Physical Activity and FVIII Elimination: Relevance to Personalized Therapy in Severe Hemophilia A

In persons with severe haemophilia A infused factor VIII half-life and other pharmacokinetic can vary according to determinants such as blood group, von Willebrand factor (VWF) level or age. However, FVIII pharmacokinetics (PK) has not been throughly studied in patients with severe HA as a function of daily physical activity. It si known that vigorous intensity physical activity/exercise can transiently but significantly increase circulating levels of endogenous VWF and consequently FVIII in normal subjects and in patients with moderate or mild haemophilia A. The proposed study is a Proof of Concept one as it will be aimed at investigating the relation between daily physical activity, measured by Sensewear armband device, as number of daily steps and PK variability of infused rec-FVIII concentrate. This kind of investigation has never been done and it is a great interest also for the evaluation of patient's qualitiy of life.

Study Overview

• Status: Completed
• Conditions: Hemophilia A
• Intervention / Treatment: Device: SenseWear armband device

Detailed Description

In persons with severe haemophilia A infused factor VIII half-life and other pharmacokinetic can vary according to determinants such as blood group, von Willebrand factor (VWF) level or age. However, FVIII pharmacokinetics (PK) has not been throughly studied in patients with severe HA as a function of daily physical activity. It si known that vigorous intensity physical activity/exercise can transiently but significantly increase circulating levels of endogenous VWF and consequently FVIII in normal subjects and in patients with moderate or mild haemophilia A. The proposed study is a Proof of Concept one as it will be aimed at investigating the relation between daily physical activity, measured by Sensewear armband device, as number of daily steps and PK variability of infused rec-FVIII concentrate. This kind of investigation has never been done and it is a great interest also for the evaluation of patient's qualitiy of life. Physical Examinations at screening and subsequent study visits a physical examination will be performed on the following body systems being described as normal or abnormal: general appearance, headand neck, eyes and ears, nose and throat, heart, abdomen, extremities and joints, lymph nodes and neurological. At screening, if an abnormal condition is detected, the condition will be described on the medical history CRF. At study visit, if a new abnormal or worsened abnormal pre-existing condition is detected, the condition will be described n the CRF.

• Study Type: Interventional
• Enrollment (Actual): 20
• Phase: Not Applicable

Contacts and Locations

Study Locations

• Italy
  • Roma, Italy, 00168
    • FPG

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years to 60 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

Description

Inclusion Criteria:

• patients 12-60 yo with severe HA under FVIII concentrates prophylaxis will be selected at the Haemostasis and Thrombosis Center of the Fondazione Policlinico Gemelli IRCCS Rome (FPG)

Exclusion Criteria:

• all patients with malignant tumors or
• patietnts treated with anticoagulant / antiplatelet agents
• patients suffering from other congenital coagulation disorders (von Willebrand disease, other congenital deficiency of coagulation factors or severe thrombocytopenia (< 30,000 Plt/uL)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: patients with severe HA under FVIII concentrates prophylaxis	Device: SenseWear armband device; Device: SenseWear armband device Metabolic Holter, Physical Activity and Lifestyle, Portable outpatient "multi-sensor" energy consumption in calories (EE), movement, physical activity, quality of life

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Study of the association between physical activity, measured by mean nuber of daily steps, with the Area Under the Curve (AUC) PK parameter in patients with severe HA under prophylaxis.	Study of the association between physical activity, measured by mean nuber of daily steps, with the Area Under the Curve (AUC) PK parameter in patients with severe HA under prophylaxis. Investigate whether and how the daily physical activity (measured as number of steps, using the SenseWear Armband device) affects the PK parameter. .	12 months
Association PK parameter and number of daily steps	In more details the study aims to evaluate whether and how the FVIII PK parameter is significantly associated with the mean number of daily steps in patients with severe HA	12 months

Collaborators and Investigators

• Sponsor: Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Study record dates

Study Major Dates

• Study Start (Actual): January 9, 2020
• Primary Completion (Actual): December 30, 2020
• Study Completion (Actual): December 30, 2020

Study Registration Dates

• First Submitted: February 20, 2023
• First Submitted That Met QC Criteria: March 1, 2023
• First Posted (Actual): March 2, 2023

Study Record Updates

• Last Update Posted (Actual): March 2, 2023
• Last Update Submitted That Met QC Criteria: March 1, 2023
• Last Verified: March 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: 2364

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: Yes
• product manufactured in and exported from the U.S.: Yes