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)

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

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 Overview

• Status: Completed
• Conditions: Haemophilia A; Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor; Severe Hereditary Factor VIII Deficiency Disease Without Inhibitor
• Intervention / Treatment: Drug: Standard of Care for Haemophilia A

• Study Type: Observational
• Enrollment (Actual): 107

Contacts and Locations

Study Locations

• Italy
  • Calabria
    • Catanzaro, Calabria, Italy, 88100
      • Az. Osp. Pugliese Ciaccio; S. C. Emofilia, Emostasi e Trombosi
  • Campania
    • Napoli, Campania, Italy, 80131
      • AOU Federico II; Medicina Clinica Chirurgia Centro Emocoaugulopatie e Emofilia
  • Emilia-Romagna
    • Cesena, Emilia-Romagna, Italy, 47521
      • PO Cesena - Osp M. Bufalini; Patologia Clinica - S.S.A. Serv. Medicina Trasf.- Centro Emofilia
  • Lazio
    • Roma, Lazio, Italy, 00161
      • Universita' Degli Studi La Sapienza-Ist.Di Ematologia; Dip Biot Cel e Ematol
    • Roma, Lazio, Italy, 00165
      • Ospedale Pediatrico Bambino Gesù; Oncoematologia Centro Emostasi e Trombosi
    • Roma, Lazio, Italy, 00168
      • Policlinico Univ. A. Gemelli; Polo di Scienze Oncologiche ed Ematologiche
  • Lombardia
    • Milano, Lombardia, Italy, 20122
      • IRCCS Ca' Granda Ospedale Maggiore Policlinico; Centro Emofilia e Trombosi "Angelo Bianchi e Bonomi"
    • Pavia, Lombardia, Italy, 27100
      • Fondazione IRCCS Policlinico San Matteo; S.C. Medicina Generale 2 - Centro Emofilia
  • Piemonte
    • Alessandria, Piemonte, Italy, 15100
      • A.O.Santi Antonio e Biagio e Cesare Arrigo; S.S. Emostasi e Trombosi - Dip. Onco-Ema e Med Spec.
    • Torino, Piemonte, Italy, 10126
      • AO Città Salute e Scienza D-Osp S. G. Battista Molinette; SSCVD Malattie Tromboti e Emorragiche
    • Torino, Piemonte, Italy, 10126
      • Osp. Infant. Regina Margherita; S.S.D. Medicina Trasfusionale Materno-infantile Traumatologica
  • Puglia
    • Bari, Puglia, Italy, 70124
      • AOU Cons. Policlinico Bari; Centro Emofilia e Trombosi - UO Medicina Trasfusionale
  • Sicilia
    • Palermo, Sicilia, Italy, 90127
      • Azienda Uni Ria Policlinico P. Giaccone ; Divisione Di Ematologia E Trapianto
    • Via S. Sofia 78, Sicilia, Italy, 95123
      • Ospedale Ferrarotto; Divisione Di Ematologia
  • Toscana
    • Firenze, Toscana, Italy, 50134
      • AOU Careggi; SOD Malattie Emorragiche
  • Veneto
    • Padova, Veneto, Italy, 35128
      • Azienda Ospedaliera di Padova; Centro Emofilia
    • Vicenza, Veneto, Italy, 36100
      • Ospedale Di Vicenza; Nefrologia, Ematologia

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years to 50 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• 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.

Description

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.)

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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		Daily from study initiation to completion (up to 18 months)
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		Daily from study initiation to completion (up to 18 months)
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	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.	Daily from study initiation to completion (up to 18 months)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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		Daily from study initiation to completion (up to 18 months)
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		Daily from study initiation to 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	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.	Daily from study initiation to completion (up to 18 months)
Number of Bleeds Over Time		Baseline and whenever bleeding occurs 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	The following are the types of regimens used to treat haemophilia A: on-demand, prophylaxis, or preventative treatment before physical activity.	Daily from study initiation to completion (up to 18 months)
Number of Participants by the Product and Dosage Used for Treatment of Haemophilia A Over the Course of the Study		Daily from study initiation to completion (up to 18 months)
Number of Participants Who Were Adherent to the Treatment Regimen Over the Course of the Study		Daily from study initiation to completion (up to 18 months)
Number of Participants by the Product and Dosage 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)
Pain Intensity, as Reported by Participants Using a Visual Analogue Scale	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".	Baseline and every month until study completion (up to 18 months)
European Quality of Life-5 Dimensions (EQ-5D-5L) Health-Related Quality of Life Score Over Time		Baseline and every 3 months until study completion (up to 18 months)
Health Status Over Time, as Reported by Participants Using a Visual Analogue Scale	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.	Baseline and every 3 months until study completion (up to 18 months)
Haemophilia Joint Health Score (HJHS) Total Score Over Time		Baseline, 6 months, and study completion (up to 18 months)
Mean Body Mass Index Over Time		Baseline and every 3 months until study completion (up to 18 months)
Number of Days Away from School for Participants		Baseline and every month until study completion (up to 18 months)
Number of Days Away from Work for Participants and/or Caregivers		Baseline and every month until study completion (up to 18 months)
Number 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)
Number of Active Versus Sedentary Participants by the Severity (Moderate or Severe) of Haemophilia A		Baseline and daily from study initiation to completion (up to 18 months)
Annualized Bleeding Rates in Active Versus Sedentary Participants		Baseline and whenever bleeding occurs from study initiation to completion (up to 18 months)
Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale	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".	Baseline and every month until study completion (up to 18 months)
Number of Active Versus Sedentary Participants by the Product and Dosage 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 Type of Regimen Used for Treatment of Haemophilia A Over the Course of the Study	The following are the types of regimens used to treat haemophilia A: on-demand, prophylaxis, or preventative treatment before physical activity.	Daily from study initiation to completion (up to 18 months)
Number of Active Versus Sedentary Participants by the Product and Dosage Used for Treatment of Haemophilia A Over the Course of the Study		Daily from study initiation to completion (up to 18 months)
EQ-5D-5L Health-Related Quality of Life Score Over Time for Active Versus Sedentary Participants		Baseline and every 3 months until study completion (up to 18 months)
Health Status Over Time for Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale	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.	Baseline and every 3 months until study completion (up to 18 months)
Number of Hospitalization Days for Active Versus Sedentary Participants		Baseline and every month until study completion (up to 18 months)
Number of Days Away from School for Active Versus Sedentary Participants		Baseline and every month until study completion (up to 18 months)
Number of Days Away from Work for Active Versus Sedentary Participants and/or Caregivers		Baseline and every month until study completion (up to 18 months)
Haemophilia Joint Health Score (HJHS) Total Score for Active Versus Sedentary Participants Over Time		Baseline, 6 months, and study completion (up to 18 months)
Annualized Bleeding Rates in Participants with Moderate Versus Severe Haemophilia A		Baseline and whenever bleeding occurs from study initiation to completion (up to 18 months)
Pain Intensity in Participants with Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale	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".	Baseline and every month until study completion (up to 18 months)
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		Baseline and every 3 months until study completion (up to 18 months)
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	The following are the types of regimens used to treat haemophilia A: on-demand, prophylaxis, or preventative treatment before physical activity.	Daily from study initiation to completion (up to 18 months)
Number of Participants with Moderate Versus Severe Haemophilia A by the Product and Dosage Used for Treatment of Haemophilia A		Daily from study initiation to completion (up to 18 months)
EQ-5D-5L Health-Related Quality of Life Score Over Time for Participants with Moderate Versus Severe Haemophilia A		Baseline and every 3 months until study completion (up to 18 months)
Health Status Over Time for Participants with Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale	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.	Baseline and every 3 months until study completion (up to 18 months)
Number of Hospitalization Days for Participants with Moderate Versus Severe Haemophilia A		Baseline and every month until study completion (up to 18 months)
Number of Days Away from School for Participants with Moderate Versus Severe Haemophilia A		Baseline and every month until study completion (up to 18 months)
Number of Days Away from Work for Participants with Moderate Versus Severe Haemophilia A and/or Caregivers		Baseline and every month until study completion (up to 18 months)
Haemophilia Joint Health Score (HJHS) Total Score for Participants with Moderate Versus Severe Haemophilia A		Baseline, 6 months, and study completion (up to 18 months)

Collaborators and Investigators

• Sponsor: Hoffmann-La Roche

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start (Actual): February 24, 2020
• Primary Completion (Actual): April 28, 2022
• Study Completion (Actual): April 28, 2022

Study Registration Dates

• First Submitted: October 31, 2019
• First Submitted That Met QC Criteria: November 13, 2019
• First Posted (Actual): November 15, 2019

Study Record Updates

• Last Update Posted (Actual): May 5, 2022
• Last Update Submitted That Met QC Criteria: May 4, 2022
• Last Verified: May 1, 2022

More Information

Terms related to this study

• Keywords: Factor VIII; Hemophilia A
• Additional Relevant MeSH Terms: Hematologic Diseases; Nutrition Disorders; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Malnutrition; Blood Coagulation Disorders; Hemophilia A; Deficiency Diseases; Coagulants; Factor VIII
• Other Study ID Numbers: ML40983

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

IPD 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).

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No