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| ID | Type | Description | Link |
|---|---|---|---|
| 2008-007883-41 | EudraCT Number | ||
| U1111-1111-9289 | Other Identifier | WHO | |
| JapicCTI-121958 | Registry Identifier | JAPIC |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| A | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| catridecacog | Drug | Monthly administration of recombinant factor XIII as preventative treatment of bleeding episodes. Dose: 35 IU/kg body weight intravenous (into the vein) |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | All AEs were collected and reported from screening (week 0) for a minimum period of 52 weeks or until the end of trial visit. |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Global Clinical Registry (GCR, 1452) | Novo Nordisk A/S | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novo Nordisk Investigational Site | Orange | California | 92868 | United States | ||
| Novo Nordisk Investigational Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 25643920 | Result | 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. | |
| 25263390 |
| Label | URL |
|---|---|
| Clinical Trials at Novo Nordisk | View source |
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Subjects who completed F13CD-1725 (CT.gov identifier: NCT00713648) end of trial visit were eligible to enroll in this trial. Also, new subjects diagnosed with congenital FXIII A-subunit deficiency (confirmed by genotyping at screening visit or documented results from previously performed genotyping) were enrolled to expand the safety population.
The trial was conducted at 34 sites in 12 countries as follows: Austria: 1 site; Canada: 1 site; Finland: 1 site; France: 4 sites; Germany: 4 sites; Israel: 1 site; Italy: 1 site: Japan: 2 sites; Spain: 2 sites; Switzerland: 1 site; United Kingdom: 4 sites; United States: 12 sites.
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| ID | Title | Description |
|---|---|---|
| FG000 | Recombinant Factor XIII (rFXIII) | Subjects received 35 IU/kg bodyweight of rFXIII slow intravenous (i.v.) injection every 4 weeks (28 days±2 days) for a minimum period of 52 weeks until the end of trial visit. The dose was identical to the dose administered in the F13CD- 1725 trial. A total of 60 unique subjects were enrolled and exposed in the trial, but 3 of these subjects were later withdrawn and subsequently re-enrolled with new subject IDs, giving rise to a total of N=63 subjects. The unique subjects (N=60) were presented as full analysis set (FAS) while summarising adverse events to avoid double-counting. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| From week 0 to week 52 |
| Washington D.C. |
| District of Columbia |
| 20007 |
| United States |
| Novo Nordisk Investigational Site | Tampa | Florida | 33607 | United States |
| Novo Nordisk Investigational Site | Atlanta | Georgia | 30322 | United States |
| Novo Nordisk Investigational Site | Boise | Idaho | 83712 | United States |
| Novo Nordisk Investigational Site | Detroit | Michigan | 48201 | United States |
| Novo Nordisk Investigational Site | Minneapolis | Minnesota | 55404 | United States |
| Novo Nordisk Investigational Site | Columbus | Ohio | 43205 | United States |
| Novo Nordisk Investigational Site | Oklahoma City | Oklahoma | 73104 | United States |
| Novo Nordisk Investigational Site | Hershey | Pennsylvania | 17033 | United States |
| Novo Nordisk Investigational Site | Richmond | Virginia | 23219 | United States |
| Novo Nordisk Investigational Site | Seattle | Washington | 98104 | United States |
| Novo Nordisk Investigational Site | Klagenfurt | A-9020 | Austria |
| Novo Nordisk Investigational Site | Toronto | Ontario | M5G 1X8 | Canada |
| Novo Nordisk Investigational Site | Helsinki | 00290 | Finland |
| Novo Nordisk Investigational Site | Le Kremlin-Bicêtre | 94270 | France |
| Novo Nordisk Investigational Site | Paris | 75015 | France |
| Novo Nordisk Investigational Site | Rouen | 76031 | France |
| Novo Nordisk Investigational Site | Valence | 26953 | France |
| Novo Nordisk Investigational Site | Bonn | 53127 | Germany |
| Novo Nordisk Investigational Site | Braunschweig | 38118 | Germany |
| Novo Nordisk Investigational Site | Duisburg | 47051 | Germany |
| Novo Nordisk Investigational Site | Petah Tikva | 49100 | Israel |
| Novo Nordisk Investigational Site | Vicenza | 36100 | Italy |
| Novo Nordisk Investigational Site | Hiroshima-shi, Hiroshima | 734 8551 | Japan |
| Novo Nordisk Investigational Site | Shinjuku-ku, Tokyo | 160 0023 | Japan |
| Novo Nordisk Investigational Site | Barcelona | 08035 | Spain |
| Novo Nordisk Investigational Site | Málaga | 29011 | Spain |
| Novo Nordisk Investigational Site | Tortosa | 43500 | Spain |
| Novo Nordisk Investigational Site | Zurich | 8091 | Switzerland |
| Novo Nordisk Investigational Site | Aberdeen | AB25 2ZN | United Kingdom |
| Novo Nordisk Investigational Site | London | WC1N 3JH | United Kingdom |
| Novo Nordisk Investigational Site | Manchester | M13 9WL | United Kingdom |
| Novo Nordisk Investigational Site | Newcastle upon Tyne | NE1 4LP | United Kingdom |
| Kerlin 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. |
| 37883802 | Derived | Byrnes 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. |
| COMPLETED |
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| NOT COMPLETED |
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All subjects who received trial product were considered for baseline characteristics including 3 subjects who were re-enrolled with new subject IDs.
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| ID | Title | Description |
|---|---|---|
| BG000 | Recombinant Factor XIII (rFXIII) | Subjects received 35 IU/kg bodyweight of rFXIII slow intravenous (i.v.) injection every 4 weeks (28 days±2 days) for a minimum period of 52 weeks until the end of trial visit. The dose was identical to the dose administered in the F13CD- 1725 trial. A total of 60 unique subjects were enrolled and exposed in the trial, but 3 of these subjects were later withdrawn and subsequently re-enrolled with new subject IDs, giving rise to a total of N=63 subjects. The unique subjects (N=60) were presented as full analysis set (FAS) while summarising adverse events to avoid double-counting. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
| ||||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| |||||||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| |||||||||||||||||||||||
| Race/Ethnicity, Customized | Number | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | The FAS included the 60 unique subjects exposed to trial product in this extension trial. | Posted | Number | Events | 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 | 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. | The safety analysis set included all subjects exposed to trial product in this extension trial. | Posted | Number | Percentage of subjects | From week 0 to week 52 |
|
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All adverse events (AEs) were collected and reported from screening (week 0) for a minimum period of 52 weeks or until the end of trial visit.
The FAS was used for AEs. In the early stage of the trial, a contract manufacturing facility (Avecia) produced the drug. Later, Novo Nordisk took over production of the drug. Hence, AE data are presented seperately who received both rFXIII Avecia and Novo Nordisk. Subsequently, subjects were counted under both groups.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | rFXIII Novo Nordisk | Subjects in this arm received identical dose of 35 IU/kg bodyweight of rFXIII slow intravenous (i.v.) administered every 4 weeks (28 days±2 days) until the end of trial for a minimum period of 52 weeks. In the early stage of the trial, a contract manufacturing facility (Avecia) produced the rFXIII drug substance (rFXIII Avecia) and subsequently, Novo Nordisk took over production of the rFXIII drug substance (rFXIII Novo Nordisk). Characterisation testing between the two products confirmed that rFXIII Novo Nordisk and rFXIII Avecia had identical structures, and similar physico-chemical properties. The AE data are presented seperately for rFXIII Novo Nordisk and rFXIII Avecia. However, subjects in this arm received rFXIII Novo Nordisk drug. | 12 | 59 | 54 | 59 | ||
| EG001 | rFXIII Avecia | Subjects in this arm received identical dose of 35 IU/kg bodyweight of rFXIII slow intravenous (i.v.) administered every 4 weeks (28 days±2 days) until the end of trial for a minimum period of 52 weeks. In the early stage of the trial, a contract manufacturing facility (Avecia) produced the rFXIII drug substance (rFXIII Avecia) and subsequently, Novo Nordisk took over production of the rFXIII drug substance (rFXIII Novo Nordisk). Characterisation testing between the two products confirmed that rFXIII Novo Nordisk and rFXIII Avecia had identical structures, and similar physico-chemical properties. The AE data are presented seperately for rFXIII Novo Nordisk and rFXIII Avecia. However, subjects in this arm received rFXIII Avecia drug. | 0 | 26 | 15 | 26 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Atrial septal defect | Congenital, familial and genetic disorders | MedDRA | Systematic Assessment |
| |
| Cerebral ischaemia | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Chest injury | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Diverticulitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Ectopic pregnancy | Pregnancy, puerperium and perinatal conditions | MedDRA | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Head injury | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Inguinal hernia | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Laceration | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Multiple fractures | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Otitis media chronic | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Patent ductus arteriosus | Congenital, familial and genetic disorders | MedDRA | Systematic Assessment |
| |
| Road traffic accident | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Spinal cord injury | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Suicide attempt | Psychiatric disorders | MedDRA | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Acne | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
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| Arthropod bite | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
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| Back pain | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
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| Constipation | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
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| Dental caries | Gastrointestinal disorders | MedDRA | Systematic Assessment |
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| Dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA | Systematic Assessment |
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| Dyspepsia | Gastrointestinal disorders | MedDRA | Systematic Assessment |
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| Ear pain | Ear and labyrinth disorders | MedDRA | Systematic Assessment |
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| Fall | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
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| Fatigue | General disorders | MedDRA | Systematic Assessment |
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| Folliculitis | Infections and infestations | MedDRA | Systematic Assessment |
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| Gastroenteritis | Infections and infestations | MedDRA | Systematic Assessment |
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| Haematuria | Renal and urinary disorders | MedDRA | Systematic Assessment |
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| Head injury | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA | Systematic Assessment |
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| Hypertension | Vascular disorders | MedDRA | Systematic Assessment |
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| Incorrect dose administered | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
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| Influenza | Infections and infestations | MedDRA | Systematic Assessment |
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| Injury | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
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| Joint injury | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
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| Laceration | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
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| Ligament sprain | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
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| Limb injury | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
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| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
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| Musculoskeletal stiffness | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
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| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
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| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA | Systematic Assessment |
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| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
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| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
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| Pain | General disorders | MedDRA | Systematic Assessment |
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| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
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| Post-traumatic pain | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
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| Procedural pain | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
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| Pruritus | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA | Systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
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| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
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| Road traffic accident | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
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| Seasonal allergy | Immune system disorders | MedDRA | Systematic Assessment |
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| Sinusitis | Infections and infestations | MedDRA | Systematic Assessment |
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| Skin abrasion | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
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| Thermal burn | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
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| Tonsillitis | Infections and infestations | MedDRA | Systematic Assessment |
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| Toothache | Gastrointestinal disorders | MedDRA | Systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | MedDRA | Systematic Assessment |
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| Viral infection | Infections and infestations | MedDRA | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA | Systematic Assessment |
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| White blood cells urine positive | Investigations | MedDRA | Systematic Assessment |
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| Wound | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
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Limitations of the trial include small number of children and adolescents (16 subjects), and the sensitivity of the Berichrome® FXIII activity assay. However, mean FXIII activity levels were consistent with few bleeds requiring haemostatic treatment.
At the end of the trial, one or more scientific publication will be prepared collaboratively between Investigator(s) and Novo Nordisk. Novo Nordisk reserves the right to postpone publication and/or communication for up to 60 days to protect intellectual property.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Global Clinical Registry (GCR, 1452) | Novo Nordisk A/S | clinicaltrials@novonordisk.com |
| ID | Term |
|---|---|
| C521905 | recombinant factor XIII-A2 |
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| Unknown or Not Reported |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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| Other |
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| Title | Measurements |
|---|---|
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| Participants |
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