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

Sponsor
Fondazione Policlinico Universitario Agostino Gemelli IRCCS (Other)
Overall Status
Completed
CT.gov ID
NCT05752565
Collaborator
(none)
20
Enrollment
1
Location
1
Arm
11.7
Actual Duration (Months)
1.7
Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase
  • Device: SenseWear armband device
 N/A

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 Design

Study Type:
Interventional
Actual Enrollment :
20 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Other
Official Title:
Physical Activity and FVIII Elimination: Relevance to Personalized Therapy in Severe Hemophilia A
Actual Study Start Date :
Jan 9, 2020
Actual Primary Completion Date :
Dec 30, 2020
Actual Study Completion Date :
Dec 30, 2020

Arms and Interventions

Arm Intervention/Treatment
Other: patients with severe HA under FVIII concentrates prophylaxis

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

Outcome Measures

Primary Outcome Measures

  1. 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. [12 months]

    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. .

  2. Association PK parameter and number of daily steps [12 months]

    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

Eligibility Criteria

Criteria

Ages Eligible for Study:
12 Years to 60 Years
Sexes Eligible for Study:
Male
Accepts Healthy Volunteers:
No
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)

Contacts and Locations

Locations

Site City State Country Postal Code
1 FPG Roma Italy 00168

Sponsors and Collaborators

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
De Cristofaro Raimondo, Principal Investigator, Fondazione Policlinico Universitario Agostino Gemelli IRCCS
ClinicalTrials.gov Identifier:
NCT05752565
Other Study ID Numbers:
  • 2364
First Posted:
Mar 2, 2023
Last Update Posted:
Mar 2, 2023
Last Verified:
Mar 1, 2023
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
Additional relevant MeSH terms:
Hemophilia A

Study Results

No Results Posted as of Mar 2, 2023