HIPS: Hemophilia Inhibitor Previously Untreated Patient Study

Sponsor

The University of Texas Health Science Center, Houston (Other)

Overall Status

Completed

CT.gov ID

NCT01652027

Collaborator

Rho, Inc. (Industry), Baxter Healthcare Corporation (Industry)

Enrollment

Locations

104

Actual Duration (Months)

1.5

Patients Per Site

Patients Per Site Per Month

Study Details

Study Description

Brief 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.

Condition or Disease	Intervention/Treatment	Phase
Hemophilia A	Drug: FVIII concentrate

Study Design

Study Type:

Observational

Actual Enrollment :

25 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

Study of Immunologic Determinants of Inhibitor Development in Previously Untreated Patients With Hemophilia

Actual Study Start Date :

Jul 1, 2011

Actual Primary Completion Date :

Mar 1, 2020

Actual Study Completion Date :

Mar 1, 2020

Arms and Interventions

Arm	Intervention/Treatment
Previously Untreated Patients with Hemophilia A	Drug: FVIII concentrate usual treatment as directed by treating physician

Arm

Intervention/Treatment

Previously Untreated Patients with Hemophilia A

Drug: FVIII concentrate

usual treatment as directed by treating physician

Outcome Measures

Primary Outcome Measures

Total number of FOXP3-positive T regulatory cells in the circulation [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.

Secondary Outcome Measures

FVIII-specific T-cells [50 exposure days to FVIII or 3 years, whichever comes first]
FVIII-specific T-cells will be compared before and after exposure to FVIII

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

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

Exclusion Criteria:

Prior exposure to clotting factor concentrates or blood products
Other chronic disease
Currently participating in another investigational drug study.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Emory University	Atlanta	Georgia	United States	30322
2	Indiana Hemophilia and Thrombosis Center	Indianapolis	Indiana	United States	46260
3	University of Kentucky	Lexington	Kentucky	United States	40536
4	Tulane University	New Orleans	Louisiana	United States	70112
5	Cornell University	New York	New York	United States	10065
6	University of North Carolina at Chapel Hill	Chapel Hill	North Carolina	United States	27599
7	Cincinnati Children's Hospital	Cincinnati	Ohio	United States	45229
8	University of Oregon	Portland	Oregon	United States
9	Hemophilia Center of Western Pennsylvania	Pittsburgh	Pennsylvania	United States	15213
10	North Texas Comprehensive Hemophilia Center	Dallas	Texas	United States	75235
11	Baylor College of Medicine	Houston	Texas	United States	77030
12	University of Texas Health Science Center-Houston	Houston	Texas	United States	77030
13	Intermountain Hemophilia and Thrombosis Center	Salt Lake City	Utah	United States	84108
14	Medical University of Vienna	Vienna		Austria	A-1090
15	Angelo Bianchi Bonomi Hemophilia & Thrombosis Center	Milan		Italy	20122
16	Emma Children's Hospital AMC	Amsterdam		Netherlands	1105
17	Malmo Centre for Thrombosis and Haemostasis	Malmo		Sweden	Se-205 02

Sponsors and Collaborators

The University of Texas Health Science Center, Houston
Rho, Inc.
Baxter Healthcare Corporation

Investigators

Principal Investigator: Elena Santagostino, M.D., Maggiore Hospital and University of Milan

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Deborah Brown, Associate Professor, The University of Texas Health Science Center, Houston

ClinicalTrials.gov Identifier:

NCT01652027

Other Study ID Numbers:

HSC-MS-11-0202
HSC-MS-11-0202

First Posted:

Jul 27, 2012

Last Update Posted:

Nov 10, 2020

Last Verified:

Nov 1, 2020

Keywords provided by Deborah Brown, Associate Professor, The University of Texas Health Science Center, Houston

FVIII inhibitors

Additional relevant MeSH terms:

Hemophilia A

Study Results

No Results Posted as of Nov 10, 2020