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

NCT05752565 | Completed DMC FDA Device

• 1 other identifier: 2364
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (2)

• 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

Study Arms (1)

patients with severe HA under FVIII concentrates prophylaxis

OTHER

patients with severe HA under FVIII concentrates prophylaxis

Device: SenseWear armband device

Interventions

SenseWear armband device 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

patients with severe HA under FVIII concentrates prophylaxis

Eligibility Criteria

• Age: 12 Years - 60 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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)

You may not qualify if:

• 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)

Sponsors & Collaborators

• Fondazione Policlinico Universitario Agostino Gemelli IRCCS: lead

Study Sites (1)

FPG

Roma, 00168, Italy

Location

MeSH Terms

Conditions

Hemophilia A

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

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: February 20, 2023
• First Posted: March 2, 2023
• Study Start: January 9, 2020
• Primary Completion: December 30, 2020
• Study Completion: December 30, 2020
• Last Updated: March 2, 2023

Record last verified: 2023-03

Locations

IT (1)