BAY14-2222 Prophylaxis and Joint Function Improvement (Adults)

Bayer20 enrolled

Overview

The purpose of the study is to evaluate the effect of prophylactic treatment on the number of joint bleeds and quality of life in severe hemophilia A subjects compared to on-demand treatment in a one-group two-treatment schedule design. In addition, the effect of prophylactic treatment on the joint function, the number of all bleeds, and on the quality of life compared to on-demand treatment and health-economic data will be assessed.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Hematologic Disease Hemophilia A

Interventions

Kogenate (BAY14-2222)DRUG

One group two treatment schedules, first on-demand then switch to prophylaxis

Eligibility

Sex: MALEMin age: 30 YearsMax age: 45 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Severe hemophilia A (\< 1% FVIII) * 30-45 years of age * Previously treated subject (\> 100 Exposure days to any FVIII) * On-demand therapy with any FVIII Exclusion Criteria: * No history of inhibitor * No planned elective orthopedic surgery during the study duration (13 months) * No severe concomitant disease * No history of anaphylactic or other severe reaction to previous FVIII treatment

Locations (11)

Unnamed facility

Aurora, Colorado, United States

Unnamed facility

Chapel Hill, North Carolina, United States

Unnamed facility

Houston, Texas, United States

Unnamed facility

Strasbourg, France

Outcomes

Primary Outcomes

Number of Joint Bleeds

Number of joint bleeds during Months 8-13 compared to number of joint bleeds during Months 1-6

Time frame: Months 1-6 (on-demand treatment) and 8-13 (prophylactic treatment)

Secondary Outcomes

Number of All Bleeds

Mean number of all bleeds during Months 8-13 (prophylactic) compared to mean number of all bleeds during Months 1-6 (on-demand)

Time frame: Months 1-6 (on-demand treatment) and 8-13 (prophylactic treatment)

Physical Assessment Compared to On-demand Treatment as Determined by the Gilbert Score

Total score with a range of 0-100, evaluating ankle, knee and elbow, 0 indicates normal function, higher values indicate joint damage

Time frame: Month 13 (end of prophylactic treatment) and Month 6 (end of on-demand treatment)

Quality of Life Compared to On-demand Treatment as Measured by the Haemo-QoL A Questionnaire

Total transformed score with a range of 0-100, higher values indicate better outcome. 41 items in 6 domains: physical functioning; role functioning; worry; consequences; positive affect; treatment concern.

Time frame: Month 13 (end of prophylactic treatment) and Month 6 (end of on-demand treatment)

Data from ClinicalTrials.gov

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