Study Evaluating Refacto For Pharmacovigilance

Pharmacovigilance Evaluation Of Refacto In Usual Care Settings

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT00195442

Enrollment

288

Registered

2005-09-19

Start date

1999-07-31

Completion date

2010-01-31

Last updated

2011-02-11

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hemophilia A

Keywords

Hemophilia A

Brief summary

The purpose of this study is to investigate the effectiveness and safety of treatment with ReFacto under conditions of routine therapy. Furthermore a continuous benefit/risk assessment will be done.

Detailed description

Non-interventional study: subjects to be selected according to the usual clinical practice of their physician

Interventions

DRUGMoroctocog alfa

Patients will be treated in accordance with the requirements of the labeling of ReFacto (Moroctocog alfa) in Germany. The dosage and duration of therapy is to be determined by the physician to meet the patients' individual needs for treatment.

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

MALE

Healthy volunteers

Inclusion criteria

* Proven diagnosis of Hemophilia A

Exclusion criteria

* Contraindications according to Summary of Product Characteristics

Design outcomes

Primary

Measure	Time frame	Description
Mean Number of Bleeding Episodes Per Patient Year	Baseline up to a mean duration of 54 months	Participants with hemophilia A suffer from a hereditary lack of blood clotting factor VIII. As a consequence, the ability of the blood to coagulate is reduced and bleedings at any site or organ of the body may occur after minor injury or even spontaneously. Predominantly, joints, muscles, and internal organs are affected by bleeding complications. Participants reported the occurrence of each bleeding episode while on study. The bleeding rate for each participant was calculated by number of reported episodes per years on study.
Mean Number of Bleeding-related Exposure Days Per Patient Year	Baseline up to a mean duration of 54 months	Exposure days are the number of days of treatment with ReFacto.
Mean Number of Exposure Days Per Patient Year	Baseline up to a mean duration of 54 months	Exposure days are the number of days of treatment with ReFacto.

Secondary

Measure	Time frame	Description
Number of Participants With de Novo Inhibitor Formation	Baseline up to a mean duration of 54 months	The applied criteria of clinical relevance for de novo inhibitor formation was defined as normal Factor VIII dosage was ineffective to control a bleeding, control of bleeding episodes required increasing Factor VIII dosage, change of concentrate type (administration of activated Prothrombin-Complex Concentrate \[aPCC\] or recombinant Factor VII \[rFVII \]) was needed to stop a bleeding, or change of therapy strategy (intensive prophylaxis or Immune Tolerance Induction \[ITI\]) was required.
Mean Annual ReFacto Consumption Per Patient Year	Baseline up to a mean duration of 54 months	ReFacto administered as International Units (IU) according to the physician's decision following the drug's summary of product characteristics (SPC) and according to usual care principles.
Number of Participants for Physicians' Assessment of Satisfaction With Treatment Success	Baseline up to a mean duration of 54 months	Subjective assessment by the physician to evaluate treatment success (i.e., control of bleeding, Factor VIII consumption, treatment efficacy and tolerance, handling of preparation, and days missing from work or school). Physician rated assessment could be categorized as Very satisfied, Satisfied, Unsatisfied, or Very unsatisfied; no criteria was pre-specified for the assessment categories in this observational study.
Number of Participants for Physicians' Assessment of Efficacy	Baseline up to a mean duration of 54 months	Subjective assessment by the physician to evaluate control of bleeding. Physician rated assessment could be categorized as Very good, Good, Moderate, or Poor; no criteria was pre-specified for the assessment categories in this observational study.
Number of Participants for Patients' Assessment of Handling of ReFacto	Baseline up to a mean duration of 54 months	Subjective assessment by the participant on handling (preparation and administration) of ReFacto. Patient rated assessment could be categorized as Very good, Good, Moderate, Poor, or No specification; no criteria was pre-specified for the assessment categories in this observational study.
Number of Participants for Physicians' Assessment of Tolerance	Baseline up to a mean duration of 54 months	Subjective assessment by the physician to evaluate the participants' tolerance of treatment with ReFacto (i.e., dose, administration method, or adverse effects). Physician rated assessment could be categorized as Very good, Good, Moderate, or Poor; no criteria was pre-specified for the assessment categories in this observational study.
Number of Participants for Patients' Assessment of Tolerance	Baseline up to a mean duration of 54 months	Subjective assessment by the participant to evaluate tolerance of treatment with ReFacto (i.e., dose, administration method, or adverse effects). Patient rated assessment could be categorized as Very good, Good, Moderate, Poor, or No specification; no criteria was pre-specified for the assessment categories in this observational study.
Number of Participants for Physicians' Assessment of Handling of ReFacto	Baseline up to a mean duration of 54 months	Subjective assessment by the physician to evaluate the participants' handling (preparation and administration) of ReFacto. Physician rated assessment could be categorized as Very good, Good, Moderate, or Poor; no criteria was pre-specified for the assessment categories in this observational study.
Number of Participants for Patients' Assessment of Efficacy	Baseline up to a mean duration of 54 months	Subjective assessment by the participant to evaluate control of bleeding. Patient rated assessment could be categorized as Very good, Good, Moderate, Poor, or No specification; no criteria was pre-specified for the assessment categories in this observational study.
Number of Participants for Days of Sick Leave Per Month	Baseline up to a mean duration of 54 months	Days of sick leave (missing work or school) per month categorized as No days of absence, Number of days of absence, Long-term inability to work or study, Not employed or at school, or No specification.
Number of Non-serious Adverse Events (AEs) and Serious Adverse Events (SAEs)	Baseline up to a mean duration of 54 months	AEs are any undesired side effect which occurred in a participant undergoing study treatment independent of whether a correlation with study treatment was suspected or not. SAEs are undesired events which were lethal or life-threatening, made hospitalization or extension of hospital stay necessary, lead to permanent damage with handicap (inability to work), as well as congenital anomalies, malignant disease, or overdosing. Also presence of inhibitors, thrombotic events, erythrocyte agglutination, allergic reactions, less than therapeutic effect, and inhibitor development were considered SAEs.

Countries

Austria, Germany

Participant flow

Recruitment details

A total of 288 participants were observed during the study in 48 centers (44 Germany and 4 Austria) from time of First subject first visit May 1999 to Last subject last visit January 2010.

Pre-assignment details

The study was terminated January 2010 due to the introduction of a successor product ReFacto® AF.

Participants by arm

Arm	Count
ReFacto (Moroctocog Alfa) B-domain deleted, recombinant factor VIII (BDDrFVIII) for participants with Hemophilia A in usual health care settings.	288
Total	288

Withdrawals & dropouts

Period	Reason	FG000
Overall Study	Death	7
Overall Study	Intermittent product shortage	21
Overall Study	Less than expected therapeutic effect	38
Overall Study	Lost to Follow-up	27
Overall Study	No reason specified	4
Overall Study	Other	14
Overall Study	Poor cooperation	2
Overall Study	Switch to other Factor VIII preparation	23

Baseline characteristics

Characteristic	ReFacto (Moroctocog Alfa)
Age, Customized Adults ≥ 18 years of age	182 participants
Age, Customized Children < 18 years of age	106 participants
Sex: Female, Male Female	0 Participants
Sex: Female, Male Male	288 Participants

Adverse events

Event type	EG000 affected / at risk
deaths Total, all-cause mortality	— / —
other Total, other adverse events	118 / 288
serious Total, serious adverse events	141 / 288

Outcome results

Primary

Mean Number of Bleeding Episodes Per Patient Year

Participants with hemophilia A suffer from a hereditary lack of blood clotting factor VIII. As a consequence, the ability of the blood to coagulate is reduced and bleedings at any site or organ of the body may occur after minor injury or even spontaneously. Predominantly, joints, muscles, and internal organs are affected by bleeding complications. Participants reported the occurrence of each bleeding episode while on study. The bleeding rate for each participant was calculated by number of reported episodes per years on study.