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

Brief Title

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

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

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.

Study Type

Observational

Primary Outcome

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

Secondary Outcome

 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

Condition

Severe Hereditary Factor VIII Deficiency Disease Without Inhibitor

Intervention

Standard of Care for Haemophilia A

Study Arms / Comparison Groups

 Haemophilia A Without FVIII Inhibitors

Description:

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status

Drug

Estimated Enrollment

Start Date

February 24, 2020

Completion Date

December 31, 2022

Primary Completion Date

December 31, 2021

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 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.)

Gender

All

Ages

12 Years - 50 Years

Accepts Healthy Volunteers

No

Contacts

Clinical Trials, 888-662-6728 (U.S. Only), [email protected]

Location Countries

Italy

Location Countries

Italy

Administrative Informations

NCT ID

NCT04165135

Organization ID

ML40983

Responsible Party

Sponsor

Study Sponsor

Hoffmann-La Roche

Study Sponsor

Clinical Trials, Study Director, Hoffmann-La Roche

Verification Date

April 2021