CompletedNCT00221195ProFEIBAUpdated Mar 2, 2020Results posted

Efficacy Study of Activated Prothrombin Complex for Prevention of Bleeds in Hemophilia A With Inhibitors

A Phase 2/3 interventional study of activated prothrombin complex concentrate (FEIBA) in Hemophilia A With Inhibitors, sponsored by Tulane University School of Medicine. Completed at 1 site in United States. Open to participants aged 24 Months and older. Per ClinicalTrials.gov, last updated 2020-03-02.

Sponsored by Tulane University School of Medicine · Phase 2/3, Interventional, and Prevention

Phase: Phase 2/3
Study type: Interventional
Enrollment: 34
Allocation: Randomized
Ages: 24 Months and older
Sex: All

Study 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.

Conditions studied

Hemophilia A With Inhibitors

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Hemophilia A 866

In context

Hemophilia A

866 studies on the registry are indexed under Hemophilia A; 137 are open to participants now.

This study's enrollment of 34 is above the median of 28 across 512 interventional studies indexed under Hemophilia A.

Browse Hemophilia A studies →

Lead sponsor

Tulane University School of Medicine is the lead sponsor of 20 studies on the registry; 1 is open to participants now.

Counted across the registry records on this site, refreshed daily.

Who can participate

Ages eligible: 24 Months and older
Sexes eligible: All
Accepts healthy volunteers: No

Inclusion criteria

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

Exclusion criteria

Exclusion Criteria:

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

Study design

Phase: Phase 2 / Phase 3
Primary purpose: Prevention
Allocation: Randomized
Intervention model: Crossover assignment
Masking: None (open label)
Enrollment: 34 participants (actual)

Study arms

Other
On-demand first
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)
Other
Prophylaxis first
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

Drugactivated prothrombin complex concentrate (FEIBA)
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

What researchers measure

Primary outcomes

Number of Bleeds During 6 Month Treatment Period
Time frame: 6 months

Results

Posted Feb 6, 2017

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.

Participant flow

The study was conducted at 16 hemophilia treatment centers in Europe and the United States.

Therapy 1 (Months 1-6)

Participant flow — Therapy 1 (Months 1-6)
Milestone	On-demand First	Prophylaxis First
Started	17	17
Completed	14	15
Not completed	3	2
Withdrew: Withdrawal by subject	3	1
Withdrew: Lost to follow-up	0	1

Washout Period (Months 7-9)

Participant flow — Washout Period (Months 7-9)
Milestone	On-demand First	Prophylaxis First
Started	14	15
Completed	14	14
Not completed	0	1
Withdrew: Death	0	1

Therapy 2 (Months 10-15)

Participant flow — Therapy 2 (Months 10-15)
Milestone	On-demand First	Prophylaxis First
Started	14	14
Completed	12	14
Not completed	2	0
Withdrew: Death	1	0
Withdrew: Adverse event	1	0

Outcome measures

PrimaryNumber of Bleeds During 6 Month Treatment Period

Time frame:: 6 months
Reported as:: Mean · bleeds

Number of Bleeds During 6 Month Treatment Period
bleeds	Bleeds During the On-demand Period	Bleeds During the Prophylaxis Period
Number of Bleeds During 6 Month Treatment Period	13.1 ± 7.1	5.0 ± 5.0

Statistical analysis

Bleeds During the On-demand Period vs Bleeds During the Prophylaxis Period · Wilcoxon (Mann-Whitney) · p = <0.001

Adverse events

Collected over Adverse events were collected from time of consent to the end of the study, which totaled 15 months.. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
Group	Deaths	Serious	Other
On-demand Period	—	3/31 (9.7%)	16/31 (51.6%)
Prophylaxis Period	—	4/31 (12.9%)	17/31 (54.8%)
Washout Period	—	4/29 (13.8%)	14/29 (48.3%)

Most frequent serious events

Showing 10 of 14

Most frequent serious events
Event	On-demand Period	Prophylaxis Period	Washout Period
Muscle HemorrhageMusculoskeletal and connective tissue disorders	0/31	0/31	2/29
Catheter-site InfectionInfections and infestations	1/31	2/31	0/29
Cerebral HemorrhageVascular disorders	0/31	0/31	1/29
Subdural HematomaVascular disorders	0/31	0/31	1/29
Chest PainCardiac disorders	0/31	1/31	0/29
PainMusculoskeletal and connective tissue disorders	1/31	0/31	0/29
Drug hypersensitivityProduct Issues	0/31	1/31	0/29
PhlebitisBlood and lymphatic system disorders	0/31	1/31	0/29
HospitalizationEndocrine disorders	1/31	0/31	0/29
Surgical ProcedureSurgical and medical procedures	1/31	0/31	0/29

Most frequent other events

Showing 10 of 18

Most frequent other events
Event	On-demand Period	Prophylaxis Period	Washout Period
PyrexiaGeneral disorders	2/31	6/31	1/29
InfluenzaInfections and infestations	1/31	1/31	4/29
PainMusculoskeletal and connective tissue disorders	3/31	2/31	0/29
EcchymosisBlood and lymphatic system disorders	2/31	3/31	1/29
CoughRespiratory, thoracic and mediastinal disorders	0/31	3/31	2/29
NasopharyngitisRespiratory, thoracic and mediastinal disorders	1/31	2/31	2/29
HypersensitivityImmune system disorders	2/31	0/31	0/29
PharyngitisRespiratory, thoracic and mediastinal disorders	1/31	2/31	0/29
VomitingGastrointestinal disorders	1/31	2/31	1/29
Catheter-site HemorrhageBlood and lymphatic system disorders	1/31	2/31	0/29

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)	On-demand First	Prophylaxis First	Total
<=18 years	4	7	11
Between 18 and 65 years	13	9	22
>=65 years	0	1	1

Age, Continuous
Age, Continuous(years)	On-demand First	Prophylaxis First	Total
Mean	29.3 ± 13.4	24.9 ± 20.6	27.1 ± 17.3

Sex: Female, Male
Sex: Female, Male(Participants)	On-demand First	Prophylaxis First	Total
Female	0	0	0
Male	17	17	34

Region of Enrollment
Region of Enrollment(participants)	On-demand First	Prophylaxis First	Total
United States	3	3	6
Europe	14	14	28

Study locations

1 site

Tulane University School of Medicine
New Orleans, Louisiana 70112, United States

References and documents

Publications

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. PubMed 23731246 ↗
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. Erratum In: N Engl J Med. 2011 Dec 22;365(25):2441. PubMed 22047559 ↗

Updates

Tracking since Sep 25, 2026

No changes since tracking began. The registry record was last updated on Mar 2, 2020, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗

Registry details

Key details

Study ID: NCT00221195
Lead sponsor: Tulane University School of Medicine
Responsible party: Sponsor
First posted: Sep 22, 2005
Start date: Jun 2003
Primary completion: Dec 2009
Completion: Jul 2010
Results posted: Feb 6, 2017
Last update: Mar 2, 2020

Study contacts

Cindy A Leissinger

principal investigator · Tulane University School of Medicine

View the source record on ClinicalTrials.gov ↗

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