Skip to main content
OpenTrials
Active, Not Recruiting

NCT Number: NCT03932201

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

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Notify Me

Key information

Conditions

Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Sex eligibility

All sexes

Study type

Observational

Primary location

Many locations, Multiple Locations, Belgium

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Damoctocog alfa pegol (Jivi, Bay94-9027)

Drug

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.

Primary outcomes

  1. Mean annualized number of reported total bleeds in patients with hemophilia A

    Time frame: Up to 36 months

  2. Median annualized number of reported total bleeds in patients with hemophilia A

    Time frame: Up to 36 months

Secondary outcomes

  1. Occurrence of AEs.

    Time frame: 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.

  2. Duration of AEs.

    Time frame: Up to 36 months

  3. Treatment of AEs.

    Time frame: Up to 36 months

  4. Severity of AEs.

    Time frame: Up to 36 months

  5. Outcome of AEs.

    Time frame: Up to 36 months

  6. Number of infusions and FVIII consumption to achieve hemostasis during surgery

    Time frame: Up to 36 months

  7. Change in joint scores (HJHS)

    Time frame: From baseline to 12 months, 24 months and 36 months

    HJHS:Hemophilia Joint Health Score

  8. Joint status evaluation by ultrasound (HEAD-US score), if available or part of standard clinical practice.

    Time frame: Up to 36 months

    HEAD-US:Hemophilia Early Arthropathy Detection with Ultrasound

  9. Change of number of affected joints by patient

    Time frame: From baseline to 12 months, 24 months and 36 months

  10. Annualized number of spontaneous, joint, and trauma bleeds

    Time frame: Up to 36 months

  11. 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.

    Time frame: Up to 36 months

    Bleeds includes total, spontaneous, joint, and trauma.

  12. Proportion of patients with 0 bleeds, and the difference in proportion comparing to previous prophylaxis treatment.

    Time frame: Up to 36 months

  13. AUC for previous FVIII products versus damoctocog alfa pegol.

    Time frame: Up to 36 months

    AUC:Area under the plasma concentration versus time curve

  14. Half-life [t½] for previous FVIII products versus damoctocog alfa pegol.

    Time frame: Up to 36 months

  15. FVIII trough for previous FVIII products versus damoctocog alfa pegol.

    Time frame: Up to 36 months

  16. FVIII peak levels for previous FVIII products versus damoctocog alfa pegol.

    Time frame: Up to 36 months

  17. In-vivo recovery for previous FVIII products versus damoctocog alfa pegol.

    Time frame: Up to 36 months

  18. Number of infusions to control for a bleed

    Time frame: Up to 36 months

  19. Changes of Hemo-SAT A score

    Time frame: 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).

  20. Changes of Hemo-QoL (A and SF) score

    Time frame: 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.

  21. Changes of WPAI score

    Time frame: 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.

Sponsors and collaborators

Lead sponsor

Bayer

Industry

Registry information

Official study title

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

Acronym: HEM-POWR

Important dates

Study start
2019
Primary completion
2026
Study completion
2027
First posted
Apr 30, 2019
Registry last updated
Jul 1, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.