Clinical trial NCT04165135: 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
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Severe Hereditary Factor VIII Deficiency Disease Without Inhibitor
Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor
Haemophilia A
Keywords: Hemophilia A
Factor VIII
Lead sponsor: Hoffmann-La Roche
Sponsor class: INDUSTRY
First posted: 2019-11-15
Enrollment: 103
Sex: ALL
Minimum age (years): 12
Maximum age (years): 50
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

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.


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.)

Primary outcomes:
measure: 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
timeFrame: From baseline to study completion (up to 18 months)
measure: 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
timeFrame: From baseline to study completion (up to 18 months)
measure: 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
timeFrame: From baseline to study completion (up to 18 months)
Secondary outcomes:
measure: 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
timeFrame: From baseline to study completion (up to 18 months)
measure: 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
timeFrame: From baseline to study completion (up to 18 months)
measure: 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
timeFrame: From baseline to study completion (up to 18 months)
measure: 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
timeFrame: From baseline to study completion (up to 18 months)
measure: Percentage of Participants Who Were Adherent to World Health Organization (WHO) Global Recommendations on Physical Activity for Health Over the Course of the Study
timeFrame: From baseline to study completion (up to 18 months)
measure: Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study, Reported Via the ePRO Application
timeFrame: Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months)
measure: 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
timeFrame: Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months).
measure: 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
timeFrame: Baseline and whenever bleeding occured from study initiation to completion (up to 18 months)
measure: Number of Participants by the Type of Regimen Used for Treatment of Haemophilia A Over the Course of the Study
timeFrame: From baseline to study completion (up to 18 months)
measure: Number of Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study
timeFrame: From baseline to study completion (up to 18 months)
measure: Number of Participants Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used
timeFrame: From baseline to the time of switch to a new therapy (up to 18 months)
measure: Number of Treated Bleeds Over the Course of the Study
timeFrame: Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months)
measure: Number of Bleeds Treated With Each Type of Treatment for Bleeding (Haemostatic Treatment, Treatment for Pain)
timeFrame: From baseline to whenever bleeding occurred (up to 18 months)
measure: Duration of Treatment for Bleeding Over the Course of the Study
timeFrame: Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months)
measure: Number of Bleeds Treated With Each Route of Administration Over the Course of the Study
timeFrame: Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months)
measure: Number of Bleeds Which Needed to Receive a Transfusion
timeFrame: Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months)
measure: Number of Bleeds Which Needed to Administer an Additional Hemostatic Therapy
timeFrame: Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months)
measure: Number of Bleeds With Adherence to Treatment Regimen for Haemophilia A in the Week Preceding the Bleeding
timeFrame: Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months)
measure: 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)
timeFrame: From baseline to study completion (up to 18 months)
measure: Number of Participants by the Product of Concomitant Medications Taken to Control Pain Over the Course of the Study
timeFrame: Baseline and every 3 months until study completion (up to 18 months)
measure: Pain Intensity, as Reported by Participants Using a Visual Analogue Scale
timeFrame: Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18
measure: Health-Related Quality of Life (HRQoL): European Quality of Life-5 Dimensions (EQ-5D-5L) Health State Utility Index Score Over Time
timeFrame: Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18
measure: Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score
timeFrame: Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18
measure: Haemophilia Joint Health Score (HJHS) Total Score Over Time
timeFrame: Baseline, Months 6 and 12
measure: Mean Body Mass Index (BMI) Over Time
timeFrame: Baseline, Months 3, 6, 9, 12, 15 and 18
measure: Yearly Rate of Days Away From School or Work for Participants
timeFrame: Baseline and every month until study completion (up to 18 months)
measure: Yearly Rate of Days Away From Work for Parent/Caregiver
timeFrame: Baseline and every month until study completion (up to 18 months)
measure: Yearly Rate of Hospitalization Days
timeFrame: Baseline and every month until study completion (up to 18 months)
measure: Age of Possible Early Retirement of Caregiver, as Reported by Caregivers in a Questionnaire
timeFrame: Baseline and every month until study completion (up to 18 months)
measure: Number of Active Versus Sedentary Participants by the Severity (Moderate or Severe) of Haemophilia A
timeFrame: From baseline up to study completion (up to 18 months)
measure: Annualized Bleeding Rates (ABR) in Active Versus Sedentary Participants
timeFrame: Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months)
measure: Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale
timeFrame: Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18
measure: Number of Active Versus Sedentary Participants by the Product of Concomitant Medications Taken to Control Pain Over the Course of the Study
timeFrame: Baseline and every 3 months until study completion (up to 18 months)
measure: Number of Active Versus Sedentary Participants by the Type of Regimen Used for Treatment of Haemophilia A Over the Course of the Study
timeFrame: From baseline to study completion (up to 18 months)
measure: Number of Active Versus Sedentary Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study
timeFrame: From baseline to study completion (up to 18 months)
measure: Number of Active Versus Sedentary Participants Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used
timeFrame: From baseline to the time of switch to a new therapy (up to 18 months)
measure: HRQoL: EQ-5D-5L Health State Utility Index Score Over Time for Active Versus Sedentary Participants
timeFrame: Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18
measure: Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Active Versus Sedentary Participants
timeFrame: Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18
measure: HJHS Total Score for Active Versus Sedentary Participants Over Time
timeFrame: Baseline, Months 6 and 12
measure: Yearly Rate of Number of Days Away From School/Work for Active Versus Sedentary Participants
timeFrame: Baseline and every month until study completion (up to 18 months)
measure: Yearly Rate of Number of Days Away From Work for Active Versus Sedentary Participant's Parents or Caregivers
timeFrame: Baseline and every month until study completion (up to 18 months)
measure: Yearly Rate of Number of Hospitalization Days for Active Versus Sedentary Participants
timeFrame: Baseline and every month until study completion (up to 18 months)
measure: Annualized Bleeding Rates in Active Versus Sedentary Participants by the Severity (Moderate Versus Severe) of Haemophilia A
timeFrame: Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months)
measure: Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale
timeFrame: Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17 and 18
measure: 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
timeFrame: Baseline and every 3 months until study completion (up to 18 months)
measure: 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
timeFrame: From baseline to study completion (up to 18 months)
measure: Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A
timeFrame: From baseline to study completion (up to 18 months)
measure: 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
timeFrame: From baseline to the time of switch to a new therapy (up to 18 months)
measure: HRQoL: EQ-5D-5L Health State Utility Index Score Over Time Moderate Versus Severe Haemophilia A
timeFrame: Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18
measure: Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Participants With Moderate Versus Severe Haemophilia A
timeFrame: Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18
measure: HJHS Total Score for Participants With Moderate Versus Severe Haemophilia A
timeFrame: Baseline, Months 6 and 12
measure: Yearly Rate of Days Away From School for Participants With Moderate Versus Severe Haemophilia A
timeFrame: Baseline and every month until study completion (up to 18 months)
measure: Yearly Rate of Days Away From Work for Parents/Caregivers of Participants With Moderate Versus Severe Haemophilia A
timeFrame: Baseline and every month until study completion (up to 18 months)
measure: Yearly Rate of Hospitalization Days for Participants With Moderate Versus Severe Haemophilia A
timeFrame: Baseline and every month until study completion (up to 18 months)
Other outcomes:
Locations:
city: Catanzaro
country: Italy
facility: Az. Osp. Pugliese Ciaccio; S. C. Emofilia, Emostasi e Trombosi
state: Calabria
city: Napoli
country: Italy
facility: AOU Federico II; Medicina Clinica Chirurgia Centro Emocoaugulopatie e Emofilia
state: Campania
city: Cesena
country: Italy
facility: PO Cesena - Osp M. Bufalini; Patologia Clinica - S.S.A. Serv. Medicina Trasf.- Centro Emofilia
state: Emilia-Romagna
city: Roma
country: Italy
facility: Universita' Degli Studi La Sapienza-Ist.Di Ematologia; Dip Biot Cel e Ematol
state: Lazio
city: Roma
country: Italy
facility: Ospedale Pediatrico Bambino Gesù; Oncoematologia Centro Emostasi e Trombosi
state: Lazio
city: Roma
country: Italy
facility: Policlinico Univ. A. Gemelli; Polo di Scienze Oncologiche ed Ematologiche
state: Lazio
city: Milano
country: Italy
facility: IRCCS Ca' Granda Ospedale Maggiore Policlinico; Centro Emofilia e Trombosi "Angelo Bianchi e Bonomi"
state: Lombardia
city: Pavia
country: Italy
facility: Fondazione IRCCS Policlinico San Matteo; S.C. Medicina Generale 2 - Centro Emofilia
state: Lombardia
city: Alessandria
country: Italy
facility: A.O.Santi Antonio e Biagio e Cesare Arrigo; S.S. Emostasi e Trombosi - Dip. Onco-Ema e Med Spec.
state: Piemonte
city: Torino
country: Italy
facility: AO Città Salute e Scienza D-Osp S. G. Battista Molinette; SSCVD Malattie Tromboti e Emorragiche
state: Piemonte
city: Torino
country: Italy
facility: Osp. Infant. Regina Margherita; S.S.D. Medicina Trasfusionale Materno-infantile Traumatologica
state: Piemonte
city: Bari
country: Italy
facility: AOU Cons. Policlinico Bari; Centro Emofilia e Trombosi - UO Medicina Trasfusionale
state: Puglia
city: Palermo
country: Italy
facility: Azienda Uni Ria Policlinico P. Giaccone ; Divisione Di Ematologia E Trapianto
state: Sicilia
city: Via S. Sofia 78
country: Italy
facility: Ospedale Ferrarotto; Divisione Di Ematologia
state: Sicilia
city: Firenze
country: Italy
facility: AOU Careggi; SOD Malattie Emorragiche
state: Toscana
city: Padova
country: Italy
facility: Azienda Ospedaliera di Padova; Centro Emofilia
state: Veneto
city: Vicenza
country: Italy
facility: Ospedale Di Vicenza; Nefrologia, Ematologia
state: Veneto
Study officials:
affiliation: Hoffmann-La Roche
name: Clinical Trials
role: STUDY_DIRECTOR
References:
citation: Petracca F, Tempre R, Cucciniello M, Ciani O, Pompeo E, Sannino L, Lovato V, Castaman G, Ghirardini A, Tarricone R. An Electronic Patient-Reported Outcome Mobile App for Data Collection in Type A Hemophilia: Design and Usability Study. JMIR Form Res. 2021 Dec 1;5(12):e25071. doi: 10.2196/25071.