Turoctocog Alfa in Haemophilic Italian Patients: Protection and Engagement in Recreational Activities (THIRd)

Turoctocog Alfa in Haemophilic Italian Patients: Protection and Engagement in Recreational Activities (THIRd). An Observational, Prospective, Multicentre, Cohort Study on Italian Patients With Haemophilia A in Prophylaxis Treatment With Turoctocog Alfa Under Current Clinical Conditions

This is a study on Italian patients with haemophilia A in prophylaxis treatment with Turoctocog alfa under routine clinical conditions. The purpose of the study is to investigate the therapeutic scheme in a population of patients treated with Turoctocog alfa and to investigate the participation in recreational activities, the level of physical activity, and quality of life. Participants will get NovoEight® (Turoctocog alfa) as prescribed to them by the study doctor. The study will last for about 12 months. Participants will be requested to fill in the Questionnaires investigating the participation in recreational activities, the level of physical activity, and quality of life.

Study Overview

• Status: Completed
• Conditions: Haemophilia A
• Intervention / Treatment: Drug: Turoctocog alfa

• Study Type: Observational
• Enrollment (Actual): 18

Contacts and Locations

Study Locations

• Italy
  • Bologna, Italy, 40138
    • Novo Nordisk Investigational Site
  • Castelfranco Veneto, Italy, 31033
    • Novo Nordisk Investigational Site
  • Catania, Italy, 95123
    • Novo Nordisk Investigational Site
  • Cesena, Italy, 47521
    • Novo Nordisk Investigational Site
  • Firenze, Italy, 50134
    • Novo Nordisk Investigational Site
  • Milano, Italy, 20089
    • Novo Nordisk Investigational Site
  • Napoli, Italy, 80147
    • Novo Nordisk Investigational Site
  • Roma, Italy, 00161
    • Novo Nordisk Investigational Site
  • Vicenza, Italy, 36100
    • Novo Nordisk Investigational Site
  • MI
    • Milano, MI, Italy, 20124
      • Novo Nordisk Investigational Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: N/A

• Sampling Method: Non-Probability Sample

Study Population

Patients enrolled will have Haemophilia A (any severity), needing turoctocog alpha prophylactic therapy.

Description

Inclusion Criteria:

• Informed consent obtained before any study-related activities. Study-related activities are any procedures that are carried out as part of the study, including activities to determine suitability for the study.
• Male or female, age above or equal to 12 years at the time of signing informed consent.
• Haemophilia A (any severity), needing prophylactic therapy (with the usual recommended doses).
• Decision to treat with turoctocog alfa, made by the treating Physician and the subject/Legally Acceptable Representative (LAR) based on local label before and independently from the decision to include the subject in this study.

Exclusion Criteria:

• Previous participation in this study, defined as previously signed informed consent;
• Presence of other coagulation disorders;
• Presence of any inhibitor;
• Mental problems, poor compliance, linguistic barriers or other conditions which can impede the understanding of the study aims and the participation to it.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Intervention / Treatment

Haemophilia A; Patients enrolled will have Haemophilia A (any severity), needing turoctocog alpha prophylactic therapy.

Drug: Turoctocog alfa; Patients will be treated with commercially available NovoEight® (turoctocog alfa) according to routine clinical practice at the discretion of the treating physician. The decision to initiate treatment with commercially available NovoEight® has been made by the patient/Legally Acceptable Representative (LAR) and the treating physician before and independently from the decision to include the patient in this study.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Individual prophylaxis (PPX) regimen with turoctocog alfa: Every second day, or three times a week (3TW), or twice a week (2TW)	Count	Baseline (week 0)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Individual PPX regimen with turoctocog alfa: every second day, or 3TW, or 2TW	Count	Final visit (12 months)
Change of PPX regimen: YES/NO	Count (participants who have changed regimen or not)	12 months after treatment initiation
Reported reasons for change of PPX regimens	Count (reasons according to the predefined list, see below) Individual reported reasons for switch will be selected among the following predefined: to improve articular protection; due to bleedings; due to patients' needs, including: physical inactivity, work, recreational activity, travel, other.	Final visit (12 months)
Difference in score of 'Bridging Hemophilia Experiences, Results, and Opportunities' BHERO questionnaire	Points The following sections of the BHERO questionnaire will be used: Haemophilia Activities List (HAL: measures the impact of haemophilia on self-perceived functional abilities in patients (42 questions in 7 domains). Scores from 1 (worst) to 6 (best). The final score is the sum of all the scores.); Recreational and sport activities; Brief pain inventory, exploring the presence and intensity of physical pain, and its impact on performance of daily activities.; International Physical Activities questionnaire (IPAQ) score (Score<700 = Inactive Patient, Score 700 to 2519 = Sufficiently active patient, Score ≥ 2520 = Active or very active patient.	Final visit (12 months) vs Baseline (week 0)
Difference in circulating turoctocog alfa levels	IU/mL Before 12 months visit, monitoring will be reported only if measured in clinical practice due to bleedings or other clinical reasons.	Final visit (12 months) vs Baseline (week 0)
Individual Annual Bleeding Rate (ABR)	Count	From baseline to final visit (12 months)

Collaborators and Investigators

• Sponsor: Novo Nordisk A/S
• Investigators: Study Director: Clinical Reporting Anchor and Disclosure 1452, Novo Nordisk A/S

Study record dates

Study Major Dates

• Study Start (Actual): April 12, 2021
• Primary Completion (Actual): March 27, 2023
• Study Completion (Actual): March 27, 2023

Study Registration Dates

• First Submitted: October 6, 2020
• First Submitted That Met QC Criteria: October 6, 2020
• First Posted (Actual): October 14, 2020

Study Record Updates

• Last Update Posted (Actual): November 28, 2023
• Last Update Submitted That Met QC Criteria: November 27, 2023
• Last Verified: November 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Hemophilia A
• Other Study ID Numbers: NN7008-4655; U1111-1247-5494 (Other Identifier: World Health Organisation (WHO))

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: According to the Novo Nordisk disclosure commitment on novonordisk-trials.com.

Drug and device information, study documents

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