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

This study has been completed.
Sponsor:
Information provided by:
Baxter Healthcare Corporation
ClinicalTrials.gov Identifier:
NCT00157118
First received: September 8, 2005
Last updated: July 22, 2013
Last verified: July 2013
  Purpose

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.


Condition Intervention Phase
Protein C Deficiency
Drug: Protein C Concentrate (Human) Vapor Heated
Phase 2
Phase 3

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

Resource links provided by NLM:


Further study details as provided by Baxter Healthcare Corporation:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: No ]

Enrollment: 18
Study Start Date: August 2003
Study Completion Date: March 2005
  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00157118

Locations
United States, California
Los Angeles, California, United States
United States, Colorado
Denver, Colorado, United States
United States, Georgia
Atlanta, Georgia, United States
United States, Indiana
Indianapolis, Indiana, United States
United States, Kentucky
Lexington, Kentucky, United States
United States, Massachusetts
Boston, Massachusetts, United States
United States, Ohio
Cincinnati, Ohio, United States
Cleveland, Ohio, United States
Dayton, Ohio, United States
United States, Texas
Fort Worth, Texas, United States
Galveston, Texas, United States
Houston, Texas, United States
Sponsors and Collaborators
Baxter Healthcare Corporation
Investigators
Principal Investigator: Taru Hays, MD Children´s Hospital, Denver, CO, USA
  More Information

No publications provided

ClinicalTrials.gov Identifier: NCT00157118     History of Changes
Other Study ID Numbers: 400101
Study First Received: September 8, 2005
Last Updated: July 22, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Baxter Healthcare Corporation:
Severe congenital protein C deficiency

Additional relevant MeSH terms:
Protein C Deficiency
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Blood Protein Disorders
Thrombophilia
Genetic Diseases, Inborn
Protein C
Anticoagulants
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions
Fibrinolytic Agents
Fibrin Modulating Agents
Molecular Mechanisms of Pharmacological Action
Cardiovascular Agents

ClinicalTrials.gov processed this record on September 30, 2014