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

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).

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Key information

Conditions

Protein C Deficiency Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Blood Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Thrombophilia

Sex eligibility

All sexes

Study type

Observational

Primary location

Children´s Hospital of Los Angeles, Los Angeles, California, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Protein C Concentrate (Human) Vapor Heated

Drug

Protein C Concentrate (Human) Vapor Heated

Primary outcomes

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.
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.
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.
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).

Sponsors and collaborators

Lead sponsor

Baxalta now part of Shire

Industry

Registry information

Official study title

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

Important dates

Study start: 2005
Primary completion: 2005
Study completion: 2005
First posted: Sep 13, 2005
Registry last updated: Apr 8, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed