Alfa Pegol in Previously Treated Patients With Hemophilia A

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03932201
• Other study ID #: 20002
• Status: Active, not recruiting
• Start date: October 21, 2019
• Est. completion date: March 31, 2027

Study information

• Verified date: April 2024
• Source: Bayer
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 371
• Est. completion date: March 31, 2027
• Est. primary completion date: December 31, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Hemophilia A

Intervention

Drug:
• Damoctocog alfa pegol (Jivi, Bay94-9027)
Follow clinical practice. BAY94-9027 is a B-domain-deleted recombinant factor VIII (rFVIII) product site-specifically conjugated to a single (dual 30-kDa branched) 60-kDa polyethylene glycol (PEG) molecule. BAY 94-9027 is intended for prophylaxis and treatment of bleeds in patients with hemophilia A aged =12 years, with a narrow, predictable weekly dose that allows for the treatment regimen to be tailored to individual patient needs.

Locations

Country	Name	City	State
Belgium	Many locations	Multiple Locations
Brazil	Many locations	Multiple Locations
Canada	Many locations	Multiple Locations
Colombia	Many locations	Multiple Locations
Denmark	Many locations	Multiple Locations
Germany	Many locations	Multiple Locations
Greece	Many locations	Multiple Locations
Italy	Many locations	Multiple Locations
Japan	Many locations	Multiple Locations
Kuwait	Many locations	Multiple Locations
Netherlands	Many locations	Multiple Locations
Norway	Many locations	Multiple Locations
Saudi Arabia	Many locations	Multiple Locations
Slovenia	Many locations	Multiple Locations
Spain	Many locations	Multiple Locations
Sweden	Many locations	Multiple Locations
Switzerland	Many locations	Multiple Locations
Taiwan	Many locations	Multiple Locations
United Arab Emirates	Many locations	Multiple Locations
United States	University of Colorado Hemophilia and Thrombosis Center	Aurora	Colorado
United States	University California Davis	Davis	California
United States	East Carolina University - Brody School of Medicine	Greenville	North Carolina
United States	Orthopaedic Hospital DBA Orthopaedic Institute for Children	Los Angeles	California
United States	Regents of University of Minnesota	Minneapolis	Minnesota
United States	South Alabama Medical Science Foundation	Mobile	Alabama
United States	Tulane University	New Orleans	Louisiana
United States	The Center for Comprehensive Care and Diagnosis of Inherited Blood Disorders	Orange	California
United States	Banner MD Anderson Cancer Center	Phoenix	Arizona
United States	Georgetown University	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Bayer

Countries where clinical trial is conducted

United States,  Belgium,  Brazil,  Canada,  Colombia,  Denmark,  Germany,  Greece,  Italy,  Japan,  Kuwait,  Netherlands,  Norway,  Saudi Arabia,  Slovenia,  Spain,  Sweden,  Switzerland,  Taiwan,  United Arab Emirates, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Mean annualized number of reported total bleeds in patients with hemophilia A		Up to 36 months
Primary	Median annualized number of reported total bleeds in patients with hemophilia A		Up to 36 months
Secondary	Occurrence of AEs.	Adverse Events (AEs) includes: AEs of special interest, Adverse Event, Serious Adverse Event, Adverse reaction.; AEs of special interest includes: hypersensitivity reactions, loss of drug effect, renal impairment, neurocognitive disorders, inhibitor development.	Up to 36 months
Secondary	Duration of AEs.		Up to 36 months
Secondary	Treatment of AEs.		Up to 36 months
Secondary	Severity of AEs.		Up to 36 months
Secondary	Outcome of AEs.		Up to 36 months
Secondary	Number of infusions and FVIII consumption to achieve hemostasis during surgery		Up to 36 months
Secondary	Change in joint scores (HJHS)	HJHS:Hemophilia Joint Health Score	From baseline to 12 months, 24 months and 36 months
Secondary	Joint status evaluation by ultrasound (HEAD-US score), if available or part of standard clinical practice.	HEAD-US:Hemophilia Early Arthropathy Detection with Ultrasound	Up to 36 months
Secondary	Change of number of affected joints by patient		From baseline to 12 months, 24 months and 36 months
Secondary	Annualized number of spontaneous, joint, and trauma bleeds		Up to 36 months
Secondary	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.	Bleeds includes total, spontaneous, joint, and trauma.	Up to 36 months
Secondary	Proportion of patients with 0 bleeds, and the difference in proportion comparing to previous prophylaxis treatment.		Up to 36 months
Secondary	AUC for previous FVIII products versus damoctocog alfa pegol.	AUC:Area under the plasma concentration versus time curve	Up to 36 months
Secondary	Half-life [t½] for previous FVIII products versus damoctocog alfa pegol.		Up to 36 months
Secondary	FVIII trough for previous FVIII products versus damoctocog alfa pegol.		Up to 36 months
Secondary	FVIII peak levels for previous FVIII products versus damoctocog alfa pegol.		Up to 36 months
Secondary	In-vivo recovery for previous FVIII products versus damoctocog alfa pegol.		Up to 36 months
Secondary	Number of infusions to control for a bleed		Up to 36 months
Secondary	Changes of Hemo-SAT A score	Hemo-SAT A:Hemophilia Treatment Satisfaction Questionnaire for adults?The Hemo-SAT questionnaire version for adults (Hemo-SAT A) consists of 34 items pertaining to 6 dimensions (Ease & Convenience, Efficacy, Burden, Specialist/Nurses, Center/Hospital, General Satisfaction).	From baseline to 12 months, 24 months and 36 months
Secondary	Changes of Hemo-QoL (A and SF) score	Hemo-QoL-A:Hemophilia Quality of Life Measure for adults Hemo-QoL-SF:Hemophilia Quality of Life short form for children Hemo-QoL-A is a hemophiliaspecific quality of life questionnaire for adults aged 18 years and above. The questionnaire has 41 items covering 6 domains: Physical Functioning, Role Functioning, Worry, Consequences of Bleeding, Emotional Impact, and Treatment Concerns. For patients younger than 18 years, the Hemo-QoL-SF Questionnaire for children and adolescents (12 to 17 years) is used. The Hemo-QoL-SF contains 35 questions covering 9 domains: Physical Health, View of Yourself, Family, Friends, Others, Sports, Dealing, and Treatment.	From baseline to 12 months, 24 months and 36 months
Secondary	Changes of WPAI score	WPAI:Work Productivity and Activity Impairment Scale? Scores are expressed as percentages of impairment/ productivity loss, with higher scores indicating greater impairment.	From baseline to 12 months, 24 months and 36 months