NCT00221195

Brief Summary

The objective of this study is to assess whether prophylactic therapy with an activated prothrombin complex concentrate (FEIBA)will result in a significant reduction in the number of bleeds in patients with hemophilia and persistent high responding inhibitors.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
34

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Jun 2003

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 1, 2003

Completed
2.3 years until next milestone

First Submitted

Initial submission to the registry

September 19, 2005

Completed
3 days until next milestone

First Posted

Study publicly available on registry

September 22, 2005

Completed
4.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2009

Completed
7 months until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2010

Completed
6.6 years until next milestone

Results Posted

Study results publicly available

February 6, 2017

Completed
Last Updated

March 2, 2020

Status Verified

February 1, 2020

Enrollment Period

6.5 years

First QC Date

September 19, 2005

Results QC Date

December 12, 2016

Last Update Submit

February 13, 2020

Conditions

Hemophilia A With Inhibitors

Outcome Measures

Primary Outcomes (1)

  • Number of Bleeds During 6 Month Treatment Period

    6 months

Study Arms (2)

On-demand first

OTHER

Patients receive 6 months of on-demand therapy with study drug followed by 6 months of prophylaxis therapy with study drug

Drug: activated prothrombin complex concentrate (FEIBA)

Prophylaxis first

OTHER

Patients receive 6 months of prophylaxis therapy with study drug followed by 6 months on-demand therapy with study drug

Drug: activated prothrombin complex concentrate (FEIBA)

Interventions

activated prothrombin complex concentrate (FEIBA)DRUG

FEIBA for prophylaxis therapy dosed at 85 U/Kg +/- 15% on three non-consecutive days each week for 6 months FEIBA for on-demand therapy dosed at 85 U/Kg +/- 15% for bleeding episodes for 6 months

On-demand firstProphylaxis first

Eligibility Criteria

Age24 Months+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • hemophilia A, any severity, with documented history of high-titer inhibitor (\>5BU); current use of bypassing agents (PCCs, aPCCs or rFVIIa) for treatment of bleeds; \>/= 6 bleeds requiring bypassing therapy in the previous 6 months

You may not qualify if:

  • concomitant immune tolerance therapy; clinically symptomatic liver disease, platelet count less than 100,000

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Tulane University School of Medicine

New Orleans, Louisiana, 70112, United States

Location

Related Publications (2)

  • Gringeri A, Leissinger C, Cortesi PA, Jo H, Fusco F, Riva S, Antmen B, Berntorp E, Biasoli C, Carpenter S, Kavakli K, Morfini M, Negrier C, Rocino A, Schramm W, Windyga J, Zulfikar B, Mantovani LG. Health-related quality of life in patients with haemophilia and inhibitors on prophylaxis with anti-inhibitor complex concentrate: results from the Pro-FEIBA study. Haemophilia. 2013 Sep;19(5):736-43. doi: 10.1111/hae.12178. Epub 2013 Jun 4.

    PMID: 23731246DERIVED

  • Leissinger C, Gringeri A, Antmen B, Berntorp E, Biasoli C, Carpenter S, Cortesi P, Jo H, Kavakli K, Lassila R, Morfini M, Negrier C, Rocino A, Schramm W, Serban M, Uscatescu MV, Windyga J, Zulfikar B, Mantovani L. Anti-inhibitor coagulant complex prophylaxis in hemophilia with inhibitors. N Engl J Med. 2011 Nov 3;365(18):1684-92. doi: 10.1056/NEJMoa1104435.

    PMID: 22047559DERIVED

MeSH Terms

Interventions

anti-inhibitor coagulant complex

Limitations and Caveats

One limitation was the relatively short duration; in addition, due to the small number of children enrolled, it is not possible to draw conclusions regarding the relationship of age and the benefits of prophylaxis.

Results Point of Contact

Title
Cindy Leissinger
Organization
Tulane University
cleissi@tulane.edu
504-988-5433

Study Officials

  • Cindy A Leissinger

    Tulane University School of Medicine

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
LTE60
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
NONE
Purpose
PREVENTION
Intervention Model
CROSSOVER
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 19, 2005

First Posted

September 22, 2005

Study Start

June 1, 2003

Primary Completion

December 1, 2009

Study Completion

July 1, 2010

Last Updated

March 2, 2020

Results First Posted

February 6, 2017

Record last verified: 2020-02

Locations

US(1)