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Hemophilia Inhibitor Previously Untreated Patient Study (HIPS)

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ClinicalTrials.gov Identifier: NCT01652027
Recruitment Status : Completed
First Posted : July 27, 2012
Last Update Posted : November 10, 2020
Sponsor:
Collaborators:
Rho, Inc.
Baxter Healthcare Corporation
Information provided by (Responsible Party):
Deborah Brown, The University of Texas Health Science Center, Houston

Tracking Information
First Submitted Date June 3, 2011
First Posted Date July 27, 2012
Last Update Posted Date November 10, 2020
Actual Study Start Date July 2011
Actual Primary Completion Date March 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: June 17, 2014)
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.
Original Primary Outcome Measures
 (submitted: July 26, 2012)
Total number of FOXP3-positive T regulatory cells in the circulation [ Time Frame: First 50 exposures to FVIII ( 7-9 days after the first treatment, and (5 +/- 2)days after the 5th, 10th, 20th, 30th, 40th, and 50th days of treatment) ]
FoxP3-positive T regulatory cells in the circulation will be quantified before and after exposure to FVIII.
Change History
Current Secondary Outcome Measures
 (submitted: February 6, 2013)
FVIII-specific T-cells [ Time Frame: 50 exposure days to FVIII or 3 years, whichever comes first ]
FVIII-specific T-cells will be compared before and after exposure to FVIII
Original Secondary Outcome Measures
 (submitted: July 26, 2012)
FVIII-specific T-cells [ Time Frame: First 50 exposures to FVIII ( 7-9 days after the first treatment, and (5 +/- 2)days after the 5th, 10th, 20th, 30th, 40th, and 50th days of treatment) ]
FVIII-specific T-cells will be analyzed before and after exposure to FVIII
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Hemophilia Inhibitor Previously Untreated Patient Study
Official Title Study of Immunologic Determinants of Inhibitor Development in Previously Untreated Patients With Hemophilia
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.
Detailed Description Not Provided
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Retention:   Samples With DNA
Description:
Plasma samples, lymphocyte cell lines, Genomic DNA, RNA
Sampling Method Non-Probability Sample
Study Population Patients with severe hemophilia A who have not previously been treated with Factor VIII concentrates.
Condition Hemophilia A
Intervention Drug: FVIII concentrate
usual treatment as directed by treating physician
Study Groups/Cohorts Previously Untreated Patients with Hemophilia A
Intervention: Drug: FVIII concentrate
Publications * 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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Completed
Actual Enrollment
 (submitted: October 30, 2017)
25
Original Estimated Enrollment
 (submitted: July 26, 2012)
50
Actual Study Completion Date March 2020
Actual Primary Completion Date March 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria

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.
Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries Austria,   Italy,   Netherlands,   Sweden,   United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT01652027
Other Study ID Numbers HSC-MS-11-0202
HSC-MS-11-0202 ( Other Identifier: University of Texas HSC-Houston Committee for the Protection of Human Subjects )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Deborah Brown, The University of Texas Health Science Center, Houston
Study Sponsor The University of Texas Health Science Center, Houston
Collaborators
  • Rho, Inc.
  • Baxter Healthcare Corporation
Investigators
Principal Investigator: Elena Santagostino, M.D. Maggiore Hospital and University of Milan
PRS Account The University of Texas Health Science Center, Houston
Verification Date November 2020