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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00157118
Recruitment Status : Completed
First Posted : September 12, 2005
Last Update Posted : October 23, 2017
Sponsor:
Information provided by:
Shire

Tracking Information
First Submitted Date  ICMJE September 8, 2005
First Posted Date  ICMJE September 12, 2005
Last Update Posted Date October 23, 2017
Study Start Date  ICMJE August 2003
Primary Completion Date Not Provided
Current Primary Outcome Measures  ICMJE
 (submitted: July 22, 2013)
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 ]
Original Primary Outcome Measures  ICMJE Not Provided
Change History Complete list of historical versions of study NCT00157118 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Efficacy and Safety Study of Protein C Concentrate in Subjects With Severe Congenital Protein C Deficiency
Official Title  ICMJE 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
Brief 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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Protein C Deficiency
Intervention  ICMJE Drug: Protein C Concentrate (Human) Vapor Heated
Study Arms  ICMJE Not Provided
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)

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: July 22, 2013)
18
Original Enrollment  ICMJE Not Provided
Actual Study Completion Date  ICMJE March 2005
Primary Completion Date Not Provided
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE Child, Adult, Older Adult
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00157118
Other Study ID Numbers  ICMJE 400101
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Not Provided
Study Sponsor  ICMJE Baxalta now part of Shire
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Taru Hays, MD Children´s Hospital, Denver, CO, USA
PRS Account Shire
Verification Date July 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP