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

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

Study Description
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.

Condition or disease Intervention/treatment
Hemophilia A Drug: Damoctocog alfa pegol (Jivi, Bay94-9027)

Study Design
Layout table for study information
Study Type : Observational
Estimated Enrollment : 200 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Observational Study Evaluating Effectiveness and Safety of Real-World Treatment With Damoctocog Alfa Pegol in Previously Treated Patients With Hemophilia A
Actual Study Start Date : October 21, 2019
Estimated Primary Completion Date : June 30, 2025
Estimated Study Completion Date : September 30, 2025
Arms 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.
Outcome Measures
Primary Outcome Measures :
  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 Outcome Measures :
  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.


Eligibility Criteria
Layout table for eligibility information
Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
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).
Criteria

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.
Contacts and Locations

Contacts
Layout table for location contacts
Contact: Bayer Clinical Trials Contact (+)1-888-84 22937 clinical-trials-contact@bayer.com
Contact: For trial location information (Phone Menu Options '3' or '4') (+)1-888-84 22937

Locations
Show Show 37 study locations
Sponsors and Collaborators
Bayer
Tracking Information
First Submitted Date April 18, 2019
First Posted Date April 30, 2019
Last Update Posted Date May 18, 2021
Actual Study Start Date October 21, 2019
Estimated Primary Completion Date June 30, 2025   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: May 14, 2021)
  • Mean annualized number of reported total bleeds in patients with hemophilia A [ Time Frame: Up to 36 months ]
  • Median annualized number of reported total bleeds in patients with hemophilia A [ Time Frame: Up to 36 months ]
Original Primary Outcome Measures
 (submitted: April 26, 2019)
  • Mean annualized number of reported total bleeds in patients with hemophilia A [ Time Frame: At 12 month ]
  • Mean annualized number of reported total bleeds in patients with hemophilia A [ Time Frame: At 24 month ]
  • Mean annualized number of reported total bleeds in patients with hemophilia A [ Time Frame: At 36 month ]
  • Median annualized number of reported total bleeds in patients with hemophilia A [ Time Frame: At 12 month ]
  • Median annualized number of reported total bleeds in patients with hemophilia A [ Time Frame: At 24 month ]
  • Median annualized number of reported total bleeds in patients with hemophilia A [ Time Frame: At 36 month ]
Change History
Current Secondary Outcome Measures
 (submitted: May 14, 2021)
  • 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.
  • Duration of AEs. [ Time Frame: Up to 36 months ]
  • Treatment of AEs. [ Time Frame: Up to 36 months ]
  • Severity of AEs. [ Time Frame: Up to 36 months ]
  • Outcome of AEs. [ Time Frame: Up to 36 months ]
  • Number of infusions and FVIII consumption to achieve hemostasis during surgery [ Time Frame: Up to 36 months ]
  • Change in joint scores (HJHS) [ Time Frame: From baseline to 12 months, 24 months and 36 months ]
    HJHS:Hemophilia Joint Health Score
  • 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
  • Change of number of affected joints by patient [ Time Frame: From baseline to 12 months, 24 months and 36 months ]
  • Annualized number of spontaneous, joint, and trauma bleeds [ Time 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. [ Time Frame: 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. [ Time Frame: Up to 36 months ]
  • AUC for previous FVIII products versus damoctocog alfa pegol. [ Time Frame: Up to 36 months ]
    AUC:Area under the plasma concentration versus time curve
  • Half-life [t½] for previous FVIII products versus damoctocog alfa pegol. [ Time Frame: Up to 36 months ]
  • FVIII trough for previous FVIII products versus damoctocog alfa pegol. [ Time Frame: Up to 36 months ]
  • FVIII peak levels for previous FVIII products versus damoctocog alfa pegol. [ Time Frame: Up to 36 months ]
  • In-vivo recovery for previous FVIII products versus damoctocog alfa pegol. [ Time Frame: Up to 36 months ]
  • Number of infusions to control for a bleed [ Time Frame: Up to 36 months ]
  • 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).
  • 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.
  • 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.
Original Secondary Outcome Measures
 (submitted: April 26, 2019)
  • 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.
  • Duration of AEs. [ Time Frame: Up to 36 months ]
  • Treatment of AEs. [ Time Frame: Up to 36 months ]
  • Severity of AEs. [ Time Frame: Up to 36 months ]
  • Outcome of AEs. [ Time Frame: Up to 36 months ]
  • Number of infusions and FVIII consumption to achieve hemostasis during surgery [ Time Frame: Up to 36 months ]
  • Change in joint scores (HJHS) [ Time Frame: From baseline to 12 months, 24 months and 36 months ]
    HJHS:Hemophilia Joint Health Score
  • 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
  • Change of number of affected joints by patient [ Time Frame: From baseline to 12 months, 24 months and 36 months ]
    Target joint is defined as a joint in which ≥3 spontaneous bleeds have occurred within a consecutive 6-month period
  • Annualized number of spontaneous, joint, and trauma bleeds. [ Time Frame: At 12 month, 24 month and 36 month ]
  • 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.
  • Proportion of patients with 0 bleeds, and the difference in proportion comparing to previous prophylaxis treatment. [ Time Frame: Up to 36 months ]
  • Cmax for previous FVIII products versus damoctocog alfa pegol. [ Time Frame: Up to 36 months ]
    Cmax: Maximum plasma Concentration
  • AUC for previous FVIII products versus damoctocog alfa pegol. [ Time Frame: Up to 36 months ]
    AUC:Area under the plasma concentration versus time curve
  • Half-life [t½] for previous FVIII products versus damoctocog alfa pegol. [ Time Frame: Up to 36 months ]
  • FVIII trough for previous FVIII products versus damoctocog alfa pegol. [ Time Frame: Up to 36 months ]
  • FVIII peak levels for previous FVIII products versus damoctocog alfa pegol. [ Time Frame: Up to 36 months ]
  • In-vivo recovery for previous FVIII products versus damoctocog alfa pegol. [ Time Frame: Up to 36 months ]
  • Date of bleeding event of treatment of break-through bleeds. [ Time Frame: Up to 36 months ]
  • Reason for infusion and location of bleed of treatment of break-through bleeds. [ Time Frame: Up to 36 months ]
  • Date of treatment of break-through bleeds. [ Time Frame: Up to 36 months ]
  • Number of infusions and dose per infusion for each bleeding event of treatment of break-through bleeds. [ Time Frame: Up to 36 months ]
  • 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).
  • 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.
  • 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.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Evaluating Effectiveness and Long Term Safety of Damoctocog Alfa Pegol in Patients, Who Have Been Diagnosed With Hemophilia A
Official Title Observational Study Evaluating Effectiveness and Safety of Real-World Treatment With Damoctocog Alfa Pegol in Previously Treated Patients With Hemophilia A
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 Provided
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
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).
Condition 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.
Study Groups/Cohorts 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)。
Intervention: Drug: Damoctocog alfa pegol (Jivi, Bay94-9027)
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: April 26, 2019) 		200
Original Estimated Enrollment Same as current
Estimated Study Completion Date September 30, 2025
Estimated Primary Completion Date June 30, 2025   (Final data collection date for primary outcome measure)
Eligibility Criteria

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.
Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts
Contact: Bayer Clinical Trials Contact (+)1-888-84 22937 clinical-trials-contact@bayer.com
Contact: For trial location information (Phone Menu Options '3' or '4') (+)1-888-84 22937
Listed Location Countries Austria,   Belgium,   Brazil,   Canada,   China,   Colombia,   Denmark,   Finland,   France,   Germany,   Greece,   Italy,   Japan,   Kuwait,   Luxembourg,   Mexico,   Netherlands,   Norway,   Portugal,   Russian Federation,   Saudi Arabia,   Slovenia,   Spain,   Sweden,   Switzerland,   Taiwan,   United Arab Emirates,   United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT03932201
Other Study ID Numbers 20002
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: Undecided
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.
Responsible Party Bayer
Study Sponsor Bayer
Collaborators Not Provided
Investigators Not Provided
PRS Account Bayer
Verification Date May 2021

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