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

Active, not recruiting

• Conditions: Hemophilia A
• Interventions: Drug: Damoctocog alfa pegol (Jivi, Bay94-9027)

• Registration Number: NCT03932201
• Lead Sponsor: Bayer

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2019-04-18
• Study First Submitted QC: 2019-04-26
• Study First Posted: 2019-04-30
• Study Start: 2019-10-21
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-07
• Last Update Posted: 2025-05-08
• Primary Completion: 2026-12-31
• Study Completion: 2027-03-31

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 371

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Previously treated patient (PTPs) with hemophilia A	Damoctocog alfa pegol (Jivi, Bay94-9027)	Previously treated patients with hemophilia A receiving damoctocog alfa pegol with any kind of treatment modality (on-demand, prophylaxis, or intermittent prophylaxis)。

Primary Outcome Measures

Name	Time	Method
Median annualized number of reported total bleeds in patients with hemophilia A	Up to 36 months
Mean annualized number of reported total bleeds in patients with hemophilia A	Up to 36 months

Secondary Outcome Measures

Name	Time	Method
Joint status evaluation by ultrasound (HEAD-US score), if available or part of standard clinical practice.	Up to 36 months	HEAD-US:Hemophilia Early Arthropathy Detection with Ultrasound
Change of number of affected joints by patient	From baseline to 12 months, 24 months and 36 months
Annualized number of spontaneous, joint, and trauma bleeds	Up to 36 months
Half-life [t½] for previous FVIII products versus damoctocog alfa pegol.	Up to 36 months
Outcome of AEs.	Up to 36 months
Number of infusions and FVIII consumption to achieve hemostasis during surgery	Up to 36 months
Changes of Hemo-QoL (A and SF) score	From baseline to 12 months, 24 months and 36 months	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.
Treatment of AEs.	Up to 36 months
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.	Up to 36 months	Bleeds includes total, spontaneous, joint, and trauma.
Proportion of patients with 0 bleeds, and the difference in proportion comparing to previous prophylaxis treatment.	Up to 36 months
FVIII peak levels for previous FVIII products versus damoctocog alfa pegol.	Up to 36 months
In-vivo recovery for previous FVIII products versus damoctocog alfa pegol.	Up to 36 months
Changes of Hemo-SAT A score	From baseline to 12 months, 24 months and 36 months	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).
Occurrence of AEs.	Up to 36 months	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.
Severity of AEs.	Up to 36 months
Change in joint scores (HJHS)	From baseline to 12 months, 24 months and 36 months	HJHS:Hemophilia Joint Health Score
AUC for previous FVIII products versus damoctocog alfa pegol.	Up to 36 months	AUC:Area under the plasma concentration versus time curve
FVIII trough for previous FVIII products versus damoctocog alfa pegol.	Up to 36 months
Number of infusions to control for a bleed	Up to 36 months
Changes of WPAI score	From baseline to 12 months, 24 months and 36 months	WPAI:Work Productivity and Activity Impairment Scale。 Scores are expressed as percentages of impairment/ productivity loss, with higher scores indicating greater impairment.
Duration of AEs.	Up to 36 months

Trial Locations

Locations (11)

The Center for Comprehensive Care and Diagnosis of Inherited Blood Disorders; 🇺🇸 Orange, California, United States

South Alabama Medical Science Foundation; 🇺🇸 Mobile, Alabama, United States

Georgetown University; 🇺🇸 Washington, District of Columbia, United States

Many locations; 🇦🇪 Multiple Locations, United Arab Emirates

University California Davis; 🇺🇸 Davis, California, United States

Banner MD Anderson Cancer Center; 🇺🇸 Phoenix, Arizona, United States

Orthopaedic Hospital DBA Orthopaedic Institute for Children; 🇺🇸 Los Angeles, California, United States

University of Colorado Hemophilia and Thrombosis Center; 🇺🇸 Aurora, Colorado, United States

Tulane University; 🇺🇸 New Orleans, Louisiana, United States

Regents of University of Minnesota; 🇺🇸 Minneapolis, Minnesota, United States

East Carolina University - Brody School of Medicine; 🇺🇸 Greenville, North Carolina, United States