Congenital Bleeding DisorderPhase 3mentor™5NCT01253811

Safety and Efficacy of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Paediatric Subjects With Congenital Factor XIII A-subunit Deficiency

A Phase 3 study testing catridecacog in people with Congenital Bleeding Disorder and Congenital FXIII Deficiency, run by Novo Nordisk A/S at 4 sites in 3 countries.

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

This trial will be conducted in Asia, Europe and the United States of America (USA). The aim of this clinical trial is to investigate long-term safety of rFXIII when administered for prevention of bleeding episodes in children aged between 1 and 6 years with congenital FXIII A-subunit deficiency. Read more about this study

Who is it for?: Ages 1 year to 6 years
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: 6 participants
Where: 4 sitesin 3 countries
Study dates: Jan 2011to Mar 2015
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 1 year to 6 years
All sexes
No healthy volunteers
Child

Who may be able to join

Completed participation in trial F13CD-3760 (NCT01230021)

Who cannot join

Known or suspected hypersensitivity to trial product or related products
Known history of development of inhibitors against FXIII (factor XIII)
Hereditary or acquired coagulation disorder other than FXIII congenital deficiency
Platelet count (thrombocytes) less than 50X10e9 / L
Previous history of autoimmune disorder involving autoantibodies e.g., systemic lupus erythematosus
Previous history of arterial or venous thromboembolic events e.g., cerebrovascular accident or deep vein thrombosis
Any disease or condition which, judged by the trial physician, could imply a potential hazard to the subject, interfere with the trial participation or trial outcome including renal and/or liver dysfunction

Trial locations and contacts

4 sites in 3 countries.

Country

City, postcode or hospital

4 sites in 3 countries

United States2 sites

Novo Nordisk Clinical Trial Call Center
Boston, Massachusetts, 02115
Novo Nordisk Clinical Trial Call Center
Columbus, Ohio, 43205

Other trials in United States

Israel1 site

Study site
Petah Tikva, 49100

Other trials in Israel

United Kingdom1 site

Study site
Leicester, LE1 5WW

Other trials in United Kingdom

About this study

Official title: A Multi-Centre, Multinational, Open-Label, Single-Arm and Multiple Dosing Trial on Safety and Efficacy of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Paediatric Subjects With Congenital Factor XIII A-subunit Deficiency. Safety Extension Trial to F13CD-3760

This trial will be conducted in Asia, Europe and the United States of America (USA).

The aim of this clinical trial is to investigate long-term safety of rFXIII when administered for prevention of bleeding episodes in children aged between 1 and 6 years with congenital FXIII A-subunit deficiency. This trial is an extension to trial F13CD-3760 (mentor™4, NCT01230021). If applicable the trial will be extended up to maximum 3 years dependent on when recombinant factor XIII will be commercially available in subject's respective country for use in children of 1-6 years of age.

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
Also called recombinant factor XIII
Intravenous injection of a single dose of recombinant factor XIII, 35 IU/kg body weight every 4th week

Study groups in NCT01253811
Study group	Role	What they receive
rFXIII 35 IU/kg	Experimental	Not described Receives: Drug: catridecacog

What is being measured

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

Number of Treatment Emergent (Serious and Non-serious) Adverse Events
An adverse event was described as any untoward medical occurrence in a subject or clinical investigation subject administered a pharmaceutical product, and which does not necessarily have a causal relationship with this treatment. Treatment emergent adverse events (serious and non-serious), defined as adverse events occurring from first trial product administration to the end of the subject's participation in the trial.
Measured: Week 0 to end of trial visit (week 173) for a minimum period of 52 weeks.

Secondary outcomes (15)

Percentage of Subjects With Development of Anti-rFXIII Antibodies, Including Inhibitors.
All subjects who received rFXIII were monitored for the frequency of development of anti-rFXIII antibodies. Samples passed through 2 tiers of ELISA testing: an initial screen with a specific cut-off point (including ~5% false positives) and a second confirmatory assay for samples yielding a result above the screening cut-off point. If samples were confirmed as antibody positive in the confirmation assay, an inhibitor assay was also carried out to detect functional inhibitors.
Measured: Week 0 to end of trial visit (week 173).
Clinical Laboratory Assessments: Biochemistry: Creatinine
Clinical laboratory assessments for creatinine at week 24 to end of trial visit.
Measured: Every 6th month, from week 24 to end of trial visit (week 173).
Clinical Laboratory Assessments: Biochemistry: Urea
Clinical laboratory assessments for urea at week 24 to end ot trial visit.
Measured: Every 6th month, week 24 to end of trial visit (week 173).
Clinical Laboratory Assessments: Biochemistry: Alanine Aminotransferase (ALAT)
Clinical laboratory assessments for ALAT at week 24 to end of trial visit.
Measured: Every 6th month, from week 24 to end of trial visit (week 173).
Clinical Laboratory Assessments: Biochemistry: Aspartate Aminotransferase (ASAT)
Clinical laboratory assessments for ASAT at week 24 to end of trial visit.
Measured: Every 6th month, from week 24 to end of trial visit (week 173).
Clinical Laboratory Assessments: Haematology: Haemoglobin
Clinical values for haemoglobin collected from week 0 to end of trial visit.
Measured: Every 6th month, from week 0 to end of trial visit (week 173).
Clinical Laboratory Assessments: Haematology: Leucocytes
Clinical laboratory values for leucocytes collected from week 0 to end of trial visit.
Measured: Every 6th month, from week 0 to end of trial visit (week 173).
Clinical Laboratory Assessments: Haematology: Thrombocytes
Clinical laboratory values for thrombocytes collected from week 0 to end of trial visit.
Measured: Every 6th month, from week 0 to end of trial visit (week 173).
Clinical Laboratory Assessments: Haematology: Erythrocytes
Clinical laboratory values for erythrocytes collected from week 0 to end of trial visit.
Measured: Every 6th month, from week 0 to end of trial visit (week 173).
Clinical Laboratory Assessments: Haematology: Haematocrit
Clinical laboratory values for haematocrit collected from week 0 to end of trial visit.
Measured: Every 6th month, from week 0 to end of trial visit (week 173).
Physical Examinations
Number of subjects in percentage with changes in values of physical examinations from week 0 to end of trial visit were collected.
Measured: Week 0 to end of trial visit (week 173).
Vital Signs: Systolic BP (Blood Pressure)
Values collected for systolic BP from week 0 to end of trial visit.
Measured: Week 0 to end of trial visit (week 173).
Vital Signs: Diastolic BP (Blood Pressure)
Values collected for diastolic BP from week 0 to end of trial visit.
Measured: Week 0 to end of trial visit (week 173).
Vital Signs: Pulse
Values collected for pulse from week 0 to end of trial visit.
Measured: Week 0 to end of trial visit (week 173).
Rate (Number Per Subject Year) of All Bleeding Episodes Requiring Treatment With a FXIII Containing Product Other Than Recombinant Factor XIII.
The rate (number per subject year) of all spontaneous, traumatic and intracranial bleeding episodes requiring treatment with FXIII-containing products during the rFXIII treatment period was assessed for treatment period.
Measured: Weeks 0 to end of trial visit (week 173).

Timeline

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

Jan 2011Mar 2015

First posted3 Dec 2010 (estimated)
Study startJan 2011
Primary completionMar 2015
Study completionMar 2015

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 (1)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: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 6 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01253811
Sponsor's protocol ID: F13CD-3835
Other IDs: U1111-1117-1063, 2010-020192-23
Acronym: mentor™5
Status last verified: May 2016
First submitted: 1 Dec 2010
Last updated: 24 Jun 2016
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Publications

Kerlin BA, Inbal A, Will A, Williams M, Garly ML, Jacobsen L, Kearney SL. Recombinant factor XIII prophylaxis is safe and effective in young children with congenital factor XIII-A deficiency: international phase 3b trial results. J Thromb Haemost. 2017 Aug;15(8):1601-1606. doi: 10.1111/jth.13748. Epub 2017 Jul 10.
PubMed 28581691

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

RecruitingObservational study
A Study in Children and, Adults With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma
Thrombotic Thrombocytopenic Purpura (TTP)
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)
Hereditary Hemorrhagic Telangiectasia (HHT), Osler Weber Rendu Disease
RecruitingObservational study
The CACHE Study: Coronary Artery Care in HaEmophilia
Haemophilia, Cardiovascular Diseases
Recruiting
Optimization of Saturation Targets And Resuscitation Trial (OptiSTART)
Premature Infants, Bronchopulmonary Dysplasia

All Congenital Bleeding Disorder trials

Study at a glance

From the registry record, last updated by the sponsor on 24 Jun 2016. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Multi-Centre, Multinational, Open-Label, Single-Arm and Multiple Dosing Trial on Safety and Efficacy of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Paediatric Subjects With Congenital Factor XIII A-subunit Deficiency. Safety Extension Trial to F13CD-3760
ClinicalTrials.gov ID: NCT01253811
Acronym / sponsor ID: mentor™5 / F13CD-3835
Conditions: Congenital Bleeding Disorder, Congenital FXIII Deficiency
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 6 participants (actual)
Lead sponsor: Novo Nordisk A/SIndustry (pharma, biotech, devices)
Study start: Jan 2011
Primary completion: Mar 2015 (actual)
Study completion: Mar 2015 (actual)
First posted: 3 Dec 2010 (estimated)
Results: Posted 24 Jun 2016

Clinical summary

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

This trial will be conducted in Asia, Europe and the United States of America (USA).

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
rFXIII 35 IU/kg	Experimental	Drug: catridecacog

Who may qualify

Ages: 1 year to 6 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (1 listed)

Completed participation in trial F13CD-3760 (NCT01230021)

Key exclusion criteria (7 listed)

Known or suspected hypersensitivity to trial product or related products
Known history of development of inhibitors against FXIII (factor XIII)
Hereditary or acquired coagulation disorder other than FXIII congenital deficiency
Platelet count (thrombocytes) less than 50X10e9 / L
Previous history of autoimmune disorder involving autoantibodies e.g., systemic lupus erythematosus

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
catridecacogAlso: recombinant factor XIIIIntravenous injection of a single dose of recombinant factor XIII, 35 IU/kg body weight every 4th week	Drug	rFXIII 35 IU/kg

Primary outcome

Number of Treatment Emergent (Serious and Non-serious) Adverse Events
Time frame: Week 0 to end of trial visit (week 173) for a minimum period of 52 weeks.
An adverse event was described as any untoward medical occurrence in a subject or clinical investigation subject administered a pharmaceutical product, and which does not necessarily have a causal relationship with this treatment. Treatment emergent adverse events (serious and non-serious), defined as adverse events occurring from first trial product administration to the end of the subject's participation in the trial.

15 secondary outcomes

Percentage of Subjects With Development of Anti-rFXIII Antibodies, Including Inhibitors.
Time frame: Week 0 to end of trial visit (week 173).
Clinical Laboratory Assessments: Biochemistry: Creatinine
Time frame: Every 6th month, from week 24 to end of trial visit (week 173).
Clinical Laboratory Assessments: Biochemistry: Urea
Time frame: Every 6th month, week 24 to end of trial visit (week 173).
Clinical Laboratory Assessments: Biochemistry: Alanine Aminotransferase (ALAT)
Time frame: Every 6th month, from week 24 to end of trial visit (week 173).
Clinical Laboratory Assessments: Biochemistry: Aspartate Aminotransferase (ASAT)
Time frame: Every 6th month, from week 24 to end of trial visit (week 173).
Clinical Laboratory Assessments: Haematology: Haemoglobin
Time frame: Every 6th month, from week 0 to end of trial visit (week 173).
Clinical Laboratory Assessments: Haematology: Leucocytes
Time frame: Every 6th month, from week 0 to end of trial visit (week 173).
Clinical Laboratory Assessments: Haematology: Thrombocytes
Time frame: Every 6th month, from week 0 to end of trial visit (week 173).
Clinical Laboratory Assessments: Haematology: Erythrocytes
Time frame: Every 6th month, from week 0 to end of trial visit (week 173).
Clinical Laboratory Assessments: Haematology: Haematocrit
Time frame: Every 6th month, from week 0 to end of trial visit (week 173).
Physical Examinations
Time frame: Week 0 to end of trial visit (week 173).
Vital Signs: Systolic BP (Blood Pressure)
Time frame: Week 0 to end of trial visit (week 173).
Vital Signs: Diastolic BP (Blood Pressure)
Time frame: Week 0 to end of trial visit (week 173).
Vital Signs: Pulse
Time frame: Week 0 to end of trial visit (week 173).
Rate (Number Per Subject Year) of All Bleeding Episodes Requiring Treatment With a FXIII Containing Product Other Than Recombinant Factor XIII.
Time frame: Weeks 0 to end of trial visit (week 173).

Results and safety

Results posted to the registry on 24 Jun 2016, 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 35 IU/kg	6	5

Primary outcome: Number of Treatment Emergent (Serious and Non-serious) Adverse Events

Time frame: Week 0 to end of trial visit (week 173) for a minimum period of 52 weeks.. Number. Unit: number of events

Measure	rFXIII 35 IU/kg n = 6
All adverse events	100
Serious adverse events	2
Non-serious adverse events	98

Population analysed: The safety analysis set included all subjects who received at least one dose of the trial product.

15 secondary outcomes are reported in the full results.

Adverse events

Time frame: All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
rFXIII 35 IU/kg	1 of 6 (16.7%)	6 of 6 (100%)	Not reported

Limitations and caveats (from the sponsor): Limitations of the trial included the small number of subjects analysed and the sensitivity of the Berichrom® FXIII activity assay. However, congenital FXIII deficiency is a rare disease and there were no bleeds requiring haemostatic treatment.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 24 Jun 2016.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 28581691Kerlin BA, Inbal A, Will A, Williams M, Garly ML, Jacobsen L, Kearney SL. Recombinant factor XIII prophylaxis is safe and effective in young children with congenital factor XIII-A deficiency: international phase 3b trial results. J Thromb Haemost. 2017 Aug;15(8):1601-1606. doi: 10.1111/jth.13748. Epub 2017 Jul 10.
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
Thrombotic Thrombocytopenic Purpura (TTP)
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)
Hereditary Hemorrhagic Telangiectasia (HHT), Osler Weber Rendu Disease
RecruitingObservational study
The CACHE Study: Coronary Artery Care in HaEmophilia
Haemophilia, Cardiovascular Diseases
Recruiting
Optimization of Saturation Targets And Resuscitation Trial (OptiSTART)
Premature Infants, Bronchopulmonary Dysplasia

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

4 sites in 3 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 4 sites in 3 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 1 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 Jun 2016. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites in United States, Israel, United Kingdom. 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 24 Jun 2016). 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 Treatment Emergent (Serious and Non-serious) Adverse Events (time frame: Week 0 to end of trial visit (week 173) for a minimum period of 52 weeks.). 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 NCT01253811, which also lists 1 linked publication.

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 Jun 2016.