Safety of Monthly Recombinant Factor XIII Replacement Therapy in Subjects With Congenital Factor XIII Deficiency: An Extension to Trial F13CD-1725

NCT ID: NCT00978380

Last Updated: 2018-01-24

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

63 participants

Study Classification

INTERVENTIONAL

Study Start Date

2009-09-21

Study Completion Date

2015-10-20

Brief Summary

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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.

Detailed Description

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Conditions

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Congenital Bleeding Disorder Congenital FXIII Deficiency

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

PREVENTION

Blinding Strategy

NONE

Study Groups

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A

Group Type EXPERIMENTAL

catridecacog

Intervention Type DRUG

Monthly administration of recombinant factor XIII as preventative treatment of bleeding episodes. Dose: 35 IU/kg body weight intravenous (into the vein)

Interventions

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catridecacog

Monthly administration of recombinant factor XIII as preventative treatment of bleeding episodes. Dose: 35 IU/kg body weight intravenous (into the vein)

Intervention Type DRUG

Other Intervention Names

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recombinant factor XIII

Eligibility Criteria

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Inclusion Criteria

* 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

Exclusion Criteria

* 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
Minimum Eligible Age

6 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novo Nordisk A/S

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Global Clinical Registry (GCR, 1452)

Role: STUDY_DIRECTOR

Novo Nordisk A/S

Locations

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Novo Nordisk Investigational Site

Orange, California, United States

Site Status

Novo Nordisk Investigational Site

Washington D.C., District of Columbia, United States

Site Status

Novo Nordisk Investigational Site

Tampa, Florida, United States

Site Status

Novo Nordisk Investigational Site

Atlanta, Georgia, United States

Site Status

Novo Nordisk Investigational Site

Boise, Idaho, United States

Site Status

Novo Nordisk Investigational Site

Detroit, Michigan, United States

Site Status

Novo Nordisk Investigational Site

Minneapolis, Minnesota, United States

Site Status

Novo Nordisk Investigational Site

Columbus, Ohio, United States

Site Status

Novo Nordisk Investigational Site

Oklahoma City, Oklahoma, United States

Site Status

Novo Nordisk Investigational Site

Hershey, Pennsylvania, United States

Site Status

Novo Nordisk Investigational Site

Richmond, Virginia, United States

Site Status

Novo Nordisk Investigational Site

Seattle, Washington, United States

Site Status

Novo Nordisk Investigational Site

Klagenfurt, , Austria

Site Status

Novo Nordisk Investigational Site

Toronto, Ontario, Canada

Site Status

Novo Nordisk Investigational Site

Helsinki, , Finland

Site Status

Novo Nordisk Investigational Site

Le Kremlin-Bicêtre, , France

Site Status

Novo Nordisk Investigational Site

Paris, , France

Site Status

Novo Nordisk Investigational Site

Rouen, , France

Site Status

Novo Nordisk Investigational Site

Valence, , France

Site Status

Novo Nordisk Investigational Site

Bonn, , Germany

Site Status

Novo Nordisk Investigational Site

Braunschweig, , Germany

Site Status

Novo Nordisk Investigational Site

Duisburg, , Germany

Site Status

Novo Nordisk Investigational Site

Petah Tikva, , Israel

Site Status

Novo Nordisk Investigational Site

Vicenza, , Italy

Site Status

Novo Nordisk Investigational Site

Hiroshima-shi, Hiroshima, , Japan

Site Status

Novo Nordisk Investigational Site

Shinjuku-ku, Tokyo, , Japan

Site Status

Novo Nordisk Investigational Site

Barcelona, , Spain

Site Status

Novo Nordisk Investigational Site

Málaga, , Spain

Site Status

Novo Nordisk Investigational Site

Tortosa, , Spain

Site Status

Novo Nordisk Investigational Site

Zurich, , Switzerland

Site Status

Novo Nordisk Investigational Site

Aberdeen, , United Kingdom

Site Status

Novo Nordisk Investigational Site

London, , United Kingdom

Site Status

Novo Nordisk Investigational Site

Manchester, , United Kingdom

Site Status

Novo Nordisk Investigational Site

Newcastle upon Tyne, , United Kingdom

Site Status

Countries

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United States Austria Canada Finland France Germany Israel Italy Japan Spain Switzerland United Kingdom

References

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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.

Reference Type RESULT
PMID: 25643920 (View on PubMed)

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.

Reference Type RESULT
PMID: 25263390 (View on PubMed)

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.

Reference Type DERIVED
PMID: 37883802 (View on PubMed)

Related Links

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http://novonordisk-trials.com

Clinical Trials at Novo Nordisk

Other Identifiers

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2008-007883-41

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

U1111-1111-9289

Identifier Type: OTHER

Identifier Source: secondary_id

JapicCTI-121958

Identifier Type: REGISTRY

Identifier Source: secondary_id

F13CD-3720

Identifier Type: -

Identifier Source: org_study_id

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