NCT02750085

Brief Summary

This is an investigator-initiated, industry-funded, multi-centre, international study that will be carried out prospectively at hemophilia treatment centres across Canada, the Czech Republic and Australia with SickKids as the coordinating site. The study will use a central laboratory not directly affiliated with any of the participating sites. Enrollment target is 50 participants, both adult and pediatric with severe hemophilia A receiving Advate, who will each complete a 2-point and 6-point pharmacokinetic (PK) sampling. The main aim is to compare the results of a 2 sample PK using clinically practical time points and myPKFiT™ (a web-based, population PK Bayesian tool) to a 6 sample population PK to determine whether the results obtained are in good agreement.

Trial Health

35
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
39

participants targeted

Target at P25-P50 for all trials

Timeline
Completed

Started Apr 2016

Longer than P75 for all trials

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

April 1, 2016

Completed
6 days until next milestone

First Submitted

Initial submission to the registry

April 7, 2016

Completed
18 days until next milestone

First Posted

Study publicly available on registry

April 25, 2016

Completed
2.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2019

Completed
1.9 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2020

Completed
Last Updated

May 15, 2020

Status Verified

May 1, 2020

Enrollment Period

2.8 years

First QC Date

April 7, 2016

Last Update Submit

May 13, 2020

Conditions

Hemophilia A

Keywords

Hemophilia ApharmacokineticsmyPKFiT

Outcome Measures

Primary Outcomes (9)

  • Terminal half-life (t1/2)

    2-point PK sampling protocol against 6-point PK sampling protocol

    2 years

  • area under the plasma concentration versus time curve (AUC)

    2-point PK sampling protocol against 6-point PK sampling protocol

    2 years

  • Area under the moment curve (AUMC)

    2-point PK sampling protocol against 6-point PK sampling protocol

    2 years

  • In vivo recovery (IVR)

    2-point PK sampling protocol against 6-point PK sampling protocol

    2 years

  • Maximum concentration (Cmax)

    2-point PK sampling protocol against 6-point PK sampling protocol

    2 years

  • Clearance (Cl)

    2-point PK sampling protocol against 6-point PK sampling protocol

    2 years

  • Volume of distribution at steady state (Vss)

    2-point PK sampling protocol against 6-point PK sampling protocol

    2 years

  • Mean residence time (MRT)

    2-point PK sampling protocol against 6-point PK sampling protocol

    2 years

  • Time to factor VIII concentration of 1% over baseline

    2-point PK sampling protocol against 6-point PK sampling protocol

    2 years

Study Arms (1)

2-point and 6-point PK sampling

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Patients with severe Hemophilia A

You may qualify if:

  • Confirmed diagnosis of Hemophilia A;
  • Severe disease (FVIII \<2%);
  • Receiving ADVATE for prevention of bleeding (prophylaxis) or receiving ADVATE on demand and a candidate for prophylaxis;
  • Body weight ≤120 kg; and ≥12kg;

You may not qualify if:

  • FVIII inhibitor positive (level of ≥0.6 Bethesda Units \[BU\] per mL using the Nijmegen modification of the Bethesda assay). Inhibitor status to be documented as negative prior to study enrollment according to the two most recent, consecutive inhibitor assays on record. If patients have \< 50 exposure days, an assay will be completed centrally within a reasonable timeframe (approximately 8 weeks suggested) to make sure that they are negative.
  • Body weight \>120 kg or \<12kg;
  • Human immunodeficiency virus (HIV) positivity with cluster of differentiation 4 (CD4) count \< 200 / microliter;
  • Significant hepatic dysfunction, alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \>5 times upper limit of normal
  • History of recent events that might affect FVIII half-life (e.g., infection, surgery or an invasive procedure) within 2 weeks of blood sampling.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Biospecimen

Retention: SAMPLES WITHOUT DNA

Plasma collected from PK blood sampling

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Victor S Blanchette, MD

    The Hospital for Sick Children

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
CASE ONLY
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Medical Director, Pediatric Thrombosis and Hemostasis Program

Study Record Dates

First Submitted

April 7, 2016

First Posted

April 25, 2016

Study Start

April 1, 2016

Primary Completion

January 1, 2019

Study Completion

December 1, 2020

Last Updated

May 15, 2020

Record last verified: 2020-05

Data Sharing

IPD Sharing
Will not share