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

NCT ID: NCT04165135

Last Updated: 2024-07-12

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 103 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2020-02-24
• Study Completion Date: 2022-04-28

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

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

Haemophilia A Without FVIII Inhibitors

Standard of Care for Haemophilia A

Intervention Type: 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.

Interventions

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.

Intervention Type: DRUG

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.)

• Minimum Eligible Age: 12 Years
• Maximum Eligible Age: 50 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Hoffmann-La Roche

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Clinical Trials

Role: STUDY_DIRECTOR

Hoffmann-La Roche

Locations

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

Bari, Apulia, Italy

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

Catanzaro, Calabria, Italy

AOU Federico II; Medicina Clinica Chirurgia Centro Emocoaugulopatie e Emofilia

Napoli, Campania, Italy

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

Cesena, Emilia-Romagna, Italy

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

Rome, Lazio, Italy

Ospedale Pediatrico Bambino Gesù; Oncoematologia Centro Emostasi e Trombosi

Rome, Lazio, Italy

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

Rome, Lazio, Italy

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

Milan, Lombardy, Italy

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

Pavia, Lombardy, Italy

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

Alessandria, Piedmont, Italy

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

Turin, Piedmont, Italy

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

Turin, Piedmont, Italy

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

Palermo, Sicily, Italy

Ospedale Ferrarotto; Divisione Di Ematologia

Via S. Sofia 78, Sicily, Italy

AOU Careggi; SOD Malattie Emorragiche

Florence, Tuscany, Italy

Azienda Ospedaliera di Padova; Centro Emofilia

Padua, Veneto, Italy

Ospedale Di Vicenza; Nefrologia, Ematologia

Vicenza, Veneto, Italy

Countries

Italy

References

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.

Reference Type: DERIVED

PMID: 34855619 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

ML40983

Identifier Type: -

Identifier Source: org_study_id