Congenital Bleeding DisorderPhase 3NCT00713648

Evaluation of Recombinant Factor XIII for Prevention of Bleeding in Patients With FXIII Inherited Deficiency

A Phase 3 study testing catridecacog in people with Congenital Bleeding Disorder and Congenital FXIII Deficiency, run by Novo Nordisk A/S at 35 sites in 11 countries.

Completed

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

Official record

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Trial summary

The trial is conducted in Europe, North America and Asia. The aim of this trial is to evaluate catridecacog (recombinant factor XIII (rFXIII)) treatment in patients with inherited FXIII deficiency. It is expected that recombinant FXIII can be used for the prevention of bleeding episodes. Read more about this study

Who is it for?: Ages 6 years and older
Conditions the study is for: Congenital Bleeding Disorder, Congenital FXIII Deficiency
What is being tested?: catridecacog
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 41 participants
Where: 35 sitesin 11 countries
Study dates: Aug 2008to Apr 2010
Phase: Phase 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 6 years and older
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Diagnosis of congenital FXIII A-subunit deficiency (confirmed by genotyping at screening visit)
Treatment with regular FXIII replacement therapy initiated at least 6 months prior to screening and one of the following : a documented history of at least one 1 treatment-requiring bleeding episode prior to initiation of regular replacement therapy or a documented family history of FXIII congenital deficiency (only for subjects on regular replacement therapy prior to screening)
Documented history of at least two 2 bleeding episodes requiring treatment with FXIII containing blood products within the last 12 months prior to screening (only for subjects receiving on-demand treatment prior to screening)

Who cannot join

Known neutralizing antibodies (inhibitors) towards FXIII
Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency
Documented history of at least 2 treatment-requiring bleeding episodes per year during previous regular replacement therapy with FXIII containing blood products (fresh frozen plasma (FFP), plasma-derived FXIII (pd FXIII) and cryoprecipitate)
Known or suspected allergy to trial product(s) or related products
Planned major surgery during the trial period. Catheter, ports and dental extractions do not count as surgeries and will not exclude the subject
Renal insufficiency defined as current dialysis therapy
Any history of confirmed venous or arterial thrombo-embolic events

Trial locations and contacts

35 sites in 11 countries.

Country

City, postcode or hospital

35 sites in 11 countries

United States11 sites

Novo Nordisk Investigational Site
Atlanta, Georgia, 30322
Novo Nordisk Investigational Site
Boston, Massachusetts, 02115
Novo Nordisk Investigational Site
Columbus, Ohio, 43205
Novo Nordisk Investigational Site
Detroit, Michigan, 48201
Novo Nordisk Investigational Site
East Lansing, Michigan, 48823
Novo Nordisk Investigational Site
Minneapolis, Minnesota, 55404
Novo Nordisk Investigational Site
Oklahoma City, Oklahoma, 73104
Novo Nordisk Investigational Site
Orange, California, 92868
Novo Nordisk Investigational Site
Phoenix, Arizona, 85016-7710
Novo Nordisk Investigational Site
Seattle, Washington, 98104
Novo Nordisk Investigational Site
Tampa, Florida, 33607

Other trials in United States

United Kingdom7 sites

Novo Nordisk Investigational Site
Aberdeen, AB25 2ZN
Novo Nordisk Investigational Site
Birmingham, B4 6NH
Novo Nordisk Investigational Site
Bradford, BD9 6RJ
Novo Nordisk Investigational Site
Bristol, BS2 8ED
Novo Nordisk Investigational Site
Liverpool, L12 2AP
Novo Nordisk Investigational Site
London, WC1N 3JH
Novo Nordisk Investigational Site
Newcastle upon Tyne, NE1 4LP

Other trials in United Kingdom

Austria3 sites

Novo Nordisk Investigational Site
Graz, 8036
Novo Nordisk Investigational Site
Klagenfurt, A-9020
Novo Nordisk Investigational Site
Vienna, 1090

Other trials in Austria

France3 sites

Novo Nordisk Investigational Site
Le Kremlin-Bicêtre, 94270
Novo Nordisk Investigational Site
Marseille, 13385
Novo Nordisk Investigational Site
Montpellier, 34295

Other trials in France

Germany3 sites

Novo Nordisk Investigational Site
Bonn, 53127
Novo Nordisk Investigational Site
Braunschweig, 38118
Novo Nordisk Investigational Site
Duisburg, 47051

Other trials in Germany

Israel2 sites

Novo Nordisk Investigational Site
Petah Tikva, 49100
Novo Nordisk Investigational Site
Tel HaShomer, 52621

Other trials in Israel

Spain2 sites

Novo Nordisk Investigational Site
Barcelona, 08035
Novo Nordisk Investigational Site
Seville, 41013

Other trials in Spain

Canada1 site

Novo Nordisk Investigational Site
Toronto, Ontario, M5G 1X8

Other trials in Canada

Finland1 site

Novo Nordisk Investigational Site
Helsinki, 00290

Other trials in Finland

Italy1 site

Novo Nordisk Investigational Site
Vicenza, 36100

Other trials in Italy

Switzerland1 site

Novo Nordisk Investigational Site
Zurich, 8091

Other trials in Switzerland

About this study

Official title: A Multi-Centre, Open-Label, Single-Arm and Multiple Dosing Trial on Efficacy and Safety of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Subjects With Congenital Factor XIII Deficiency

Conditions: Congenital Bleeding Disorder, Congenital FXIII Deficiency
Being tested: catridecacog

What participants receive

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

catridecacogDrug
35 IU/kg body weight, i.v. administration, once every 4 weeks

Study groups in NCT00713648
Study group	Role	What they receive
rFXIII	Experimental	Not described Receives: Drug: catridecacog

What is being measured

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

Rate (Number Per Subject Year) of Bleeding Episodes Requiring Treatment With a FXIII Containing Product During the Treatment Period
It represents the incidence of bleeding episodes requiring treatment with a FXIII-containing product.
Measured: For a period of 322 days (approximately one year) comprised of a screening visit (Visit 1), treatment period (Visits 2-15), unscheduled visit and end-of-trial visit (Visit 16).

Secondary outcomes (3)

Percentage of Subjects Having a Normal Clot Solubility One Hour After rFXIII Administration and 28 Days After rFXIII Administration for All Dosing Visits
Blood samples for clot solubility drawn at each visit (1 hour before and after dose administration). A clot solubility assay was used to screen for FXIII deficiency. The assay is based on the ability of urea to dissolve fibrin clots that have not undergone FXIII-induced stabilization. Normal blood clots generally remain stable for 24 hours or more, while clots in which fibrin molecules have not been cross-linked are soluble within minutes. The outcome of the test is normal (FXIII present; a clot is observed in the test tube) or abnormal (FXIII absent or very low level; no clot in test tube).
Measured: For a period of 322 days (approximately one year) comprised of a screening visit (Visit 1), treatment period (Visits 2-15), unscheduled visit and end-of-trial visit (Visit 16).
Level of FXIII Activity One Hour After rFXIII Administration and 28 Days After rFXIII Administration for All Dosing Visits
Subjects entered a 52-week treatment period of monthly (28±2 days) doses of 35 IU/kg rFXIII. Blood samples for analysis of FXIII activity were drawn at each visit; at dosing visits blood was drawn 1 hour after administration and before administration(corresponding to 28 days after the previous dose). All Dosing Visits are visits where a dose is given (i.e. Visit 2-15 except Visit 3).
Measured: For a period of 322 days (approximately one year) comprised of a screening visit (Visit 1), treatment period (Visits 2-15), unscheduled visit and end-of-trial visit (Visit 16).
Number of Subjects With rFXIII Antibody Development
Subjects receiving rFXIII were monitored for the development of binding antibodies. Blood sampling was done before administration of trial product at all visits (Visits 1-16 and unscheduled visit)
Measured: For a period of 322 days (approximately one year) comprised of a screening visit (Visit 1), treatment period (Visits 2-15), unscheduled visit and end-of-trial visit (Visit 16).

Timeline

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

Aug 2008Apr 2010

First posted11 Jul 2008 (estimated)
Study startAug 2008
Primary completionApr 2010
Study completionApr 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 Congenital Bleeding DisorderStudies of the same condition, open and under way.
More trials run by Novo Nordisk A/SThe sponsor's other registered studies.
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 3
Primary purpose: Prevention
Allocation: Non-randomised
Intervention model: Single group
Masking: None (open label)
Enrolment: 41 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00713648
Sponsor's protocol ID: F13CD-1725
Other IDs: 2006-003148-51
Status last verified: Jan 2017
First submitted: 7 Jul 2008
Last updated: 24 Feb 2017
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Publications

Inbal A, Oldenburg J, Carcao M, Rosholm A, Tehranchi R, Nugent D. Recombinant factor XIII: a safe and novel treatment for congenital factor XIII deficiency. Blood. 2012 May 31;119(22):5111-7. doi: 10.1182/blood-2011-10-386045. Epub 2012 Mar 26.
PubMed 22451421
Brand-Staufer B, Carcao M, Kerlin BA, Will A, Williams M, Tornoe CW, Sandberg Lundblad M, Nugent D. Pharmacokinetic characterization of recombinant factor XIII (FXIII)-A2 across age groups in patients with FXIII A-subunit congenital deficiency. Haemophilia. 2015 May;21(3):380-385. doi: 10.1111/hae.12616. Epub 2015 Jan 21.
PubMed 25643920

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

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All Congenital Bleeding Disorder trials

Study at a glance

From the registry record, last updated by the sponsor on 24 Feb 2017. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Multi-Centre, Open-Label, Single-Arm and Multiple Dosing Trial on Efficacy and Safety of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Subjects With Congenital Factor XIII Deficiency
ClinicalTrials.gov ID: NCT00713648
Acronym / sponsor ID: F13CD-1725
Conditions: Congenital Bleeding Disorder, Congenital FXIII Deficiency
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 41 participants (actual)
Lead sponsor: Novo Nordisk A/SIndustry (pharma, biotech, devices)
Study start: Aug 2008
Primary completion: Apr 2010 (actual)
Study completion: Apr 2010 (actual)
First posted: 11 Jul 2008 (estimated)
Results: Posted 14 Jul 2014

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: Non-randomised
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
rFXIII	Experimental	Drug: catridecacog

Who may qualify

Ages: 6 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (3 listed)

Diagnosis of congenital FXIII A-subunit deficiency (confirmed by genotyping at screening visit)
Treatment with regular FXIII replacement therapy initiated at least 6 months prior to screening and one of the following : a documented history of at least one 1 treatment-requiring bleeding episode prior to initiation of regular replacement therapy or a documented family history of FXIII congenital deficiency (only for subjects on regular replacement therapy prior to screening)
Documented history of at least two 2 bleeding episodes requiring treatment with FXIII containing blood products within the last 12 months prior to screening (only for subjects receiving on-demand treatment prior to screening)

Key exclusion criteria (7 listed)

Known neutralizing antibodies (inhibitors) towards FXIII
Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency
Documented history of at least 2 treatment-requiring bleeding episodes per year during previous regular replacement therapy with FXIII containing blood products (fresh frozen plasma (FFP), plasma-derived FXIII (pd FXIII) and cryoprecipitate)
Known or suspected allergy to trial product(s) or related products
Planned major surgery during the trial period. Catheter, ports and dental extractions do not count as surgeries and will not exclude the subject

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
catridecacog35 IU/kg body weight, i.v. administration, once every 4 weeks	Drug	rFXIII

Primary outcome

Rate (Number Per Subject Year) of Bleeding Episodes Requiring Treatment With a FXIII Containing Product During the Treatment Period
Time frame: For a period of 322 days (approximately one year) comprised of a screening visit (Visit 1), treatment period (Visits 2-15), unscheduled visit and end-of-trial visit (Visit 16).
It represents the incidence of bleeding episodes requiring treatment with a FXIII-containing product.

3 secondary outcomes

Percentage of Subjects Having a Normal Clot Solubility One Hour After rFXIII Administration and 28 Days After rFXIII Administration for All Dosing Visits
Time frame: For a period of 322 days (approximately one year) comprised of a screening visit (Visit 1), treatment period (Visits 2-15), unscheduled visit and end-of-trial visit (Visit 16).
Level of FXIII Activity One Hour After rFXIII Administration and 28 Days After rFXIII Administration for All Dosing Visits
Time frame: For a period of 322 days (approximately one year) comprised of a screening visit (Visit 1), treatment period (Visits 2-15), unscheduled visit and end-of-trial visit (Visit 16).
Number of Subjects With rFXIII Antibody Development
Time frame: For a period of 322 days (approximately one year) comprised of a screening visit (Visit 1), treatment period (Visits 2-15), unscheduled visit and end-of-trial visit (Visit 16).

Results and safety

Results posted to the registry on 14 Jul 2014, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
rFXIII	41	33

Primary outcome: Rate (Number Per Subject Year) of Bleeding Episodes Requiring Treatment With a FXIII Containing Product During the Treatment Period

Time frame: For a period of 322 days (approximately one year) comprised of a screening visit (Visit 1), treatment period (Visits 2-15), unscheduled visit and end-of-trial visit (Visit 16).. Mean (Full Range). Unit: bleeding episodes per subject per year

Measure	rFXIII n = 41
Value	0.138 (0 to 2)

rFXIII: Mean (Lambda): 0.048 (95% CI 0.0094 to 0.2501), Poisson model (log-link)

Population analysed: Full analysis set - consisting of a total of 41 subjects who received at least one dose of trial product.

3 secondary outcomes are reported in the full results.

Adverse events

Time frame: Adverse events (AEs) were collected and reported during the entire study period i.e. approximately one year (322 days). Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
rFXIII	6 of 41 (14.6%)	25 of 41 (61%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 24 Feb 2017.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Results publication: PubMed 22451421Inbal A, Oldenburg J, Carcao M, Rosholm A, Tehranchi R, Nugent D. Recombinant factor XIII: a safe and novel treatment for congenital factor XIII deficiency. Blood. 2012 May 31;119(22):5111-7. doi: 10.1182/blood-2011-10-386045. Epub 2012 Mar 26.
Results publication: PubMed 25643920Brand-Staufer B, Carcao M, Kerlin BA, Will A, Williams M, Tornoe CW, Sandberg Lundblad M, Nugent D. Pharmacokinetic characterization of recombinant factor XIII (FXIII)-A2 across age groups in patients with FXIII A-subunit congenital deficiency. Haemophilia. 2015 May;21(3):380-385. doi: 10.1111/hae.12616. Epub 2015 Jan 21.
Clinical Trials at Novo NordiskLink provided by the sponsor.

Related trials for Congenital Bleeding Disorder

RecruitingObservational study
A Study in Children and, Adults With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma
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Not yet recruitingPhase 1
Topical TOR-582 Treatment of Epistaxis in HHT
Hereditary Hemorrhagic Telangiectasia (HHT), Epistaxis
RecruitingPhase 1/2
Harmony-HHT: ATV-1601 in Participants With Hereditary Hemorrhagic Telangiectasia (HHT)
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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.

Global Clinical Registry (GCR, 1452) study director

Novo Nordisk A/S

35 sites in 11 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing catridecacog in people with Congenital Bleeding Disorder and Congenital FXIII Deficiency, run by Novo Nordisk A/S at 35 sites in 11 countries.

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 3 inclusion and 7 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 24 Feb 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 35 sites in United States, United Kingdom, Austria, France, Germany and 6 more countries. 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 phase mean?

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 14 Jul 2014). 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?

Rate (Number Per Subject Year) of Bleeding Episodes Requiring Treatment With a FXIII Containing Product During the Treatment Period (time frame: For a period of 322 days (approximately one year) comprised of a screening visit (Visit 1), treatment period (Visits 2-15), unscheduled visit and end-of-trial visit (Visit 16).). 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 NCT00713648, 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 24 Feb 2017.