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

Haemophilia A Clinical Trial

— POWER  

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04165135
• Other study ID #: ML40983
• Status: Completed
• Start date: February 24, 2020
• Est. completion date: April 28, 2022

Study information

• Verified date: May 2022
• Source: Hoffmann-La Roche
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 107
• Est. completion date: April 28, 2022
• Est. primary completion date: April 28, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years to 50 Years

Eligibility

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.)

Related Conditions & MeSH terms

• Deficiency Diseases
• Haemophilia A
• Hemophilia A
• Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor
• Severe Hereditary Factor VIII Deficiency Disease Without Inhibitor

Intervention

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.

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Hoffmann-La Roche

Country where clinical trial is conducted

Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
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		Daily from study initiation to completion (up to 18 months)
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		Daily from study initiation to completion (up to 18 months)
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	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	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)
Secondary	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)
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	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)
Secondary	Number of Bleeds Over Time		Baseline and whenever bleeding occurs from study initiation to completion (up to 18 months)
Secondary	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)
Secondary	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)
Secondary	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)
Secondary	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)
Secondary	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)
Secondary	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)
Secondary	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)
Secondary	Haemophilia Joint Health Score (HJHS) Total Score Over Time		Baseline, 6 months, and study completion (up to 18 months)
Secondary	Mean Body Mass Index Over Time		Baseline and every 3 months until study completion (up to 18 months)
Secondary	Number of Days Away from School for Participants		Baseline and every month until study completion (up to 18 months)
Secondary	Number of Days Away from Work for Participants and/or Caregivers		Baseline and every month until study completion (up to 18 months)
Secondary	Number of Hospitalization Days		Baseline and every month until study completion (up to 18 months)
Secondary	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)
Secondary	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)
Secondary	Annualized Bleeding Rates in Active Versus Sedentary Participants		Baseline and whenever bleeding occurs from study initiation to completion (up to 18 months)
Secondary	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)
Secondary	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)
Secondary	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)
Secondary	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)
Secondary	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)
Secondary	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)
Secondary	Number of Hospitalization Days for Active Versus Sedentary Participants		Baseline and every month until study completion (up to 18 months)
Secondary	Number of Days Away from School for Active Versus Sedentary Participants		Baseline and every month until study completion (up to 18 months)
Secondary	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)
Secondary	Haemophilia Joint Health Score (HJHS) Total Score for Active Versus Sedentary Participants Over Time		Baseline, 6 months, and study completion (up to 18 months)
Secondary	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)
Secondary	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)
Secondary	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)
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	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)
Secondary	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)
Secondary	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)
Secondary	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)
Secondary	Number of Hospitalization Days for Participants with Moderate Versus Severe Haemophilia A		Baseline and every month until study completion (up to 18 months)
Secondary	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)
Secondary	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)
Secondary	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)