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
- Conditions
- Protein C Deficiency
- Interventions
- Drug: Protein C Concentrate (Human) Vapor Heated
- Registration Number
- NCT00161720
- Lead Sponsor
- Baxalta now part of Shire
- Brief Summary
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).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 11
- 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.
Not provided
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description All participants Protein C Concentrate (Human) Vapor Heated Participants with severe congenital protein C deficiency who were treated under an emergency use IND.
- Primary Outcome Measures
Name Time Method Number of Participants who Received a Dose of Protein C in Retrospective Study 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 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 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 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).
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (10)
University of Texas Medical Branch
🇺🇸Galveston, Texas, United States
Texas Children´s Hospital
🇺🇸Houston, Texas, United States
Cook Children´s Medical Center
🇺🇸Fort Worth, Texas, United States
Rainbow Babies & Children´s Hospital
🇺🇸Cleveland, Ohio, United States
Children´s Hospital of Los Angeles
🇺🇸Los Angeles, California, United States
Evans Army Hospital
🇺🇸Fort Carson, Colorado, United States
Children´s Hospital Denver
🇺🇸Denver, Colorado, United States
The Children´s Medical Center - Dayton
🇺🇸Dayton, Ohio, United States
Children´s Hospital Boston
🇺🇸Boston, Massachusetts, United States
Rhode Island Hospital
🇺🇸Providence, Rhode Island, United States