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Hemophilia Inhibitor Previously Untreated Patient Study

NCT ID: NCT01652027

Last Updated: 2020-11-10

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

25 participants

Study Classification

OBSERVATIONAL

Study Start Date

2011-07-31

Study Completion Date

2020-03-31

Brief Summary

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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.

Detailed Description

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Conditions

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Hemophilia A

Keywords

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FVIII inhibitors

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Previously Untreated Patients with Hemophilia A

FVIII concentrate

Intervention Type DRUG

usual treatment as directed by treating physician

Interventions

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FVIII concentrate

usual treatment as directed by treating physician

Intervention Type DRUG

Eligibility Criteria

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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.
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Rho, Inc.

INDUSTRY

Sponsor Role collaborator

Baxter Healthcare Corporation

INDUSTRY

Sponsor Role collaborator

The University of Texas Health Science Center, Houston

OTHER

Sponsor Role lead

Responsible Party

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Deborah Brown

Associate Professor

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Elena Santagostino, M.D.

Role: PRINCIPAL_INVESTIGATOR

Maggiore Hospital and University of Milan

Locations

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Emory University

Atlanta, Georgia, United States

Site Status

Indiana Hemophilia and Thrombosis Center

Indianapolis, Indiana, United States

Site Status

University of Kentucky

Lexington, Kentucky, United States

Site Status

Tulane University

New Orleans, Louisiana, United States

Site Status

Cornell University

New York, New York, United States

Site Status

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States

Site Status

Cincinnati Children's Hospital

Cincinnati, Ohio, United States

Site Status

University of Oregon

Portland, Oregon, United States

Site Status

Hemophilia Center of Western Pennsylvania

Pittsburgh, Pennsylvania, United States

Site Status

North Texas Comprehensive Hemophilia Center

Dallas, Texas, United States

Site Status

Baylor College of Medicine

Houston, Texas, United States

Site Status

University of Texas Health Science Center-Houston

Houston, Texas, United States

Site Status

Intermountain Hemophilia and Thrombosis Center

Salt Lake City, Utah, United States

Site Status

Medical University of Vienna

Vienna, , Austria

Site Status

Angelo Bianchi Bonomi Hemophilia & Thrombosis Center

Milan, , Italy

Site Status

Emma Children's Hospital AMC

Amsterdam, , Netherlands

Site Status

Malmo Centre for Thrombosis and Haemostasis

Malmo, , Sweden

Site Status

Countries

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United States Austria Italy Netherlands Sweden

References

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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.

Reference Type DERIVED
PMID: 36074992 (View on PubMed)

Reipert 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.

Reference Type DERIVED
PMID: 33232473 (View on PubMed)

Other Identifiers

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HSC-MS-11-0202

Identifier Type: OTHER

Identifier Source: secondary_id

HSC-MS-11-0202

Identifier Type: -

Identifier Source: org_study_id