Efficacy and Safety Study of Protein C Concentrate in Subjects With Severe Congenital Protein C Deficiency

NCT ID: NCT00157118

Last Updated: 2021-05-05

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 18 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2003-08-22
• Study Completion Date: 2005-03-17

Brief Summary

The purpose of this study is to show that Protein C Concentrate is a safe and effective treatment for subjects with congenital protein C deficiency. Depending on the type of treatment required, patients are assigned to one of 3 study parts: Part 1 is for the treatment of acute episodes, Part 2 is for short-term prophylaxis, and Part 3 is for long-term prophylaxis in infants aged less than 6 months only.

Conditions

Protein C Deficiency

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Interventions

Protein C Concentrate (Human) Vapor Heated

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Newborn subjects <= 6 months of age: diagnosis of severe congenital protein C deficiency, with documented functional protein C level < 20%. If a genetic diagnosis is not available prior to initiation of Protein C Concentrate treatment, a documented family history of protein C deficiency is required.
• Subjects > 6 months of age: confirmed diagnosis of severe congenital protein C deficiency, i.e., by
• a genetic analysis of severe congenital protein C deficiency (i.e., homozygous or double heterozygous) OR
• a documented family history of protein C deficiency AND a documented functional protein C level < 20% while the subject is in an asymptomatic state and not receiving oral anticoagulation therapy
• Signed and dated informed consent from either the subject or the subject's legally authorized representative prior to enrollment. Informed consent includes consent for conducting a genetic analysis (if the genetic defect is unknown).

Exclusion Criteria

• Subjects with a history of allergic reactions to Protein C Concentrate. In the case of allergic reactions occurring at the Protein C Concentrate injection site; exclusion from the study is at the discretion of the investigator but should be reported as an AE regardless of the subject's inclusion in or exclusion from the study.
• Participation in any clinical study in which another investigational agent is used within 30 days prior to enrollment or its use is expected at any time for the duration of study participation.

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Baxalta now part of Shire

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Study Director

Role: STUDY_DIRECTOR

Takeda

Locations

Los Angeles, California, United States

Denver, Colorado, United States

Atlanta, Georgia, United States

Indianapolis, Indiana, United States

Lexington, Kentucky, United States

Boston, Massachusetts, United States

Cincinnati, Ohio, United States

Cleveland, Ohio, United States

Dayton, Ohio, United States

Fort Worth, Texas, United States

Galveston, Texas, United States

Houston, Texas, United States

Countries

United States

References

Gelmont D., Hays T., Bomgaars L., Palascak J., Shapiro A., Geil J., Montgomery J., Fritsch S., Maritsch F., Pavlova B., Ehrlich H.J. A Phase II/III Clinical Study for the Determination of the Efficacy and Safety of Protein C Concentrate in Subjects with Severe Congenital Protein C Deficiency -Interim Study Analysis. Accepted for poster presentation at the 20th International Society on Thrombosis and Haemostasis Congress. Sydney, Australia, August 6-12 2005. (ISTH 2005)

Reference Type: RESULT

Other Identifiers

400101

Identifier Type: -

Identifier Source: org_study_id