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

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

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Hemophilia A
• Intervention / Treatment: Drug: Damoctocog alfa pegol (Jivi, Bay94-9027)

• Study Type: Observational
• Enrollment (Actual): 371

Contacts and Locations

Study Locations

• Belgium
  • Multiple Locations, Belgium
    • Many Locations
• Brazil
  • Multiple Locations, Brazil
    • Many Locations
• Canada
  • Multiple Locations, Canada
    • Many Locations
• Colombia
  • Multiple Locations, Colombia
    • Many Locations
• Denmark
  • Multiple Locations, Denmark
    • Many Locations
• Germany
  • Multiple Locations, Germany
    • Many Locations
• Greece
  • Multiple Locations, Greece
    • Many Locations
• Italy
  • Multiple Locations, Italy
    • Many Locations
• Japan
  • Multiple Locations, Japan
    • Many Locations
• Kuwait
  • Multiple Locations, Kuwait
    • Many Locations
• Netherlands
  • Multiple Locations, Netherlands
    • Many Locations
• Norway
  • Multiple Locations, Norway
    • Many Locations
• Saudi Arabia
  • Multiple Locations, Saudi Arabia
    • Many Locations
• Slovenia
  • Multiple Locations, Slovenia
    • Many Locations
• Spain
  • Multiple Locations, Spain
    • Many Locations
• Sweden
  • Multiple Locations, Sweden
    • Many Locations
• Switzerland
  • Multiple Locations, Switzerland
    • Many Locations
• Taiwan
  • Multiple Locations, Taiwan
    • Many Locations
• United Arab Emirates
  • Multiple Locations, United Arab Emirates
    • Many Locations
• United States
  • Alabama
    • Mobile, Alabama, United States, 36688
      • South Alabama Medical Science Foundation
  • Arizona
    • Phoenix, Arizona, United States, 85012
      • Banner MD Anderson Cancer Center
  • California
    • Davis, California, United States, 95616
      • University California Davis
    • Los Angeles, California, United States, 90007
      • Orthopaedic Hospital DBA Orthopaedic Institute for Children
    • Orange, California, United States, 92868
      • The Center for Comprehensive Care and Diagnosis of Inherited Blood Disorders
  • Colorado
    • Aurora, Colorado, United States, 80045
      • University of Colorado Hemophilia and Thrombosis Center
  • District of Columbia
    • Washington D.C., District of Columbia, United States, 20007
      • Georgetown University
  • Louisiana
    • New Orleans, Louisiana, United States, 70112
      • Tulane University
  • Minnesota
    • Minneapolis, Minnesota, United States, 55454
      • Regents of University of Minnesota
  • North Carolina
    • Greenville, North Carolina, United States, 27834
      • East Carolina University - Brody School of Medicine

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• 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).

Description

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 Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Intervention / Treatment

Previously treated patient (PTPs) with hemophilia A; Previously treated patients with hemophilia A receiving damoctocog alfa pegol with any kind of treatment modality (on-demand, prophylaxis, or intermittent prophylaxis)。

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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Mean annualized number of reported total bleeds in patients with hemophilia A	Up to 36 months
Median annualized number of reported total bleeds in patients with hemophilia A	Up to 36 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
Duration of AEs.		Up to 36 months
Treatment of AEs.		Up to 36 months
Severity of AEs.		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
Change in joint scores (HJHS)	HJHS:Hemophilia Joint Health Score	From baseline to 12 months, 24 months and 36 months
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
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
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
Proportion of patients with 0 bleeds, and the difference in proportion comparing to previous prophylaxis treatment.		Up to 36 months
AUC for previous FVIII products versus damoctocog alfa pegol.	AUC:Area under the plasma concentration versus time curve	Up to 36 months
Half-life [t½] for previous FVIII products versus damoctocog alfa pegol.		Up to 36 months
FVIII trough for previous FVIII products versus damoctocog alfa pegol.		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
Number of infusions to control for a bleed		Up to 36 months
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
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
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

Collaborators and Investigators

• Sponsor: Bayer

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start (Actual): October 21, 2019
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): March 31, 2027

Study Registration Dates

• First Submitted: April 18, 2019
• First Submitted That Met QC Criteria: April 26, 2019
• First Posted (Actual): April 30, 2019

Study Record Updates

• Last Update Posted (Actual): April 9, 2026
• Last Update Submitted That Met QC Criteria: April 8, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hemic and Lymphatic Diseases; Hemophilia A; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors; Blood Proteins; Blood Coagulation Factors; Protein Precursors; Factor VIII
• Other Study ID Numbers: 20002

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

IPD Plan Description

Availability of this study's data will be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access.

As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014.

Interested researchers can use www.clinicalstudydatarequest.com to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the Study sponsors section of the portal.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No