NCT04165135已完成不适用
A Multicenter, Non-Interventional Study to Evaluate Physical Activity, Bleeding Incidence and Health Related Quality of Life, in Patients With Haemophilia A Without Inhibitors Receiving Standard of Care Treatment
适应症
干预措施
相关药物
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 103
- 试验地点
- 17
- 主要终点
- Mean Number of Daily Active Minutes of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study
研究概览
简要总结
This multicenter, non-interventional, prospective study will collect information about activity status, bleeds, health-related quality of life (HRQoL), health status, and safety in participants with moderate or severe haemophilia A without factor VIII (FVIII) inhibitors, who are being treated in accordance with normal clinical practice.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 12 Years 至 50 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Must own a device compatible with the electronic Patient-Reported Outcome (ePRO) application and with the fitness tracker that will be provided to the patient
- •Must have on his/her own device a data traffic availability of at least 2 gigabytes (GB) in total per month intended only for use of study applications and data transfer. If the data traffic plan is exhausted, the participant must be able to connect to a wi-fi network at least once every day in order to transfer the data collected for the study purpose
- •Must accept to run on his/her own device the ePRO application and the fitness tracker application
- •Must be available to turn on daily the bluetooth connection of his/her own device in order to allow the synchronization with the fitness tracker
- •Ability and willingness to comply with all aspects of the protocol, including completion of questions on the ePRO application (for underage population, ePRO questions can be answered by legally authorized representative if deemed necessary)
- •Ability and willingness to wear the activity tracking device as indicated
- •Diagnosis of severe (FVIII <1%) or moderate (FVIII ≥1% and ≤2%) congenital haemophilia A
- •No prior history of a positive inhibitor against FVIII. If participant has a previous history of inhibitor development, the participant must have successfully eradicated inhibitors since 3 years.
- •At least 150 exposure days of FVIII prior to enrolment
排除标准
- •Bleeding disorder other than congenital haemophilia A
- •Ongoing (or planned during the study) immune tolerance induction or FVIII prophylaxis if the participant has currently low titre of inhibitors or had inhibitors in the past 3 years
- •Previous or concomitant autoimmune or connective tissue disease
- •History of or suspected allergy or intolerance to any of the component of the fitness device (e.g., aluminium anodised)
- •History of clinically significant hypersensitivity associated with monoclonal antibody
- •Obesity (Body Mass Index [BMI] ≥30 kilograms/metre squared of body surface area [kg/m^2})
- •Clinically important cardiovascular, metabolic, endocrine disorders or any other concomitant diseases or conditions that could limit the mobility of participant or could represent any risk according to the Investigator's judgment, or that could interfere with the study evaluation parameters
- •Participation in any other interventional clinical trial, including Roche sponsored studies or in any other support program that may include drug administration other than standard clinical practice (e.g., compassionate use, use not in agreement with the authorized indications, patient support programs, etc.)
研究组 & 干预措施
Haemophilia A Without FVIII Inhibitors
干预措施: Standard of Care for Haemophilia A (Drug)
结局指标
主要结局
Mean Number of Daily Active Minutes of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study
时间窗: From baseline to study completion (up to 18 months)
Physical activity data was collected as daily active minutes measured on each valid day by wearing a fitness tracker continuously during study participation. Valid day = a day in which fitness tracker was worn for at least 10 hours. Daily active minutes were saved in two different sets of data collected by fitness tracker. For each set minutes were categorized by intensity of activity done. First set = minutes categorized according to time collected in each heart zone. Overall heart zone minutes = fat-burn minutes (heart rate: 50-69% of maximum) + cardio minutes (medium-to-high intensity exercise zone corresponds to heart rate: 70-84% of maximum) + peak minutes (high-intensity exercise zone, corresponds to heart rate \> 85% of maximum). Second set =minutes categorized according to active zones. Overall active zone minutes=lightly active minutes +fairly active minutes+very active minutes. Moderate-to-Vigorous Physical Activity (MVPA) minutes=Fairly active minutes+Very active minutes.
Mean Metabolic Equivalent of Tasks (METs) Per Day Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study
时间窗: From baseline to study completion (up to 18 months)
The MET is an objective measure of the ratio of the rate at which a person expends energy, relative to the mass of that person, while performing some specific physical activity compared to a reference. The METs were derived as: (amount of kilo calories (kcal) collected in the fat-burn, cardio, and peak minutes) / \[(sum of fat-burn, cardio and peak hours) \* weight of the participant in kilograms (kg)\].
Mean Number of Steps Taken Per Day by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study
时间窗: From baseline to study completion (up to 18 months)
Number of steps taken by the participants on each valid day were measured by wearing a fitness tracker continuously during study participation. Valid day was defined as a day in which fitness tracker was worn for at least 10 hours.
次要结局
- Mean Rate of the Number of Times Engaging in Physical Activity Per Week by Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study(From baseline to study completion (up to 18 months))
- Number of Bleeds Treated With Each Route of Administration Over the Course of the Study(Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months))
- Yearly Rate of Days Away From School or Work for Participants(Baseline and every month until study completion (up to 18 months))
- Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Different Types of Bleeds (Spontaneous, Traumatic and Procedure/Surgery Related), Reported Via the ePRO Application(Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months).)
- Number of Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study(From baseline to study completion (up to 18 months))
- Number of Bleeds Which Needed to Receive a Transfusion(Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months))
- Number of Bleeds Which Needed to Administer an Additional Hemostatic Therapy(Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months))
- Number of Participants Who Were Adherent to the Treatment Regimen for Haemophilia A Over the Course of the Study by Adherence Category (<80% vs. ≥80% of Study Weeks)(From baseline to study completion (up to 18 months))
- Number of Active Versus Sedentary Participants by the Severity (Moderate or Severe) of Haemophilia A(From baseline up to study completion (up to 18 months))
- Number of Treated Bleeds Over the Course of the Study(Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months))
- Number of Active Versus Sedentary Participants by the Product of Concomitant Medications Taken to Control Pain Over the Course of the Study(Baseline and every 3 months until study completion (up to 18 months))
- Number of Active Versus Sedentary Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study(From baseline to study completion (up to 18 months))
- HJHS Total Score for Active Versus Sedentary Participants Over Time(Baseline, Months 6 and 12)
- Mean Active Minutes by Intensity (Lightly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study(From baseline to study completion (up to 18 months))
- Pain Intensity, as Reported by Participants Using a Visual Analogue Scale(Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18)
- Health-Related Quality of Life (HRQoL): European Quality of Life-5 Dimensions (EQ-5D-5L) Health State Utility Index Score Over Time(Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18)
- Yearly Rate of Days Away From Work for Parent/Caregiver(Baseline and every month until study completion (up to 18 months))
- Number of Active Versus Sedentary Participants by the Type of Regimen Used for Treatment of Haemophilia A Over the Course of the Study(From baseline to study completion (up to 18 months))
- Percentage of Participants Who Were Adherent to World Health Organization (WHO) Global Recommendations on Physical Activity for Health Over the Course of the Study(From baseline to study completion (up to 18 months))
- Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study, Reported Via the ePRO Application(Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months))
- Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Bleed Sites (Urine, Feces, and Vomit, Joint, Muscle, and Other Bleeds), Reported Via the ePRO Application(Baseline and whenever bleeding occured from study initiation to completion (up to 18 months))
- Number of Participants by the Type of Regimen Used for Treatment of Haemophilia A Over the Course of the Study(From baseline to study completion (up to 18 months))
- Number of Participants Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used(From baseline to the time of switch to a new therapy (up to 18 months))
- Number of Bleeds Treated With Each Type of Treatment for Bleeding (Haemostatic Treatment, Treatment for Pain)(From baseline to whenever bleeding occurred (up to 18 months))
- Duration of Treatment for Bleeding Over the Course of the Study(Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months))
- Number of Participants by the Product of Concomitant Medications Taken to Control Pain Over the Course of the Study(Baseline and every 3 months until study completion (up to 18 months))
- Mean Body Mass Index (BMI) Over Time(Baseline, Months 3, 6, 9, 12, 15 and 18)
- Mean Active Minutes by Intensity (Fairly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study(From baseline to study completion (up to 18 months))
- Mean Active Minutes by Intensity (MVPA Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study(From baseline to study completion (up to 18 months))
- Number of Bleeds With Adherence to Treatment Regimen for Haemophilia A in the Week Preceding the Bleeding(Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months))
- Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score(Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18)
- Haemophilia Joint Health Score (HJHS) Total Score Over Time(Baseline, Months 6 and 12)
- Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale(Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18)
- Yearly Rate of Hospitalization Days(Baseline and every month until study completion (up to 18 months))
- Age of Possible Early Retirement of Caregiver, as Reported by Caregivers in a Questionnaire(Baseline and every month until study completion (up to 18 months))
- Yearly Rate of Number of Days Away From School/Work for Active Versus Sedentary Participants(Baseline and every month until study completion (up to 18 months))
- Yearly Rate of Number of Days Away From Work for Active Versus Sedentary Participant's Parents or Caregivers(Baseline and every month until study completion (up to 18 months))
- Yearly Rate of Days Away From Work for Parents/Caregivers of Participants With Moderate Versus Severe Haemophilia A(Baseline and every month until study completion (up to 18 months))
- Yearly Rate of Hospitalization Days for Participants With Moderate Versus Severe Haemophilia A(Baseline and every month until study completion (up to 18 months))
- Annualized Bleeding Rates (ABR) in Active Versus Sedentary Participants(Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months))
- Number of Active Versus Sedentary Participants Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used(From baseline to the time of switch to a new therapy (up to 18 months))
- HRQoL: EQ-5D-5L Health State Utility Index Score Over Time for Active Versus Sedentary Participants(Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18)
- Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Active Versus Sedentary Participants(Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18)
- Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale(Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17 and 18)
- Number of Participants With Moderate Versus Severe Haemophilia A by the Type of Regimen Used for Treatment of Haemophilia A Over the Course of the Study(From baseline to study completion (up to 18 months))
- Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A(From baseline to study completion (up to 18 months))
- Number of Participants With Moderate Versus Severe Haemophilia A Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used(From baseline to the time of switch to a new therapy (up to 18 months))
- Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Participants With Moderate Versus Severe Haemophilia A(Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18)
- HJHS Total Score for Participants With Moderate Versus Severe Haemophilia A(Baseline, Months 6 and 12)
- Yearly Rate of Days Away From School for Participants With Moderate Versus Severe Haemophilia A(Baseline and every month until study completion (up to 18 months))
- Yearly Rate of Number of Hospitalization Days for Active Versus Sedentary Participants(Baseline and every month until study completion (up to 18 months))
- Number of Participants With Moderate Versus Severe Haemophilia A by the Product of Concomitant Medications Taken to Control Pain Over the Course of the Study(Baseline and every 3 months until study completion (up to 18 months))
- HRQoL: EQ-5D-5L Health State Utility Index Score Over Time Moderate Versus Severe Haemophilia A(Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18)
- Annualized Bleeding Rates in Active Versus Sedentary Participants by the Severity (Moderate Versus Severe) of Haemophilia A(Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months))
研究者
研究点 (17)
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