Hemophilia Inhibitor Previously Untreated Patient Study
Public ClinicalTrials.gov record NCT01652027. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Study of Immunologic Determinants of Inhibitor Development in Previously Untreated Patients With Hemophilia
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT01652027
- Recruitment status
- Completed
- Study type
- Observational
- Phase
- Not listed
- Enrollment
- 25 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- Not listed
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jun 30, 2011
- Primary completion
- Feb 29, 2020
- Completion
- Feb 29, 2020
- Last update posted
- Nov 9, 2020
2011 – 2020
United States locations
- U.S. sites
- 13
- U.S. states
- 11
- U.S. cities
- 12
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Emory University | Atlanta | Georgia | 30322 | — |
| Indiana Hemophilia and Thrombosis Center | Indianapolis | Indiana | 46260 | — |
| University of Kentucky | Lexington | Kentucky | 40536 | — |
| Tulane University | New Orleans | Louisiana | 70112 | — |
| Cornell University | New York | New York | 10065 | — |
| University of North Carolina at Chapel Hill | Chapel Hill | North Carolina | 27599 | — |
| Cincinnati Children's Hospital | Cincinnati | Ohio | 45229 | — |
| University of Oregon | Portland | Oregon | — | — |
| Hemophilia Center of Western Pennsylvania | Pittsburgh | Pennsylvania | 15213 | — |
| North Texas Comprehensive Hemophilia Center | Dallas | Texas | 75235 | — |
| Baylor College of Medicine | Houston | Texas | 77030 | — |
| University of Texas Health Science Center-Houston | Houston | Texas | 77030 | — |
| Intermountain Hemophilia and Thrombosis Center | Salt Lake City | Utah | 84108 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 4 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT01652027, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Nov 9, 2020 · Synced Sep 21, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete protocol, eligibility criteria, and contact information for NCT01652027 live on ClinicalTrials.gov.