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

Information source: Baxalta US Inc.
ClinicalTrials.gov processed this data on August 20, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Protein C Deficiency

Intervention: Protein C Concentrate (Human) Vapor Heated (Drug)

Phase: Phase 2/Phase 3

Status: Completed

Sponsored by: Baxalta US Inc.

Official(s) and/or principal investigator(s):
Taru Hays, MD, Principal Investigator, Affiliation: ChildrenĀ“s Hospital, Denver, CO, USA

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.

Clinical Details

Official title: 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

Study design: Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Primary outcome: Whether episodes of purpura fulminans, Coumarin-induced skin necrosis and/or other thromboembolic event could be treated effectively, effectively with complications, or not treated effectively with Protein C Concentrate.

Eligibility

Minimum age: N/A. Maximum age: N/A. Gender(s): Both.

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.

Locations and Contacts

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

Additional Information

Starting date: August 2003
Last updated: June 26, 2015

Page last updated: August 20, 2015

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