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Congenital Bleeding DisorderPhase 3mentor™4NCT01230021

Safety of a Single Intravenous Dose of Recombinant Factor XIII in Children With Congenital FXIII 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 10 sites in 3 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

This trial is conducted in Europe and United States of America (USA). The aim of this clinical trial is to investigate the pharmacokinetics (at which rate the substance is distributed and eliminated from the body) and the safety profile of catridecacog (recombinant factor XIII (rFXIII)) in children with congenital… Read more about this study

Who is it for?
Ages 1 year to 6 years
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
10 sitesin 3 countries
Study dates
Nov 2010to Jan 2012
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

  • Signed Informed Consent by subject's parents or subject's legally acceptable representative before any trial related activities. Trial related activities are any procedures that would not have been performed during the normal management of the subject
  • Age 1 to less than 6 years old at the time of enrolment
  • Congenital FXIII subunit-A deficiency previously documented by genotyping or evaluated by genotyping through blood sampling at screening visit
  • Body weight at least 10 kg

Who cannot join

  • Known antibodies to FXIII
  • Hereditary or acquired coagulation disorder other than FXIII A-subunit congenital deficiency
  • Platelet count (thrombocytes) of less than 50 × 10^9/L (at screening visit)
  • 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
  • Known or suspected allergy to trial product or related products
  • Any surgical procedure in the 30 days prior to enrolment and any planned surgery during the trial period
  • Any disease or condition which, judged by the Investigator, could imply a potential hazard to the subject or interfere with the trial participation or trial outcome including renal and/or liver dysfunction

Trial locations and contacts

10 sites in 3 countries.

10 sites in 3 countries

United Kingdom5 sites

  • Novo Nordisk Investigational Site

    Birmingham, B4 6NH

  • Novo Nordisk Investigational Site

    Leicester, LE1 5WW

  • Novo Nordisk Investigational Site

    Manchester, M13 9WL

  • Novo Nordisk Investigational Site

    Newcastle upon Tyne, NE1 4LP

  • Novo Nordisk Investigational Site

    Reading, RG1 5AN

Other trials in United Kingdom

United States4 sites

  • 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

    Minneapolis, Minnesota, 55404

Other trials in United States

Israel1 site

  • Novo Nordisk Investigational Site

    Petah Tikva, 49100

Other trials in Israel

About this study

Official title: A Phase 3b Trial Investigating the Pharmacokinetics and Safety Profile of a Single Intravenous Dose of rFXIII in Paediatric (1 to Less Than 6 Years Old) Subjects With Congenital FXIII A-subunit Deficiency

This trial is conducted in Europe and United States of America (USA). The aim of this clinical trial is to investigate the pharmacokinetics (at which rate the substance is distributed and eliminated from the body) and the safety profile of catridecacog (recombinant factor XIII (rFXIII)) in children with congenital FXIII A-subunit deficiency. Young children (1 to less than 6 years old) with congenital FXIII deficiency are evaluated.

Being tested
catridecacog

What participants receive

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

  • catridecacogDrug

    Intravenous injection of a single dose of recombinant factor XIII, 35 IU/kg bodyweight

Study groups in NCT01230021
Study groupRoleWhat they receive
recombinant factor XIIIExperimentalNot described
Receives: Drug: catridecacog

What is being measured

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

  1. Area Under the Concentration vs. Time Curve (AUC)

    A measure of the exposure. Blood samples for the PK assessment were drawn pre-dose and up to 30 days after dosing. The PK of FXIII in children was assessed after a single i.v. dose of rFXIII 35 IU/kg.

    Measured: At pre-dose, 30 minutes, 24 hours, 7, 14, 21 and 30 days after dosing

Secondary outcomes (16)

  1. Area Under the Concentration vs. Time Curve (AUC0-∞)

    A measure of exposure.

    Measured: From day 0 to day 30

  2. Maximum Plasma Concentration (Cmax) for FXIII

    Maximum plasma concentration of the drug reached.

    Measured: At pre-dose, 30 minutes, 24 hours, 7, 14, 21 and 30 days after dosing

  3. Terminal Half-life (t½)

    Time point when half of the maximum plasma concentration is reached.

    Measured: From day 0 to day 30

  4. Mean Residence Time (MRT)

    The mean residence time (MRT) of a drug in the body and related functions are derived for drugs which are intravenously administered.

    Measured: From day 0 to day 30

  5. Total Plasma Clearance (CL)

    The total plasma clearance is a measure of the elimination of a drug from the body. Drugs are excreted primarily by the kidneys into the urine. Clearance is calculated as 'CL=Dose / AUC0-30 days').

    Measured: From day 0 to day 30

  6. Volume of Distribution at Steady State (Vss)

    Volume of distribution at steady state (Vss) is the theoretical volume that the total amount of administered drug would have to occupy (if it were uniformly distributed), to provide the same concentration as it is in blood plasma at steady state. Steady state is achieved when all variables are constant in spite of ongoing processes.

    Measured: At steady state

  7. Percentage of Subjects With One or More Adverse Events (AEs) Recorded

    Measured: From day 0 to day 30

  8. Percentage of Subjects With One or More Serious Adverse Events (SAEs)

    Measured: From day 0 to day 30

  9. Percentage of Subjects With Development of Anti-rFXIII Antibodies, Including Inhibitors (Neutralising Antibodies Against Factor XIII)

    Measured: At screening and day 30

  10. Coagulation Related Parameters - Fibrinogen

    Measured: Day 0 and at day 30

  11. Coagulation Related Parameters - Activated Partial Thromboplastin Time (aPTT, Seconds)

    Measured: Day 0 and day 30

  12. Coagulation Related Parameters - Prothrombin Time (PT) (Seconds)

    Measured: Day 0 and day 30

  13. Clot Solubility Test (Evaluated as Normal/Abnormal)

    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: Day 0 and day 30

  14. Vital Signs - Pulse

    Measured: Day 0 and day 30

  15. Vital Signs - Blood Pressure (Systolic and Diastolic)

    Measured: Day 0 and day 30

  16. Physical Examination (Evaluated as Normal/Abnormal)

    Measured: From day 0 to day 30

Timeline

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

  1. First posted28 Oct 2010 (estimated)
  2. Study startNov 2010
  3. Primary completionJan 2012
  4. Study completionJan 2012

Resources

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
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
6 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01230021
Sponsor's protocol ID
F13CD-3760
Other IDs
U1111-1116-2533, 2009-016869-28, 2009
Acronym
mentor™4
Status last verified
Jan 2017
First submitted
27 Oct 2010
Last updated
24 Feb 2017
Data monitoring committee
No

Publications

  • Williams M, Will A, Stenmo C, Rosholm A, Tehranchi R. Pharmacokinetics of recombinant factor XIII in young children with congenital FXIII deficiency and comparison with older patients. Haemophilia. 2014 Jan;20(1):99-105. doi: 10.1111/hae.12224. Epub 2013 Jul 9.

    PubMed 23834599

  • 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

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 10 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 4 inclusion and 8 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 10 sites in United Kingdom, United States, Israel. 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 12 Jun 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?

Area Under the Concentration vs. Time Curve (AUC) (time frame: At pre-dose, 30 minutes, 24 hours, 7, 14, 21 and 30 days after dosing). 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 NCT01230021, 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.

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