Protein C DeficiencyNCT00161720
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
An observational study of Protein C Deficiency, run by Baxalta now part of Shire at 10 sites in United States.
Trial 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). Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Protein C Deficiency
- What is being tested?
- Protein C Concentrate (Human) Vapor Heated
- How many people
- 11 participants
- Where
- 10 sitesUnited States
- Study dates
- 1 Jun 2005to 1 Dec 2005
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
None listed.
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
United States10 sites
Children´s Hospital Boston
Boston, Massachusetts, 02115
Rainbow Babies & Children´s Hospital
Cleveland, Ohio, 44106
The Children´s Medical Center - Dayton
Dayton, Ohio, 45404
Children´s Hospital Denver
Denver, Colorado, 80218
Evans Army Hospital
Fort Carson, Colorado, 80973
Cook Children´s Medical Center
Fort Worth, Texas, 76104
University of Texas Medical Branch
Galveston, Texas, 77555
Texas Children´s Hospital
Houston, Texas, 80218
Children´s Hospital of Los Angeles
Los Angeles, California, 90027
Rhode Island Hospital
Providence, Rhode Island, 02903
About this study
Official 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
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).
- Conditions
- Protein C Deficiency
- Being tested
- Protein C Concentrate (Human) Vapor Heated
- Keywords
- Severe Congenital Protein C Deficiency
- Medical subject headings
- Protein C Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Protein C Concentrate (Human) Vapor HeatedDrug
Protein C Concentrate (Human) Vapor Heated
| Study group | Role | What they receive |
|---|---|---|
| All participants | Participants with severe congenital protein C deficiency who were treated under an emergency use IND. Receives: Drug: Protein C Concentrate (Human) Vapor Heated |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants who Received a Dose of Protein C in Retrospective Study
Number of participants who received a dose of Protein C in retrospective study will be reported.
Measured: Throughout the study period (up to 6 months)
Number of Participants with Prophylactic Treatment Outcome
Participants experience any episodes of purpura fulminans (PF), coumarin induced skin necrosis (CISN), or thromboembolic event during prophylactic treatment will be assessed.
Measured: Throughout the study period (up to 6 months)
Number of Participants with Acute Episode Treatment Outcome
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.
Measured: Throughout the study period (up to 6 months)
Number of Participants with Adverse Experiences (AEs) Related to Protein C Concentrate Treatment
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).
Measured: Throughout the study period (up to 6 months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Sep 2005 (estimated)
- Study start1 Jun 2005
- Primary completion1 Dec 2005
- Study completion1 Dec 2005
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Protein C DeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baxalta now part of Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00161720
- Sponsor's protocol ID
- 400501
- Status last verified
- Apr 2021
- First submitted
- 8 Sep 2005
- Last updated
- 8 Apr 2021
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Apr 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT00161720
- Acronym / sponsor ID
- 400501
- Conditions
- Protein C Deficiency
- Keywords
- Severe Congenital Protein C Deficiency
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 11 participants (actual)
- Lead sponsor
- Baxalta now part of ShireIndustry (pharma, biotech, devices)
- Study start
- 1 Jun 2005 (actual)
- Primary completion
- 1 Dec 2005 (actual)
- Study completion
- 1 Dec 2005 (actual)
- First posted
- 13 Sep 2005 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| All participants | – | Drug: Protein C Concentrate (Human) Vapor HeatedParticipants with severe congenital protein C deficiency who were treated under an emergency use IND. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Protein C Concentrate (Human) Vapor HeatedProtein C Concentrate (Human) Vapor Heated | Drug | All participants |
Primary outcome
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).
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Apr 2021.
- To obtain more information on the study, click here/on this link.Link provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director study director
Takeda
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Protein C Deficiency, run by Baxalta now part of Shire at 10 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Apr 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants who Received a Dose of Protein C in Retrospective Study (time frame: Throughout the study period (up to 6 months)). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Apr 2021; AzTrials last refreshed its copy on 6 Nov 2026.