Clinical Trial of Factor XIII (FXIII) Concentrate

NCT00640289 | Completed Results

• 1 other identifier: BB-IND5986
• Study Type: interventional
• Target: 72
• Locations: 1 country
• Sites: 1

Brief Summary

Congenital deficiency of Factor XIII is a rare but potentially life threatening disorder. It is inherited in an autosomal recessive fashion. Infusion of Factor XIII has proved to be useful for prevention and treatment of bleeding episodes, especially of spontaneous intracranial bleedings. In this study, Fibrogammin P will be given to patients with congenital Factor XIII deficiency and congenital/acquired FXIII deficiency to prevent bleeding and to treat established bleeding episodes. For Factor XIII prophylaxis to prevent hemorrhages, the dosage will depend on the weight of the subject. The frequency of Factor XIII administration will be determined by the factor's circulating half-life. During the first month only, a Factor XIII pharmacokinetic study will be determined over a 4-week period. Safety data will include accrual of information on viral safety, liver function, complete blood counts and adverse events. Historical data concerning spontaneous bleeds will be collected whenever possible two years prior to treatment with Fibrogammin P.

Conditions

Hemophilia; Factor XIII Deficiency

Keywords

Factor XIII Deficiency; Rare Bleeding Disorder; Hemophilia; Fibrogammin P

Outcome Measures

Primary Outcomes (1)

• Response to Treatment of Bleeding Events Requiring Additional Factor XIII Infusions

  Response is defined as: Excellent/Good = adequate hemostasis, similar to that expected for subjects without known bleeding disorders; Fair/Poor = hemostasis less than expected; None = severe bleeding, judged due to disease despite Factor XIII (FXIII) therapy. Only the subjects who needed additional FXIII infusions (apart from the prophylactic treatment) to control a bleed and who had investigator assessment of efficacy were counted in this outcome.

  Within 12 hours of FXIII infusion

Secondary Outcomes (1)

• Surgical Efficacy Assessments With Factor XIII

  During surgical procedure

Study Arms (1)

A

EXPERIMENTAL

Treatment

Drug: Fibrogammin P

Interventions

Fibrogammin P DRUG

Prophylaxis treatment

A

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients may be of either sex or age. Children and newborn infants are specifically included in this study.
• Patient must have documented congenital Factor XIII deficiency
• Patient or legal guardian must sign informed consent
• Patients who have negative serology for hepatitis B should receive Hepatitis B vaccination.

You may not qualify if:

• Patient has acquired Factor XIII deficiency

Sponsors & Collaborators

• Children's Hospital of Orange County: lead
• CSL Behring: collaborator

Study Sites (1)

Children's Hospital of Orange Co.

Orange, California, 92868, United States

Location

MeSH Terms

Conditions

Hemophilia A; Factor XIII Deficiency

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Clinical Trial Coordinator
• Organization: Children's Hospital of Orange County

KBirschbach@choc.org

Study Officials

• Diane J. Nugent, MD

  Children's Hospital of Orange Co.

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 17, 2008
• First Posted: March 21, 2008
• Study Start: January 1, 2000
• Primary Completion: December 1, 2011
• Study Completion: December 1, 2011
• Last Updated: November 7, 2018
• Results First Posted: November 7, 2018

Record last verified: 2008-03

Locations

US (1)