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

Protein C Deficiency Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00157118
• Other study ID #: 400101
• Status: Completed
• Phase: Phase 2/Phase 3
• Start date: August 22, 2003
• Est. completion date: March 17, 2005

Study information

• Verified date: May 2021
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 18
• Est. completion date: March 17, 2005
• Est. primary completion date: March 17, 2005
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Protein C Deficiency

Intervention

Drug:
• Protein C Concentrate (Human) Vapor Heated

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Baxalta now part of Shire

Country where clinical trial is conducted

United States, 

References & Publications (1)

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 S

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.		Part 1 of the study