N/A
N=103
An Observational Study to Evaluate Physical Activity, Bleeding Incidence and Health Related Quality of Life, in Participants With Haemophilia A Without Inhibitors Receiving Standard of Care Treatment
Severe Hereditary Factor VIII Deficiency Disease Without Inhibitor · Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor · Haemophilia A
Bottom Line
View on ClinicalTrials.gov: NCT04165135 ↗Enrolled (actual)
103
Serious AEs
—
Results posted
Jul 2024
Primary outcome: Primary: 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 — 53.2; 24.6; 39.2; 226.5 number of daily active minutes — p=0.7630
Study Design & Population
- Study type
- Observational
- Phase
- N/A
- Interventions
- Standard of Care for Haemophilia A (Drug)
- Age
- Pediatric, Adult · 12+ yrs
- Sex
- All
- Sponsor
- Hoffmann-La Roche
- Primary completion
- Apr 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY 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 |
53.2; 24.6; 39.2; 226.5; 309.4; 284.1 | 0.7630 |
| PRIMARY 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 |
5402.4; 8788.3; 8279.8 | 0.0649 |
| PRIMARY 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 |
5.1; 5.3; 4.5 | 0.0026 sig |
| SECONDARY 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 |
0.06; 0.12; 0.18; 0.03; 0.06; 0.03 | 0.9493 |
| SECONDARY 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 |
0.1; 0.5; 0.3; 2.7; 2.8; 3.8 | 0.6151 |
| SECONDARY 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 |
0.0; 1.1; 0.3; 1.7; 6.3; 4.5 | 0.0745 |
| SECONDARY 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 |
4.3; 36.6; 12.8; 2.3; 14.4; 6.4 | 0.0085 sig |
| SECONDARY Percentage of Participants Who Were Adherent to World Health Organization (WHO) Global Recommendations on Physical Activity for Health Over the Course of the Study |
0; 68.2; 42.3; 100; 31.8; 57.7 | 0.0088 sig |
| SECONDARY Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study, Reported Via the ePRO Application |
11; 25; 22; 6; 21; 18 | — |
| SECONDARY 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 |
15; 36; 33; 2; 10; 7 | — |
| SECONDARY 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 |
17; 45; 36; 0; 1; 4 | — |
| SECONDARY Number of Participants by the Type of Regimen Used for Treatment of Haemophilia A Over the Course of the Study |
102; 1 | — |
| SECONDARY Number of Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study |
39; 25; 8; 8; 6; 6 | — |
| SECONDARY Number of Participants Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used |
7; 3; 3; 8 | — |
| SECONDARY Number of Treated Bleeds Over the Course of the Study |
94 | — |
| SECONDARY Number of Bleeds Treated With Each Type of Treatment for Bleeding (Haemostatic Treatment, Treatment for Pain) |
93; 1 | — |
| SECONDARY Duration of Treatment for Bleeding Over the Course of the Study |
2.0 | — |
| SECONDARY Number of Bleeds Treated With Each Route of Administration Over the Course of the Study |
90; 0; 0; 0; 0; 4 | — |
| SECONDARY Number of Bleeds Which Needed to Receive a Transfusion |
— | — |
| SECONDARY Number of Bleeds Which Needed to Administer an Additional Hemostatic Therapy |
4 | — |
| SECONDARY Number of Bleeds With Adherence to Treatment Regimen for Haemophilia A in the Week Preceding the Bleeding |
77 | — |
| SECONDARY 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) |
72; 27 | — |
| SECONDARY Number of Participants by the Product of Concomitant Medications Taken to Control Pain Over the Course of the Study |
5; 4 | — |
| SECONDARY Pain Intensity, as Reported by Participants Using a Visual Analogue Scale |
14.24; 18.38; 16.91; 17.53; 21.77; 18.76 | — |
| SECONDARY Health-Related Quality of Life (HRQoL): European Quality of Life-5 Dimensions (EQ-5D-5L) Health State Utility Index Score Over Time |
0.93; 0.89; 0.90; 0.94; 0.94; 0.90 | — |
| SECONDARY Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score |
81.9; 81.0; 80.6; 86.0; 83.5; 75.8 | — |
| SECONDARY Haemophilia Joint Health Score (HJHS) Total Score Over Time |
10; 13.7; 12.5 | — |
| SECONDARY Mean Body Mass Index (BMI) Over Time |
23.34; 23.56; 23.42; 23.76; 22.98 | — |
| SECONDARY Yearly Rate of Days Away From School or Work for Participants |
— | — |
| SECONDARY Yearly Rate of Days Away From Work for Parent/Caregiver |
— | — |
| SECONDARY Yearly Rate of Hospitalization Days |
0.0 | — |
| SECONDARY Age of Possible Early Retirement of Caregiver, as Reported by Caregivers in a Questionnaire |
57.8 | — |
| SECONDARY Number of Active Versus Sedentary Participants by the Severity (Moderate or Severe) of Haemophilia A |
3; 54; 3; 34; 0; 9 | — |
| SECONDARY Annualized Bleeding Rates (ABR) in Active Versus Sedentary Participants |
1.945; 1.384 | — |
| SECONDARY Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale |
17.86; 14.12; 21.29; 15.73; 18.32; 16.75 | — |
| SECONDARY Number of Active Versus Sedentary Participants by the Product of Concomitant Medications Taken to Control Pain Over the Course of the Study |
2; 2; 1; 2 | — |
| SECONDARY Number of Active Versus Sedentary Participants by the Type of Regimen Used for Treatment of Haemophilia A Over the Course of the Study |
28; 25; 0; 1 | — |
| SECONDARY Number of Active Versus Sedentary Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study |
8; 10; 8; 7; 3; 2 | — |
| SECONDARY Number of Active Versus Sedentary Participants Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used |
1; 4; 1; 1; 1; 0 | — |
| SECONDARY HRQoL: EQ-5D-5L Health State Utility Index Score Over Time for Active Versus Sedentary Participants |
0.90; 0.93; 0.89; 0.89; 0.90; 0.95 | — |
| SECONDARY Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Active Versus Sedentary Participants |
79.9; 78.1; 81.0; 76.9; 81.8; 80.0 | — |
| SECONDARY HJHS Total Score for Active Versus Sedentary Participants Over Time |
11.8; 12.1; 29.3; 8.7; 17.3; 15.0 | — |
| SECONDARY Yearly Rate of Number of Days Away From School/Work for Active Versus Sedentary Participants |
0.0; 0.0 | — |
| SECONDARY Yearly Rate of Number of Days Away From Work for Active Versus Sedentary Participant's Parents or Caregivers |
0.0; 0.0 | — |
| SECONDARY Yearly Rate of Number of Hospitalization Days for Active Versus Sedentary Participants |
0; 0 | — |
| SECONDARY Annualized Bleeding Rates in Active Versus Sedentary Participants by the Severity (Moderate Versus Severe) of Haemophilia A |
0.368; 2.536; 1.517; 1.035 | — |
| SECONDARY Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale |
23.33; 13.66; 23.67; 17.96; 19.20; 16.75 | — |
| SECONDARY 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 |
0; 5; 0; 4 | — |
| SECONDARY 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 |
5; 97; 1; 0 | — |
| SECONDARY Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A |
1; 38; 2; 23; 1; 7 | — |
| SECONDARY 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 |
2; 5; 0; 3; 0; 3 | — |
| SECONDARY HRQoL: EQ-5D-5L Health State Utility Index Score Over Time Moderate Versus Severe Haemophilia A |
0.92; 0.93; 0.89; 0.88; 0.90; 0.90 | — |
| SECONDARY Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Participants With Moderate Versus Severe Haemophilia A |
88.3; 81.5; 81.0; 81.2; 80.5; 75.0 | — |
| SECONDARY HJHS Total Score for Participants With Moderate Versus Severe Haemophilia A |
8.3; 10.1; 20.0; 13.3; 0.0; 12.8 | — |
| SECONDARY Yearly Rate of Days Away From School for Participants With Moderate Versus Severe Haemophilia A |
0.0; 0.0 | — |
| SECONDARY Yearly Rate of Days Away From Work for Parents/Caregivers of Participants With Moderate Versus Severe Haemophilia A |
0.0; 0 | — |
| SECONDARY Yearly Rate of Hospitalization Days for Participants With Moderate Versus Severe Haemophilia A |
0; 0 | — |
Summary
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.
Eligibility Criteria
Inclusion Criteria
- 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
Exclusion Criteria
- 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.)
Data sourced from ClinicalTrials.gov (NCT04165135). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.