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Trial record 1 of 1 for:    ml40983
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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 (POWER)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04165135
Recruitment Status : Completed
First Posted : November 15, 2019
Last Update Posted : May 5, 2022
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche

Brief 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.

Condition or disease Intervention/treatment
Severe Hereditary Factor VIII Deficiency Disease Without Inhibitor Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor Haemophilia A Drug: Standard of Care for Haemophilia A

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Study Type : Observational
Actual Enrollment : 107 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: 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
Actual Study Start Date : February 24, 2020
Actual Primary Completion Date : April 28, 2022
Actual Study Completion Date : April 28, 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Group/Cohort Intervention/treatment
Haemophilia A Without FVIII Inhibitors Drug: Standard of Care for Haemophilia A
There is no pre-determined studied medicinal product. Any treatment (including treatments started during the observational period) will be administered according to standard clinical practice, independently of participation in the current study, according to clinician's independent therapeutic decision.




Primary Outcome Measures :
  1. 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 [ Time Frame: Daily from study initiation to completion (up to 18 months) ]
  2. 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 [ Time Frame: Daily from study initiation to completion (up to 18 months) ]
  3. 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 [ Time Frame: Daily from study initiation to 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.


Secondary Outcome Measures :
  1. Mean Number of Daily Active Minutes 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 [ Time Frame: Daily from study initiation to completion (up to 18 months) ]
  2. Mean Number of Daily Active Minutes by Intensity of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study [ Time Frame: Daily from study initiation to completion (up to 18 months) ]
  3. Percentage of Participants Who Were Adherent to World Health Organization (WHO) Global Recommendations on Physical Activity for Health Over the Course of the Study [ Time Frame: Daily from study initiation to completion (up to 18 months) ]
    Adherence to WHO recommendations on is defined for adults as performing 10000 steps per day and accumulating at least 150 minutes of moderate-intensity physical activity throughout the week.

  4. Number of Bleeds Over Time [ Time Frame: Baseline and whenever bleeding occurs from study initiation to completion (up to 18 months) ]
  5. Number of Participants by the Type of Regimen Used for Treatment of Haemophilia A Over the Course of the Study [ Time Frame: Daily from study initiation to completion (up to 18 months) ]
    The following are the types of regimens used to treat haemophilia A: on-demand, prophylaxis, or preventative treatment before physical activity.

  6. Number of Participants by the Product and Dosage Used for Treatment of Haemophilia A Over the Course of the Study [ Time Frame: Daily from study initiation to completion (up to 18 months) ]
  7. Number of Participants Who Were Adherent to the Treatment Regimen Over the Course of the Study [ Time Frame: Daily from study initiation to completion (up to 18 months) ]
  8. Number of Participants by the Product and Dosage of Concomitant Medications Taken to Control Pain Over the Course of the Study [ Time Frame: Baseline and every 3 months until study completion (up to 18 months) ]
  9. Pain Intensity, as Reported by Participants Using a Visual Analogue Scale [ Time Frame: Baseline and every month until study completion (up to 18 months) ]
    Participants will evaluate the intensity of pain in their main joints (elbow, knee, ankle) by marking on a visual analogue scale (VAS), a horizontal line 100 millimetres in length, anchored by word descriptors at each end, where the left extreme means "No pain = 0" while the right extreme means "Worst Pain Imaginable = 100".

  10. European Quality of Life-5 Dimensions (EQ-5D-5L) Health-Related Quality of Life Score Over Time [ Time Frame: Baseline and every 3 months until study completion (up to 18 months) ]
  11. Health Status Over Time, as Reported by Participants Using a Visual Analogue Scale [ Time Frame: Baseline and every 3 months until study completion (up to 18 months) ]
    Participants will be asked to rate on a visual analogue scale (VAS) from 0 to 100 how good or bad their health is at assessment; 0 means the worst health you can imagine, and 100 means the best health you can imagine.

  12. Haemophilia Joint Health Score (HJHS) Total Score Over Time [ Time Frame: Baseline, 6 months, and study completion (up to 18 months) ]
  13. Mean Body Mass Index Over Time [ Time Frame: Baseline and every 3 months until study completion (up to 18 months) ]
  14. Number of Days Away from School for Participants [ Time Frame: Baseline and every month until study completion (up to 18 months) ]
  15. Number of Days Away from Work for Participants and/or Caregivers [ Time Frame: Baseline and every month until study completion (up to 18 months) ]
  16. Number of Hospitalization Days [ Time Frame: Baseline and every month until study completion (up to 18 months) ]
  17. Age of Possible Early Retirement of Caregiver, as Reported by Caregivers in a Questionnaire [ Time Frame: Baseline and every month until study completion (up to 18 months) ]
  18. Number of Active Versus Sedentary Participants by the Severity (Moderate or Severe) of Haemophilia A [ Time Frame: Baseline and daily from study initiation to completion (up to 18 months) ]
  19. Annualized Bleeding Rates in Active Versus Sedentary Participants [ Time Frame: Baseline and whenever bleeding occurs from study initiation to completion (up to 18 months) ]
  20. Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale [ Time Frame: Baseline and every month until study completion (up to 18 months) ]
    Participants will evaluate the intensity of pain in their main joints (elbow, knee, ankle) by marking on a visual analogue scale (VAS), a horizontal line 100 millimetres in length, anchored by word descriptors at each end, where the left extreme means "No pain = 0" while the right extreme means "Worst Pain Imaginable = 100".

  21. Number of Active Versus Sedentary Participants by the Product and Dosage of Concomitant Medications Taken to Control Pain Over the Course of the Study [ Time Frame: Baseline and every 3 months until study completion (up to 18 months) ]
  22. Number of Active Versus Sedentary Participants by the Type of Regimen Used for Treatment of Haemophilia A Over the Course of the Study [ Time Frame: Daily from study initiation to completion (up to 18 months) ]
    The following are the types of regimens used to treat haemophilia A: on-demand, prophylaxis, or preventative treatment before physical activity.

  23. Number of Active Versus Sedentary Participants by the Product and Dosage Used for Treatment of Haemophilia A Over the Course of the Study [ Time Frame: Daily from study initiation to completion (up to 18 months) ]
  24. EQ-5D-5L Health-Related Quality of Life Score Over Time for Active Versus Sedentary Participants [ Time Frame: Baseline and every 3 months until study completion (up to 18 months) ]
  25. Health Status Over Time for Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale [ Time Frame: Baseline and every 3 months until study completion (up to 18 months) ]
    Participants will be asked to rate on a visual analogue scale (VAS) from 0 to 100 how good or bad their health is at assessment; 0 means the worst health you can imagine, and 100 means the best health you can imagine.

  26. Number of Hospitalization Days for Active Versus Sedentary Participants [ Time Frame: Baseline and every month until study completion (up to 18 months) ]
  27. Number of Days Away from School for Active Versus Sedentary Participants [ Time Frame: Baseline and every month until study completion (up to 18 months) ]
  28. Number of Days Away from Work for Active Versus Sedentary Participants and/or Caregivers [ Time Frame: Baseline and every month until study completion (up to 18 months) ]
  29. Haemophilia Joint Health Score (HJHS) Total Score for Active Versus Sedentary Participants Over Time [ Time Frame: Baseline, 6 months, and study completion (up to 18 months) ]
  30. Annualized Bleeding Rates in Participants with Moderate Versus Severe Haemophilia A [ Time Frame: Baseline and whenever bleeding occurs from study initiation to completion (up to 18 months) ]
  31. Pain Intensity in Participants with Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale [ Time Frame: Baseline and every month until study completion (up to 18 months) ]
    Participants will evaluate the intensity of pain in their main joints (elbow, knee, ankle) by marking on a visual analogue scale (VAS), a horizontal line 100 millimetres in length, anchored by word descriptors at each end, where the left extreme means "No pain = 0" while the right extreme means "Worst Pain Imaginable = 100".

  32. Number of Participants with Moderate Versus Severe Haemophilia A by the Product and Dosage of Concomitant Medications Taken to Control Pain Over the Course of the Study [ Time Frame: Baseline and every 3 months until study completion (up to 18 months) ]
  33. 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 [ Time Frame: Daily from study initiation to completion (up to 18 months) ]
    The following are the types of regimens used to treat haemophilia A: on-demand, prophylaxis, or preventative treatment before physical activity.

  34. Number of Participants with Moderate Versus Severe Haemophilia A by the Product and Dosage Used for Treatment of Haemophilia A [ Time Frame: Daily from study initiation to completion (up to 18 months) ]
  35. EQ-5D-5L Health-Related Quality of Life Score Over Time for Participants with Moderate Versus Severe Haemophilia A [ Time Frame: Baseline and every 3 months until study completion (up to 18 months) ]
  36. Health Status Over Time for Participants with Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale [ Time Frame: Baseline and every 3 months until study completion (up to 18 months) ]
    Participants will be asked to rate on a visual analogue scale (VAS) from 0 to 100 how good or bad their health is at assessment; 0 means the worst health you can imagine, and 100 means the best health you can imagine.

  37. Number of Hospitalization Days for Participants with Moderate Versus Severe Haemophilia A [ Time Frame: Baseline and every month until study completion (up to 18 months) ]
  38. Number of Days Away from School for Participants with Moderate Versus Severe Haemophilia A [ Time Frame: Baseline and every month until study completion (up to 18 months) ]
  39. Number of Days Away from Work for Participants with Moderate Versus Severe Haemophilia A and/or Caregivers [ Time Frame: Baseline and every month until study completion (up to 18 months) ]
  40. Haemophilia Joint Health Score (HJHS) Total Score for Participants with Moderate Versus Severe Haemophilia A [ Time Frame: Baseline, 6 months, and study completion (up to 18 months) ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   12 Years to 50 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Approximately 150 participants overall, 125 with severe haemophilia A and 25 with moderate haemophilia A without inhibitors against factor VIII (FVIII), are planned to be enrolled at sites across Italy.
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 patient 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 patient has a previous history of inhibitor development, the patient 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 patient 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 patients 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.)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04165135


Locations
Show Show 17 study locations
Sponsors and Collaborators
Hoffmann-La Roche
Investigators
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Study Director: Clinical Trials Hoffmann-La Roche
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT04165135    
Other Study ID Numbers: ML40983
First Posted: November 15, 2019    Key Record Dates
Last Update Posted: May 5, 2022
Last Verified: May 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description:

Qualified researchers may request access to individual patient level data through the request platform (www.vivli.org). Further details on Roche's criteria for eligible studies are available here (https://vivli.org/ourmember/roche/).

For further details on Roche's Global Policy on Sharing of Clinical Study Information and how to request access to related clinical study documents, see here (https://www.roche.com/research_and_development/who_we_are_how_we_work/clinical_trials/our_commitment_to_data_sharing.htm).


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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Hoffmann-La Roche:
Hemophilia A
Factor VIII
Additional relevant MeSH terms:
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Hemophilia A
Deficiency Diseases
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Malnutrition
Nutrition Disorders
Factor VIII
Coagulants