do_not_disturb_altRecruitment Complete

Hemophilia A

Bayer Identifier:

20002

ClinicalTrials.gov Identifier:

NCT03932201

EudraCT Number:

Not Available

EU CT Number:

Not Available
Evaluating effectiveness and long term safety of Damoctocog alfa pegol in patients, who have been diagnosed with Hemophilia A

Trial purpose

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.

Key Participants Requirements

Sex

All

Age

NaN - N/A
  • - 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.
  • - 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.

Trial summary

Enrollment Goal
371
Trial Dates
October 2019 - March 2027
Phase
N/A
Could I Receive a placebo
No
Products
Jivi (Damoctocog, BAY94-9027)
Accepts Healthy Volunteer
No

Where to participate

StatusInstitutionLocation
Recruiting
Regents of University of MinnesotaMinneapolis, 55454, United States
Recruiting
Many locationsMany locations, Japan
Recruiting
Many locationsMany locations, Italy
Recruiting
Many locationsMany locations, Spain
Recruiting
Many locationsMany locations, Canada
Recruiting
Many locationsMany locations, Belgium
Recruiting
Many locationsMany locations, Netherlands
Recruiting
Many locationsMany locations, Sweden
Recruiting
Many locationsMany locations, Denmark
Recruiting
Many locationsMany locations, Greece
Recruiting
Many locationsMany locations, Taiwan
Recruiting
Many locationsMany locations, Slovenia
Recruiting
University California DavisDavis, 95616, United States
Recruiting
East Carolina University - Brody School of MedicineGreenville, 27834, United States
Recruiting
Tulane UniversityNew Orleans, 70112, United States
Recruiting
Tulane UniversityNew Orleans, 70112, United States
Recruiting
South Alabama Medical Science FoundationMobile, 36688, United States
Recruiting
Orthopaedic Hospital DBA Orthopaedic Institute for ChildrenLos Angelos, 90007, United States
Recruiting
The Center for Comprehensive Care and Diagnosis of Inherited Blood DisordersOrange, 92868, United States
Recruiting
University of Colorado Hemophilia and Thrombosis CenterAurora, 80045, United States
Recruiting
Many locationsMany locations, Germany
Recruiting
Many locationsMany locations, Switzerland
Recruiting
Banner MD Anderson Cancer CenterPhoenix, 85012, United States
Recruiting
Georgetown UniversityWashington, 20007, United States
Withdrawn
Many locationsMany locations, Austria
Recruiting
Many locationsMany locations, Brazil
Recruiting
Many locationsMany locations, Colombia
Withdrawn
Many locationsMany locations, Finland
Withdrawn
Many locationsMany locations, France
Not yet recruiting
Many locationsMany locations, Kuwait
Withdrawn
Many locationsMany locations, Luxembourg
Withdrawn
Many locationsMany locations, Mexico
Not yet recruiting
Many locationsMany locations, Norway
Withdrawn
Many locationsMany locations, Portugal
Withdrawn
Many locationsMany locations, Russian Federation
Not yet recruiting
Many locationsMany locations, Saudi Arabia
Not yet recruiting
Many locationsMany locations, United Arab Emirates
Withdrawn
Many locationsMany locations, China

Primary Outcome

  • Mean annualized number of reported total bleeds in patients with hemophilia A
    date_rangeTime Frame:
    Up to 36 months
  • Median annualized number of reported total bleeds in patients with hemophilia A
    date_rangeTime Frame:
    Up to 36 months

Secondary Outcome

  • 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.
    date_rangeTime Frame:
    Up to 36 months
  • Duration of AEs.
    date_rangeTime Frame:
    Up to 36 months
  • Treatment of AEs.
    date_rangeTime Frame:
    Up to 36 months
  • Severity of AEs.
    date_rangeTime Frame:
    Up to 36 months
  • Outcome of AEs.
    date_rangeTime Frame:
    Up to 36 months
  • Number of infusions and FVIII consumption to achieve hemostasis during surgery
    date_rangeTime Frame:
    Up to 36 months
  • Change in joint scores (HJHS)
    HJHS:Hemophilia Joint Health Score
    date_rangeTime Frame:
    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
    date_rangeTime Frame:
    Up to 36 months
  • Change of number of affected joints by patient
    date_rangeTime Frame:
    From baseline to 12 months, 24 months and 36 months
  • Annualized number of spontaneous, joint, and trauma bleeds
    date_rangeTime Frame:
    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.
    date_rangeTime Frame:
    Up to 36 months
  • Proportion of patients with 0 bleeds, and the difference in proportion comparing to previous prophylaxis treatment.
    date_rangeTime Frame:
    Up to 36 months
  • AUC for previous FVIII products versus damoctocog alfa pegol.
    AUC:Area under the plasma concentration versus time curve
    date_rangeTime Frame:
    Up to 36 months
  • Half-life [t½] for previous FVIII products versus damoctocog alfa pegol.
    date_rangeTime Frame:
    Up to 36 months
  • FVIII trough for previous FVIII products versus damoctocog alfa pegol.
    date_rangeTime Frame:
    Up to 36 months
  • FVIII peak levels for previous FVIII products versus damoctocog alfa pegol.
    date_rangeTime Frame:
    Up to 36 months
  • In-vivo recovery for previous FVIII products versus damoctocog alfa pegol.
    date_rangeTime Frame:
    Up to 36 months
  • Number of infusions to control for a bleed
    date_rangeTime Frame:
    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).
    date_rangeTime Frame:
    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.
    date_rangeTime Frame:
    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.
    date_rangeTime Frame:
    From baseline to 12 months, 24 months and 36 months

Trial design

Observational Study Evaluating Effectiveness and Safety of Real-World Treatment with Damoctocog alfa pegol in Previously Treated Patients with Hemophilia A
Trial Type
Observational
Intervention Type
Drug
Trial Purpose
N/A
Allocation
N/A
Blinding
N/A
Assignment
N/A
Trial Arms
N/A