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

NCT04165135 | Completed Results

• 1 other identifier: ML40983
• Study Type: observational
• Target: 103
• Locations: 1 country
• Sites: 17

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.

Conditions

Severe Hereditary Factor VIII Deficiency Disease Without Inhibitor; Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor; Haemophilia A

Keywords

Hemophilia A; Factor VIII

Outcome Measures

Primary Outcomes (3)

• 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

  Physical activity data was collected as daily active minutes measured on each valid day by wearing a fitness tracker continuously during study participation. Valid day = a day in which fitness tracker was worn for at least 10 hours. Daily active minutes were saved in two different sets of data collected by fitness tracker. For each set minutes were categorized by intensity of activity done. First set = minutes categorized according to time collected in each heart zone. Overall heart zone minutes = fat-burn minutes (heart rate: 50-69% of maximum) + cardio minutes (medium-to-high intensity exercise zone corresponds to heart rate: 70-84% of maximum) + peak minutes (high-intensity exercise zone, corresponds to heart rate \> 85% of maximum). Second set =minutes categorized according to active zones. Overall active zone minutes=lightly active minutes +fairly active minutes+very active minutes. Moderate-to-Vigorous Physical Activity (MVPA) minutes=Fairly active minutes+Very active minutes.

  From baseline to study 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

  Number of steps taken by the participants on each valid day were measured by wearing a fitness tracker continuously during study participation. Valid day was defined as a day in which fitness tracker was worn for at least 10 hours.

  From baseline to study 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. The METs were derived as: (amount of kilo calories (kcal) collected in the fat-burn, cardio, and peak minutes) / \[(sum of fat-burn, cardio and peak hours) \* weight of the participant in kilograms (kg)\].

  From baseline to study completion (up to 18 months)

Secondary Outcomes (54)

• 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

  From baseline to study completion (up to 18 months)

• 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

  From baseline to study completion (up to 18 months)

• 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

  From baseline to study completion (up to 18 months)

• 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

  From baseline to study 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

  From baseline to study completion (up to 18 months)

Study Arms (1)

Haemophilia A Without FVIII Inhibitors

Drug: Standard of Care for Haemophilia A

Interventions

Standard of Care for Haemophilia A DRUG

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.

Haemophilia A Without FVIII Inhibitors

Eligibility Criteria

• Age: 12 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• 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.

You may qualify if:

• 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

You may not qualify if:

• 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.)

Sponsors & Collaborators

• Hoffmann-La Roche: lead

Study Sites (17)

AOU Cons. Policlinico Bari; Centro Emofilia e Trombosi - UO Medicina Trasfusionale

Bari, Apulia, 70124, Italy

Location

Az. Osp. Pugliese Ciaccio; S. C. Emofilia, Emostasi e Trombosi

Catanzaro, Calabria, 88100, Italy

Location

AOU Federico II; Medicina Clinica Chirurgia Centro Emocoaugulopatie e Emofilia

Napoli, Campania, 80131, Italy

Location

PO Cesena - Osp M. Bufalini; Patologia Clinica - S.S.A. Serv. Medicina Trasf.- Centro Emofilia

Cesena, Emilia-Romagna, 47521, Italy

Location

Universita' Degli Studi La Sapienza-Ist.Di Ematologia; Dip Biot Cel e Ematol

Rome, Lazio, 00161, Italy

Location

Ospedale Pediatrico Bambino Gesù; Oncoematologia Centro Emostasi e Trombosi

Rome, Lazio, 00165, Italy

Location

Policlinico Univ. A. Gemelli; Polo di Scienze Oncologiche ed Ematologiche

Rome, Lazio, 00168, Italy

Location

IRCCS Ca' Granda Ospedale Maggiore Policlinico; Centro Emofilia e Trombosi "Angelo Bianchi e Bonomi"

Milan, Lombardy, 20122, Italy

Location

Fondazione IRCCS Policlinico San Matteo; S.C. Medicina Generale 2 - Centro Emofilia

Pavia, Lombardy, 27100, Italy

Location

A.O.Santi Antonio e Biagio e Cesare Arrigo; S.S. Emostasi e Trombosi - Dip. Onco-Ema e Med Spec.

Alessandria, Piedmont, 15100, Italy

Location

AO Città Salute e Scienza D-Osp S. G. Battista Molinette; SSCVD Malattie Tromboti e Emorragiche

Turin, Piedmont, 10126, Italy

Location

Osp. Infant. Regina Margherita; S.S.D. Medicina Trasfusionale Materno-infantile Traumatologica

Turin, Piedmont, 10126, Italy

Location

Azienda Uni Ria Policlinico P. Giaccone ; Divisione Di Ematologia E Trapianto

Palermo, Sicily, 90127, Italy

Location

Ospedale Ferrarotto; Divisione Di Ematologia

Via S. Sofia 78, Sicily, 95123, Italy

Location

AOU Careggi; SOD Malattie Emorragiche

Florence, Tuscany, 50134, Italy

Location

Azienda Ospedaliera di Padova; Centro Emofilia

Padua, Veneto, 35128, Italy

Location

Ospedale Di Vicenza; Nefrologia, Ematologia

Vicenza, Veneto, 36100, Italy

Location

Related Publications (1)

• 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.

  PMID: 34855619 DERIVED

MeSH Terms

Conditions

Hemophilia A

Interventions

Standard of Care; Factor VIII

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Quality Indicators, Health Care; Quality of Health Care; Health Services Administration; Health Care Quality, Access, and Evaluation; Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Results Point of Contact

• Title: Medical Communications
• Organization: Hoffmann-La Roche

genentech@druginfo.com

800 821 8590

Study Officials

• Clinical Trials

  Hoffmann-La Roche

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 31, 2019
• First Posted: November 15, 2019
• Study Start: February 24, 2020
• Primary Completion: April 28, 2022
• Study Completion: April 28, 2022
• Last Updated: July 12, 2024
• Results First Posted: July 12, 2024

Record last verified: 2024-01

Data Sharing

• IPD Sharing: Will not share

Locations

IT (17)