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

N/AActive Not RecruitingNCT03932201

Bayer371 enrolled

Overview

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 Type

OBSERVATIONAL

Enrollment

371

Conditions

Hemophilia A

Interventions

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.

Eligibility

Sex: ALL

Medical Language ↔ Plain English

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.

Locations (29)

South Alabama Medical Science Foundation

Outcomes

Primary Outcomes

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

Secondary Outcomes

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.

Time frame: Up to 36 months

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)

HJHS:Hemophilia Joint Health Score

Time 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

Data from ClinicalTrials.gov

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Mobile, Alabama, United States

Banner MD Anderson Cancer Center

Phoenix, Arizona, United States

University California Davis

Davis, California, United States

Orthopaedic Hospital DBA Orthopaedic Institute for Children

Los Angeles, California, United States

The Center for Comprehensive Care and Diagnosis of Inherited Blood Disorders

Orange, California, United States

University of Colorado Hemophilia and Thrombosis Center

Aurora, Colorado, United States

Georgetown University

Washington D.C., District of Columbia, 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

...and 19 more locations

Time frame: Up to 36 months

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.

Bleeds includes total, spontaneous, joint, and trauma.

Time frame: Up to 36 months

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.

AUC:Area under the plasma concentration versus time curve

Time frame: Up to 36 months

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

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).

Time 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.

Time 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.

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