Web-based Application for the Population Pharmacokinetic Service - Phase 1

NCT02061072 | Completed

• 1 other identifier: 13-351-D
• Study Type: observational
• Target: 400
• Locations: 1 country
• Sites: 1

Brief Summary

The aims of this trials are:

1. 1.to collect published and unpublished individual classic pharmacokinetic data (individual patient data from independent investigators and pharmaceutical companies)
2. 2.to make available population pharmacokinetic models for the concentrates derived from the data collected
3. 3.to develop a web based application intended to use the above models to calculate pharmacokinetic parameters for individual patients, and
4. 4.to test the system functionality via simulation of the use of the prototype by use of faked test data.

Conditions

Hemophilia A; Hemophilia B

Keywords

pharmacokinetics; population pharmacokinetics; hemophilia; factor VIII; factor IX

Outcome Measures

Primary Outcomes (1)

• Population pharmacokinetic model(s) performance measure

  The performance of the model(s) will be assessed with statistical indexes of goodness of fit, and by simulation exercises where partial data from individual PK estimates will be used to predict concentration at different time points, subsequently comparing observed and expected values, then compared by chi-square statistics. Similarly, the equivalence of estimates produced on a reduced set of points will be compared with those produced from the analysis of the full data set. Since any PK analysis is by definition providing estimates of the true unknown value, and it is as well know a priori that the population PK estimation will produce estimates with higher uncertainty, only partly dependent on the sample size, the usual approach is not to predefine a cut off to accept or refuse the model, but more so to describe its statistical properties.

  2 year

Secondary Outcomes (4)

• Usability testing of the web-interface

  2 Years

• Tally of number of patients with successful individual PK estimation

  2 years

• Tally of the number of patients with concordant population based and classic PK estimation

  2 years

• Tally of the minimum number of data point allowing a reliable PK estimate for an individual subject

  2 years

Study Arms (1)

Population pharmacokinetic estimation

Patients with severe or moderate hemophilia A or B, providing sparse data for population PK estimation

Other: Population pharmacokinetic estimation

Interventions

Population pharmacokinetic estimation OTHER

Pre-existing individual PK data will be used to derive population PK models. Sparse concentration time data, acquired via a website, will be used to estimate individual pharmacokinetic parameters

Population pharmacokinetic estimation

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with severe or moderate Hemophilia A or B

You may qualify if:

• Patients with severe or moderate Hemophilia A or B
• Informed consent to data processing

You may not qualify if:

• Non measurable plasma factor VIII due to high titre inhibitor

Sponsors & Collaborators

• McMaster University: lead
• Canadian Hemophilia Society: collaborator

Study Sites (1)

Hemophilia Clinic, McMaster University

Hamilton, Ontario, L8S 4K1, Canada

Location

Related Publications (3)

• Hajducek DM, Sinkovic O, Chelle P, Iorio A, Edginton A. A Global Cross-Sectional Database Study of Low Dose FVIII SHL Prophylaxis in Haemophilia A. Haemophilia. 2025 Jul;31(4):646-656. doi: 10.1111/hae.70061. Epub 2025 May 10.

  PMID: 40347119 DERIVED

• Iorio A, Keepanasseril A, Foster G, Navarro-Ruan T, McEneny-King A, Edginton AN, Thabane L; WAPPS-Hemo co-investigator network. Development of a Web-Accessible Population Pharmacokinetic Service-Hemophilia (WAPPS-Hemo): Study Protocol. JMIR Res Protoc. 2016 Dec 15;5(4):e239. doi: 10.2196/resprot.6558.

  PMID: 27977390 DERIVED

• McEneny-King A, Foster G, Iorio A, Edginton AN. Data Analysis Protocol for the Development and Evaluation of Population Pharmacokinetic Models for Incorporation Into the Web-Accessible Population Pharmacokinetic Service - Hemophilia (WAPPS-Hemo). JMIR Res Protoc. 2016 Dec 7;5(4):e232. doi: 10.2196/resprot.6559.

  PMID: 27927609 DERIVED

MeSH Terms

Conditions

Hemophilia A; Hemophilia B

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; Genetic Diseases, X-Linked

Study Officials

• Alfonso Iorio, MD,PhD

  McMaster University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: OTHER
• Target Duration: 1 Week
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 4, 2014
• First Posted: February 12, 2014
• Study Start: January 1, 2015
• Primary Completion: June 1, 2019
• Study Completion: June 1, 2019
• Last Updated: March 16, 2021

Record last verified: 2020-03

Locations

CA (1)