MedTrace Logo

Hemophilia Inhibitor Previously Untreated Patient Study

NCT01652027 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 25

Last updated 2020-11-10

No results posted yet for this study

Summary

Hemophilia A is a congenital bleeding disorder caused by deficiency of factor VIII (FVIII) and is treated by replacement therapy with FVIII concentrate. Approximately 30% of people with severe hemophilia A develop neutralizing antibodies, called FVIII inhibitors, which interfere with the function of FVIII concentrates. The reason that some, but not all, people with severe hemophilia A develop inhibitors is incompletely understood. Understanding individual and environmental risk factors is important to be able to prevent and possibly treat inhibitors. This study will look at individual and treatment characteristics in babies with severe hemophilia A who have not yet received treatment with FVIII (called Previously Untreated Patients, or PUPS). Subjects in the study will be asked to provide diaries of treatments, medications, and illnesses. Treatment will be directed by the subjects' physician, but all subjects will receive Advate, a third-generation recombinant FVIII product. Subjects will have blood drawn for laboratory tests, which include studies of the immune system and genetic studies of the FVIII mutation, before and 7-9 days after the first treatment with FVIII, and 5 days (+/-2 days) after the 5th, 10th, 20th, 30th, 40th, and 50th days of treatment with FVIII (exposure days). The duration of the study will be first 50 treatments or 3 years, whichever comes first.

Conditions

Interventions

DRUG

FVIII concentrate

usual treatment as directed by treating physician

Sponsors & Collaborators

  • Rho, Inc.

    collaborator INDUSTRY

  • Baxter Healthcare Corporation

    collaborator INDUSTRY

  • The University of Texas Health Science Center, Houston

    lead OTHER

Principal Investigators

  • Elena Santagostino, M.D. · Maggiore Hospital and University of Milan

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2011-07-31
Primary Completion
2020-03-31
Completion
2020-03-31

Countries

  • United States
  • Austria
  • Italy
  • Netherlands
  • Sweden

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01652027 on ClinicalTrials.gov