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

May 3, 2021 updated by: Baxalta now part of Shire

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

18

Phase

  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Los Angeles, California, United States
    • Colorado
      • Denver, Colorado, United States
    • Georgia
      • Atlanta, Georgia, United States
    • Indiana
      • Indianapolis, Indiana, United States
    • Kentucky
      • Lexington, Kentucky, United States
    • Massachusetts
      • Boston, Massachusetts, United States
    • Ohio
      • Cincinnati, Ohio, United States
      • Cleveland, Ohio, United States
      • Dayton, Ohio, United States
    • Texas
      • Fort Worth, Texas, United States
      • Galveston, Texas, United States
      • Houston, Texas, United States

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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.
Time Frame: Part 1 of the study
Part 1 of the study

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Baxalta now part of Shire

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

  • 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)

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 22, 2003

Primary Completion (Actual)

March 17, 2005

Study Completion (Actual)

March 17, 2005

Study Registration Dates

First Submitted

September 8, 2005

First Submitted That Met QC Criteria

September 8, 2005

First Posted (Estimate)

September 12, 2005

Study Record Updates

Last Update Posted (Actual)

May 5, 2021

Last Update Submitted That Met QC Criteria

May 3, 2021

Last Verified

May 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 400101

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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