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
- Are there results?
- None posted in the registry
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.
- More trials run by The University of Texas Health Science Center, HoustonThe sponsor's other registered studies.
- 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
- 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
Study at a glance
From the registry record, last updated by the sponsor on 22 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of Immunologic Determinants of Inhibitor Development in Previously Untreated Patients With Hemophilia
- ClinicalTrials.gov ID
- NCT01652027
- Acronym / sponsor ID
- HIPS / HSC-MS-11-0202
- Conditions
- Hemophilia A
- Keywords
- FVIII inhibitors
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- The University of Texas Health Science Center, HoustonUniversity, hospital or other
- Collaborators
- Rho, Inc., Baxter Healthcare Corporation
- Study start
- Jul 2011 (actual)
- Primary completion
- Mar 2020 (actual)
- Study completion
- Mar 2020 (actual)
- First posted
- 27 Jul 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Previously Untreated Patients with Hemophilia A | – | Drug: FVIII concentrate |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with severe hemophilia A who have not previously been treated with Factor VIII concentrates.
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (3 listed)
- Prior exposure to clotting factor concentrates or blood products
- Other chronic disease
- Currently participating in another investigational drug study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| FVIII concentrateusual treatment as directed by treating physician | Drug | Previously Untreated Patients with Hemophilia A |
Primary outcome
Total number of FOXP3-positive T regulatory cells in the circulation
Time frame: 50 exposure days to FVIII or 3 years, whichever comes first
FoxP3(a protein involved in immune system responses)-positive T regulatory cells in the circulation will be compared before and after exposure to FVIII.
1 secondary outcome
FVIII-specific T-cells
Time frame: 50 exposure days to FVIII or 3 years, whichever comes first
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Oct 2026.
- Derived from this study: PubMed 36074992Paul 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.
- Derived from this study: PubMed 33232473Reipert 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Elena Santagostino, M.D. principal investigator
Maggiore Hospital and University of Milan
17 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia A, run by The University of Texas Health Science Center, Houston at 17 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in United States, Austria, Italy, Netherlands, Sweden. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Total number of FOXP3-positive T regulatory cells in the circulation (time frame: 50 exposure days to FVIII or 3 years, whichever comes first). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01652027, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Oct 2026.