Retrospective Study to Capture Dosing and Treatment Outcome Data in Participants With Severe Congenital Protein C Deficiency Who Were Treated With Protein C Concentrate Under an Emergency Use IND

April 7, 2021 updated by: Baxalta now part of Shire

A Retrospective Study to Capture Dosing and Treatment Outcome Data in Subjects With Severe Congenital Protein C Deficiency Who Were Treated With Protein C Concentrate Under an Emergency Use IND

This is a data collection study with the purpose of capturing dosing and treatment outcome data in participants with severe congenital protein C deficiency who were treated with protein C concentrate under an Emergency Use Investigational New Drug Application (IND).

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Actual)

11

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, 90027
        • Children´s Hospital of Los Angeles
    • Colorado
      • Denver, Colorado, United States, 80218
        • Children´s Hospital Denver
      • Fort Carson, Colorado, United States, 80973
        • Evans Army Hospital
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Children´s Hospital Boston
    • Ohio
      • Cleveland, Ohio, United States, 44106
        • Rainbow Babies & Children´s Hospital
      • Dayton, Ohio, United States, 45404
        • The Children´s Medical Center - Dayton
    • Rhode Island
      • Providence, Rhode Island, United States, 02903
        • Rhode Island Hospital
    • Texas
      • Fort Worth, Texas, United States, 76104
        • Cook Children´s Medical Center
      • Galveston, Texas, United States, 77555
        • University of Texas Medical Branch
      • Houston, Texas, United States, 80218
        • Texas Children´s Hospital

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:

  • Participant treated with protein C concentrate under a Baxter Healthcare Corporation Emergency Use IND.
  • Participant with diagnosis of severe congenital protein C deficiency.
  • Participant or parent/legally authorized representative has provided written informed consent.

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

  • Observational Models: Cohort
  • Time Perspectives: Retrospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
All participants
Participants with severe congenital protein C deficiency who were treated under an emergency use IND.
Drug: Protein C Concentrate (Human) Vapor Heated
Protein C Concentrate (Human) Vapor Heated

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Participants who Received a Dose of Protein C in Retrospective Study
Time Frame: Throughout the study period (up to 6 months)
Number of participants who received a dose of Protein C in retrospective study will be reported.
Throughout the study period (up to 6 months)
Number of Participants with Prophylactic Treatment Outcome
Time Frame: Throughout the study period (up to 6 months)
Participants experience any episodes of purpura fulminans (PF), coumarin induced skin necrosis (CISN), or thromboembolic event during prophylactic treatment will be assessed.
Throughout the study period (up to 6 months)
Number of Participants with Acute Episode Treatment Outcome
Time Frame: Throughout the study period (up to 6 months)
Acute episode treatment outcome includes type of episode (PF, CISN, Thromboembolic); Was treatment effective or not effective; Was patient successfully transitioned to anticoagulation following the resolution of the episode will be assessed.
Throughout the study period (up to 6 months)
Number of Participants with Adverse Experiences (AEs) Related to Protein C Concentrate Treatment
Time Frame: Throughout the study period (up to 6 months)
An AE is defined as any untoward medical occurrence in a participant administered study product that does not necessarily have a casual relationship with the treatment. AEs considered possibly or probably related to Protein C Concentrate treatment will be recorded on case report forms (CRFs).
Throughout the study period (up to 6 months)

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.

Helpful Links

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)

June 1, 2005

Primary Completion (Actual)

December 1, 2005

Study Completion (Actual)

December 1, 2005

Study Registration Dates

First Submitted

September 8, 2005

First Submitted That Met QC Criteria

September 8, 2005

First Posted (Estimate)

September 13, 2005

Study Record Updates

Last Update Posted (Actual)

April 8, 2021

Last Update Submitted That Met QC Criteria

April 7, 2021

Last Verified

April 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 400501

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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