Congenital Bleeding DisorderPhase 3mentor™2NCT00978380
Safety of Monthly Recombinant Factor XIII Replacement Therapy in Subjects With Congenital Factor XIII Deficiency: An Extension to Trial F13CD-1725
A Phase 3 study testing catridecacog in people with Congenital Bleeding Disorder and Congenital FXIII Deficiency, run by Novo Nordisk A/S at 34 sites in 12 countries.
Trial summary
This trial is conducted in Asia, Europe and North America. The aim of the trial is to investigate the safety of monthly replacement therapy of recombinant factor XIII in patients with congenital FXIII deficiency. 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
- 63 participants
- Where
- 34 sitesin 12 countries
- Study dates
- 21 Sep 2009to 20 Oct 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 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- For subjects who participated in F13CD-1725:
- Previous participation (means up to and inclusive Visit 16, (End of Trial)) in F13CD-1725
- For all other subjects:
- Diagnosis of congenital FXIII A-subunit deficiency (confirmed by genotyping at screening visit or documented results from previously performed genotyping)
- Body weight at least 20 kg
Who cannot join
- Known neutralizing antibodies (inhibitors) towards FXIII
- Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency
- Platelet count (thrombocytes) of less than 50 × 109/L. For subjects who participated in F13CD-1725 platelet count from visit 15 in F13CD-1725 must be used for evaluation.
- Females of childbearing potential who are pregnant, breastfeeding or are not using adequate contraceptive methods
Trial locations and contacts
34 sites in 12 countries.
34 sites in 12 countries
United States12 sites
Novo Nordisk Investigational Site
Atlanta, Georgia, 30322
Novo Nordisk Investigational Site
Boise, Idaho, 83712
Novo Nordisk Investigational Site
Columbus, Ohio, 43205
Novo Nordisk Investigational Site
Detroit, Michigan, 48201
Novo Nordisk Investigational Site
Hershey, Pennsylvania, 17033
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
Richmond, Virginia, 23219
Novo Nordisk Investigational Site
Seattle, Washington, 98104
Novo Nordisk Investigational Site
Tampa, Florida, 33607
Novo Nordisk Investigational Site
Washington D.C., District of Columbia, 20007
France4 sites
Novo Nordisk Investigational Site
Le Kremlin-Bicêtre, 94270
Novo Nordisk Investigational Site
Paris, 75015
Novo Nordisk Investigational Site
Rouen, 76031
Novo Nordisk Investigational Site
Valence, 26953
United Kingdom4 sites
Novo Nordisk Investigational Site
Aberdeen, AB25 2ZN
Novo Nordisk Investigational Site
London, WC1N 3JH
Novo Nordisk Investigational Site
Manchester, M13 9WL
Novo Nordisk Investigational Site
Newcastle upon Tyne, NE1 4LP
Germany3 sites
Novo Nordisk Investigational Site
Bonn, 53127
Novo Nordisk Investigational Site
Braunschweig, 38118
Novo Nordisk Investigational Site
Duisburg, 47051
Spain3 sites
Novo Nordisk Investigational Site
Barcelona, 08035
Novo Nordisk Investigational Site
Málaga, 29011
Novo Nordisk Investigational Site
Tortosa, 43500
Japan2 sites
Novo Nordisk Investigational Site
Hiroshima-shi, Hiroshima, 734 8551
Novo Nordisk Investigational Site
Shinjuku-ku, Tokyo, 160 0023
About this study
Official title: A Multi-Centre, Open-Label, Single-Arm, and Multiple Dosing Trial on Safety of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Subjects With Congenital Factor XIII Deficiency
This trial is conducted in Asia, Europe and North America. The aim of the trial is to investigate the safety of monthly replacement therapy of recombinant factor XIII in patients with congenital FXIII deficiency. The trial continues until the product is commercially available, but an interim assessment will take place when all subjects have completed 52 weeks in the trial.
- 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
Monthly administration of recombinant factor XIII as preventative treatment of bleeding episodes. Dose: 35 IU/kg body weight intravenous (into the vein)
| Study group | Role | What they receive |
|---|---|---|
| A | Experimental | Not described Receives: Drug: catridecacog |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events (AEs)(Serious and Non-serious)
An AE was defined as any unfavourable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. Trial AEs (serious) included any event such as death, life-threatening experience, in-subject hospitalisation, significant disability/ congential anomaly experienced from the trial product.
Measured: All AEs were collected and reported from screening (week 0) for a minimum period of 52 weeks or until the end of trial visit.
Secondary outcomes (1)
Antibody and Inhibitor Development
All subjects who received rFXIII were monitored for anti-rFXIII antibodies and inhibitor development. 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. Percentage of subjects with antibody and inhibitor development were reported.
Measured: From week 0 to week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Sep 2009 (estimated)
- Study start21 Sep 2009
- Primary completion20 Oct 2015
- Study completion20 Oct 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 (3)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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 63 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novo Nordisk A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Global Clinical Registry (GCR, 1452), Study director, Novo Nordisk A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00978380
- Sponsor's protocol ID
- F13CD-3720
- Other IDs
- 2008-007883-41, U1111-1111-9289, JapicCTI-121958, 2008
- Acronym
- mentor™2
- Status last verified
- Jan 2018
- First submitted
- 15 Sep 2009
- Last updated
- 24 Jan 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
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 25643920Kerlin B, Brand B, Inbal A, Halimeh S, Nugent D, Lundblad M, Tehranchi R. Pharmacokinetics of recombinant factor XIII at steady state in patients with congenital factor XIII A-subunit deficiency. J Thromb Haemost. 2014 Dec;12(12):2038-43. doi: 10.1111/jth.12739. Epub 2014 Oct 25.
PubMed 25263390Byrnes JR, Lee T, Sharaby S, Campbell RA, Dobson DA, Holle LA, Luo M, Kangro K, Homeister JW, Aleman MM, Luyendyk JP, Kerlin BA, Dumond JB, Wolberg AS. Reciprocal stabilization of coagulation factor XIII-A and -B subunits is a determinant of plasma FXIII concentration. Blood. 2024 Feb 1;143(5):444-455. doi: 10.1182/blood.2023022042.
PubMed 37883802
Study at a glance
From the registry record, last updated by the sponsor on 24 Jan 2018. 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 Safety of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Subjects With Congenital Factor XIII Deficiency
- ClinicalTrials.gov ID
- NCT00978380
- Acronym / sponsor ID
- mentor™2 / F13CD-3720
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 63 participants (actual)
- Lead sponsor
- Novo Nordisk A/SIndustry (pharma, biotech, devices)
- Study start
- 21 Sep 2009 (actual)
- Primary completion
- 20 Oct 2015 (actual)
- Study completion
- 20 Oct 2015 (actual)
- First posted
- 16 Sep 2009 (estimated)
- Results
- Posted 13 Dec 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 is conducted in Asia, Europe and North America. The aim of the trial is to investigate the safety of monthly replacement therapy of recombinant factor XIII in patients with congenital FXIII deficiency. The trial continues until the product is commercially available, but an interim assessment will take place when all subjects have completed 52 weeks in the trial.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| A | 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 (5 listed)
- For subjects who participated in F13CD-1725:
- Previous participation (means up to and inclusive Visit 16, (End of Trial)) in F13CD-1725
- For all other subjects:
- Diagnosis of congenital FXIII A-subunit deficiency (confirmed by genotyping at screening visit or documented results from previously performed genotyping)
- Body weight at least 20 kg
Key exclusion criteria (4 listed)
- Known neutralizing antibodies (inhibitors) towards FXIII
- Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency
- Platelet count (thrombocytes) of less than 50 × 109/L. For subjects who participated in F13CD-1725 platelet count from visit 15 in F13CD-1725 must be used for evaluation.
- Females of childbearing potential who are pregnant, breastfeeding or are not using adequate contraceptive methods
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 XIIIMonthly administration of recombinant factor XIII as preventative treatment of bleeding episodes. Dose: 35 IU/kg body weight intravenous (into the vein) | Drug | A |
Primary outcome
Adverse Events (AEs)(Serious and Non-serious)
Time frame: All AEs were collected and reported from screening (week 0) for a minimum period of 52 weeks or until the end of trial visit.
An AE was defined as any unfavourable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. Trial AEs (serious) included any event such as death, life-threatening experience, in-subject hospitalisation, significant disability/ congential anomaly experienced from the trial product.
1 secondary outcome
Antibody and Inhibitor Development
Time frame: From week 0 to week 52
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jan 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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.
- Results publication: PubMed 25263390Kerlin B, Brand B, Inbal A, Halimeh S, Nugent D, Lundblad M, Tehranchi R. Pharmacokinetics of recombinant factor XIII at steady state in patients with congenital factor XIII A-subunit deficiency. J Thromb Haemost. 2014 Dec;12(12):2038-43. doi: 10.1111/jth.12739. Epub 2014 Oct 25.
- Derived from this study: PubMed 37883802Byrnes JR, Lee T, Sharaby S, Campbell RA, Dobson DA, Holle LA, Luo M, Kangro K, Homeister JW, Aleman MM, Luyendyk JP, Kerlin BA, Dumond JB, Wolberg AS. Reciprocal stabilization of coagulation factor XIII-A and -B subunits is a determinant of plasma FXIII concentration. Blood. 2024 Feb 1;143(5):444-455. doi: 10.1182/blood.2023022042.
- Clinical Trials at Novo NordiskLink provided by the sponsor.
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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
34 sites in 12 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 34 sites in 12 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 5 inclusion and 4 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 Jan 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 34 sites in United States, France, United Kingdom, Germany, Spain and 7 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 13 Dec 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?
Adverse Events (AEs)(Serious and Non-serious) (time frame: All AEs were collected and reported from screening (week 0) for a minimum period of 52 weeks or until the end of trial visit.). 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 NCT00978380, which also lists 3 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 Jan 2018.