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Web-based Application for the Population Pharmacokinetic Service - Phase 1 (WAPPS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02061072
Recruitment Status : Unknown
Verified May 2018 by McMaster University.
Recruitment status was:  Enrolling by invitation
First Posted : February 12, 2014
Last Update Posted : May 16, 2018
Canadian Hemophilia Society
Information provided by (Responsible Party):
McMaster University

Brief Summary:

The aims of this trials are:

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 to collect published and unpublished individual classic pharmacokinetic data (individual patient data from independent investigators and pharmaceutical companies)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

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Study Type : Observational [Patient Registry]
Estimated Enrollment : 400 participants
Observational Model: Other
Time Perspective: Other
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

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Group/Cohort Intervention/treatment
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

Primary Outcome Measures :
  1. 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.

Secondary Outcome Measures :
  1. 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.

  2. 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

  3. 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".

  4. 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.

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with severe or moderate Hemophilia A or B

Inclusion Criteria:

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

Exclusion Criteria:

  • Non measurable plasma factor VIII due to high titre inhibitor

Information from the National Library of Medicine

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 identifier (NCT number): NCT02061072

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Canada, Ontario
Hemophilia Clinic, McMaster University
Hamilton, Ontario, Canada, L8S 4K1
Sponsors and Collaborators
McMaster University
Canadian Hemophilia Society
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Principal Investigator: Alfonso Iorio, MD,PhD McMaster University
Additional Information:

Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: McMaster University Identifier: NCT02061072    
Other Study ID Numbers: 13-351-D
First Posted: February 12, 2014    Key Record Dates
Last Update Posted: May 16, 2018
Last Verified: May 2018
Keywords provided by McMaster University:
population pharmacokinetics
factor VIII
factor IX
Additional relevant MeSH terms:
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Hemophilia A
Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked