Hemophilia AHIPSNCT01652027
Hemophilia Inhibitor Previously Untreated Patient Study
An observational study of Hemophilia A, run by The University of Texas Health Science Center, Houston at 17 sites in 5 countries.
Trial 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… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hemophilia A
- What is being tested?
- FVIII concentrate
- How many people
- 25 participants
- Where
- 17 sitesin 5 countries
- Study dates
- Jul 2011to Mar 2020
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with severe hemophilia A who have not previously been treated with Factor VIII concentrates.
Who may be able to join
- Severe hemophilia A with FVIII activity < 1% normal
- Weight > 3.5 kg at the time of baseline study evaluation
- Informed consent, approved by appropriate Institutional Review Board/Independent Ethics Committee, has been administered, signed, and dated
Who cannot join
- Prior exposure to clotting factor concentrates or blood products
- Other chronic disease
- Currently participating in another investigational drug study.
Trial locations and contacts
17 sites in 5 countries.
17 sites in 5 countries
United States13 sites
Emory University
Atlanta, Georgia, 30322
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229
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
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
Hemophilia Center of Western Pennsylvania
Pittsburgh, Pennsylvania, 15213
University of Oregon
Portland, Oregon
Intermountain Hemophilia and Thrombosis Center
Salt Lake City, Utah, 84108
About this study
Official title: Study of Immunologic Determinants of Inhibitor Development in Previously Untreated Patients With Hemophilia
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
- Hemophilia A
- Being tested
- FVIII concentrate
- Keywords
- FVIII inhibitors
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FVIII concentrateDrug
usual treatment as directed by treating physician
| Study group | Role | What they receive |
|---|---|---|
| Previously Untreated Patients with Hemophilia A | Not described Receives: Drug: FVIII concentrate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Total number of FOXP3-positive T regulatory cells in the circulation
FoxP3(a protein involved in immune system responses)-positive T regulatory cells in the circulation will be compared before and after exposure to FVIII.
Measured: 50 exposure days to FVIII or 3 years, whichever comes first
Secondary outcomes (1)
FVIII-specific T-cells
FVIII-specific T-cells will be compared before and after exposure to FVIII
Measured: 50 exposure days to FVIII or 3 years, whichever comes first
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jul 2012 (estimated)
- Study startJul 2011
- Primary completionMar 2020
- Study completionMar 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- The University of Texas Health Science Center, Houston
- Sponsor type
- University, hospital or other
- Collaborators
- Rho, Inc., Baxter Healthcare Corporation
- Responsible party
- Principal investigator, Deborah Brown, The University of Texas Health Science Center, Houston
- Investigators
- Elena Santagostino, M.D., Principal investigator, Maggiore Hospital and University of Milan
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01652027
- Sponsor's protocol ID
- HSC-MS-11-0202
- Other IDs
- HSC-MS-11-0202
- Acronym
- HIPS
- Status last verified
- Oct 2026
- First submitted
- 3 Jun 2011
- Last updated
- 22 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Paul H, Berg V, Gangadharan B, Bowen J, LeBeau P, Blatny J, Male C, Radulescu VC, Diaz R, Mancuso ME, Brown DL, Reipert BM. Prospective Hemophilia Inhibitor PUP Study reveals distinct antibody signatures during FVIII inhibitor eradication. Blood Adv. 2023 May 9;7(9):1831-1848. doi: 10.1182/bloodadvances.2022007267.
PubMed 36074992Reipert BM, Gangadharan B, Hofbauer CJ, Berg V, Schweiger H, Bowen J, Blatny J, Fijnvandraat K, Mullins ES, Klintman J, Male C, McGuinn C, Meeks SL, Radulescu VC, Ragni MV, Recht M, Shapiro AD, Staber JM, Yaish HM, Santagostino E, Brown DL. The prospective Hemophilia Inhibitor PUP Study reveals distinct antibody signatures prior to FVIII inhibitor development. Blood Adv. 2020 Nov 24;4(22):5785-5796. doi: 10.1182/bloodadvances.2020002731.
PubMed 33232473