Hemophilia A With InhibitorsPhase 2/3ProFEIBANCT00221195

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

A Phase 2/3 study testing activated prothrombin complex concentrate (FEIBA) in people with Hemophilia A With Inhibitors, run by Tulane University School of Medicine at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial 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. Read more about this study

Who is it for?: Ages 24 months and older
Conditions the study is for: Hemophilia A With Inhibitors
What is being tested?: activated prothrombin complex concentrate (FEIBA)
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 34 participants
Where: 1 siteUnited States
Study dates: Jun 2003to Jul 2010
Phase: Phase 2/3
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 24 months and older
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

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

Who cannot join

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

Trial locations and contacts

1 site in 1 country.

United States1 site

Tulane University School of Medicine
New Orleans, Louisiana, 70112

Other trials in United States

About this study

Official title: A Prospective, Randomized, Cross-over Study of an Activated Prothrombin Complex Concentrate for Secondary Prophylaxis in Patients With Hemophilia A and Inhibitors

Conditions: Hemophilia A With Inhibitors
Being tested: activated prothrombin complex concentrate (FEIBA)

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

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

Study groups in NCT00221195
Study group	Role	What they receive
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 Receives: 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 Receives: Drug: activated prothrombin complex concentrate (FEIBA)

What is being measured

The primary outcome is the main question the study is designed to answer.

Number of Bleeds During 6 Month Treatment Period
Measured: 6 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Jun 2003Jul 2010

First posted22 Sep 2005 (estimated)
Study startJun 2003
Primary completionDec 2009
Study completionJul 2010

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (2)Papers the sponsor has linked to this study.
Other trials for Hemophilia A With InhibitorsStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2/3
Primary purpose: Prevention
Allocation: Randomised
Intervention model: Crossover
Masking: None (open label)
Enrolment: 34 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00221195
Sponsor's protocol ID: PRO-FEIBA Study
Acronym: ProFEIBA
Status last verified: Feb 2020
First submitted: 19 Sep 2005
Last updated: 2 Mar 2020
Results posted: Yes, on ClinicalTrials.gov

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.
PubMed 22047559

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 3
Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
Hemophilia A, Hemophilia B
RecruitingPhase 3
A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A
Hemophilia A
Not yet recruitingObservational study
A Study to Describe Patient REal-world FitusiRan ExpeRience
Hemophilia A or B
Active, not recruitingPhase 4
A Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes in Participants With Severe or Moderate Hemophilia A Without Factor VIII Inhibitors on Emicizumab Prophylaxis
Severe Hemophilia A, Moderate Hemophilia A
Not yet recruitingPhase 3
Clinical Trial of NXT007 for the Prophylactic Treatment of Hemophilia A in Infants and Children
Hemophilia A

All Hemophilia A With Inhibitors trials

Study at a glance

From the registry record, last updated by the sponsor on 2 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Prospective, Randomized, Cross-over Study of an Activated Prothrombin Complex Concentrate for Secondary Prophylaxis in Patients With Hemophilia A and Inhibitors
ClinicalTrials.gov ID: NCT00221195
Acronym / sponsor ID: ProFEIBA / PRO-FEIBA Study
Conditions: Hemophilia A With Inhibitors
Phase: Phase 2/3
Study type: Interventional
Recruitment status: Completed
Enrolment: 34 participants (actual)
Lead sponsor: Tulane University School of MedicineUniversity, hospital or other
Study start: Jun 2003
Primary completion: Dec 2009 (actual)
Study completion: Jul 2010 (actual)
First posted: 22 Sep 2005 (estimated)
Results: Posted 6 Feb 2017

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Prevention
Allocation: Randomised
Intervention model: Crossover
Masking: None (open label)
Number of arms: 2

Arm	Type	Receives
On-demand first	Other	Drug: activated prothrombin complex concentrate (FEIBA)Patients receive 6 months of on-demand therapy with study drug followed by 6 months of prophylaxis therapy with study drug
Prophylaxis first	Other	Drug: activated prothrombin complex concentrate (FEIBA)Patients receive 6 months of prophylaxis therapy with study drug followed by 6 months on-demand therapy with study drug

Who may qualify

Ages: 24 months and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (1 listed)

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

Key exclusion criteria (1 listed)

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

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
activated 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	Drug	On-demand first, Prophylaxis first

Primary outcome

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

Results and safety

Results posted to the registry on 6 Feb 2017, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
On-demand First	17	14
Prophylaxis First	17	15

Primary outcome: Number of Bleeds During 6 Month Treatment Period

Time frame: 6 months. Mean (Standard Deviation). Unit: bleeds

Measure	Bleeds During the On-demand Period n = 26	Bleeds During the Prophylaxis Period n = 26
Value	13.1 (7.1)	5.0 (5.0)

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

Adverse events

Time frame: Adverse events were collected from time of consent to the end of the study, which totaled 15 months.. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
On-demand Period	3 of 31 (9.7%)	16 of 31 (51.6%)	Not reported
Prophylaxis Period	4 of 31 (12.9%)	17 of 31 (54.8%)	Not reported
Washout Period	4 of 29 (13.8%)	14 of 29 (48.3%)	Not reported

Limitations and caveats (from the sponsor): 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.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 2 Mar 2020.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 23731246Gringeri 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.
Derived from this study: PubMed 22047559Leissinger 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.

Related trials for Hemophilia A With Inhibitors

RecruitingPhase 3
Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
Hemophilia A, Hemophilia B
RecruitingPhase 3
A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A
Hemophilia A
Not yet recruitingObservational study
A Study to Describe Patient REal-world FitusiRan ExpeRience
Hemophilia A or B
Active, not recruitingPhase 4
A Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes in Participants With Severe or Moderate Hemophilia A Without Factor VIII Inhibitors on Emicizumab Prophylaxis
Severe Hemophilia A, Moderate Hemophilia A
Not yet recruitingPhase 3
Clinical Trial of NXT007 for the Prophylactic Treatment of Hemophilia A in Infants and Children
Hemophilia A

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Cindy A Leissinger principal investigator

Tulane University School of Medicine

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing activated prothrombin complex concentrate (FEIBA) in people with Hemophilia A With Inhibitors, run by Tulane University School of Medicine at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 2 Mar 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 6 Feb 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Bleeds During 6 Month Treatment Period (time frame: 6 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00221195, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Mar 2020.