Web-based Application for the Population Pharmacokinetic Service - Phase 1 (WAPPS)
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|ClinicalTrials.gov Identifier: NCT02061072|
Recruitment Status : Enrolling by invitation
First Posted : February 12, 2014
Last Update Posted : May 16, 2018
The aims of this trials are:
- to collect published and unpublished individual classic pharmacokinetic data (individual patient data from independent investigators and pharmaceutical companies)
- to make available population pharmacokinetic models for the concentrates derived from the data collected
- to develop a web based application intended to use the above models to calculate pharmacokinetic parameters for individual patients, and
- to test the system functionality via simulation of the use of the prototype by use of faked test data.
|Condition or disease||Intervention/treatment|
|Hemophilia A Hemophilia B||Other: Population pharmacokinetic estimation|
Show Detailed Description
|Study Type :||Observational [Patient Registry]|
|Estimated Enrollment :||400 participants|
|Target Follow-Up Duration:||1 Week|
|Official Title:||The Development of the Web-based Application for the Population Pharmacokinetic Service - Hemophilia (WAPPS-Hemo) - Phase 1|
|Study Start Date :||January 2015|
|Estimated Primary Completion Date :||June 2019|
|Estimated Study Completion Date :||June 2019|
Population pharmacokinetic estimation
Patients with severe or moderate hemophilia A or B, providing sparse data for population PK estimation
Other: Population pharmacokinetic estimation
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 model(s) performance measure [ Time Frame: 2 year ]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.
- Usability testing of the web-interface [ Time Frame: 2 Years ]Users feed-back will be sought by interviews, questionnaires and focus groups during the entire time frame of the study, until saturation will be reached. Suggested changes to the system will be implemented in cycles. Standard techniques for audit-feed-back processes will be adopted.
- Tally of number of patients with successful individual PK estimation [ Time Frame: 2 years ]A tally will be taken of the number of patient PK estimates successfully estimated by the WAPPS-Hemo system as a proportion of the total estimation requested
- Tally of the number of patients with concordant population based and classic PK estimation [ Time Frame: 2 years ]The definition of concordance will be based on clinical ground as "a difference non impacting on treatment decision".
- Tally of the minimum number of data point allowing a reliable PK estimate for an individual subject [ Time Frame: 2 years ]A tally will be taken of the number of data points needed for the WAPPS-Hemo system to provide a clinically useful estimate. The outcome will be explored by collecting full data-points (7-11) on a set of 100 patients, and performing iteratively the estimation with a progressively reduced number of data points down to the minimum number allowing the system to converge and produce estimates.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02061072
|Hemophilia Clinic, McMaster University|
|Hamilton, Ontario, Canada, L8S 4K1|
|Principal Investigator:||Alfonso Iorio, MD,PhD||McMaster University|