Clinical trials: Protein C Deficiency
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Protein C Deficiency
Ceprotin Treatment Registry
The overall objective is to collect and assess data on the treatment, safety, and treatment outcomes of subjects prescribed, receiving and participating in the Ceprotin treatment registry.
- Trial name
- NCT01127529
- Testing
- Protein C Concentrate (Human)
- Where
- 28 sites in United States, Austria and 4 more
- Volunteers
- 43 people
- Sponsor
- Baxalta now part of Shire
Hemophilia, AIDS
Host Genetic Factors Influencing HIV1 and HCV Viral Loads and AIDS Clinical Progression in a Hemophilia Cohort (HGDS-3)
Background: Over 80% of the hemophiliac population who became infected with HIV prior to 1985 are also co-infected with HCV. Thus, hemophiliacs represent an important population for studies of the natural history of these chronic…
- Trial name
- NCT00340548
- Where
- 15 sites in United States
- Volunteers
- 333 people
- Sponsor
- National Cancer Institute (NCI)
Protein C Deficiency
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
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…
- Trial name
- NCT00161720
- Testing
- Protein C Concentrate (Human) Vapor Heated
- Where
- 10 sites in United States
- Volunteers
- 11 people
- Sponsor
- Baxalta now part of Shire
Protein C Deficiency
Efficacy and Safety Study of Protein C Concentrate in Subjects With Severe Congenital Protein C Deficiency
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.
- Trial name
- NCT00157118
- Testing
- Protein C Concentrate (Human) Vapor Heated
- Where
- 12 sites in United States
- Volunteers
- 18 people
- Sponsor
- Baxalta now part of Shire
Liver Decompensation, Hepatocellular Carcinoma
The Second Multicenter Hemophilia Cohort Study
The Second Multicenter Hemophilia Cohort Study (MHCS-II) will evaluate and prospectively follow approximately 4500 persons with hemophilia who were exposed to hepatitis C virus (HCV).
- Conditions
- Liver Decompensation, Hepatocellular Carcinoma, Non-Hodgkin Lymphoma
- Trial name
- NCT00341705
- Where
- 48 sites in United States, Brazil and 6 more
- Volunteers
- 2,565 people
- Sponsor
- National Cancer Institute (NCI)
Respiratory Distress Syndrome, Newborn
Genetic Regulation of Surfactant Deficiency
Inherited deficiencies in any one of 3 genes (surfactant protein B, surfactant protein C, and ATP-binding cassette transporter A3) can cause neonatal respiratory distress syndrome by disrupting metabolism of the pulmonary…
- Trial name
- NCT00828243
- Testing
- Nutrient
- Where
- United States
- Volunteers
- 525 people
- Sponsor
- Washington University School of Medicine
Protein C Deficiency
A Study of Freeze-dried Human Protein C Concentrate (TAK-662) in Participants With Congenital Protein C Deficiency
This study is conducted in Japan of Freeze-dried Human Protein C Concentrate (TAK-662) used to treat participants with congenital protein C deficiency.
- Trial name
- NCT06590974
- Testing
- Freeze-dried Human Protein C Concentrate
- Where
- Japan
- Volunteers
- 7 people
- Sponsor
- Takeda
Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Generalized Arterial Calcification of Infancy
Natural History Study of ENPP1 Deficiency and the Early-onset Form of ABCC6 Deficiency
The purpose of this prospective study is to characterize the natural history of ENPP1 Deficiency and the early-onset form of ABCC6 Deficiency longitudinally.
- Conditions
- Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Generalized Arterial Calcification of Infancy, Autosomal Recessive Hypophosphatemic Rickets, Adenosine Triphosphate Binding Cassette Transporter Protein Subfamily C Member 6
- Trial name
- NCT05050669
- Testing
- No Intervention for this observational study
- Where
- 5 sites in United States, Canada and 1 more
- Volunteers
- 12 people
- Sponsor
- Inozyme Pharma
Heart Failure, Vitamin D Deficiency
Vitamin D3 Supplementation for Heart Failure Patients
The purpose of this study is to determine if treatment with vitamin D3 of 10,000 International Units (IU) daily in vitamin D deficient patients for 6 months will improve B type natriuretic peptide (BNP), a marker of heart…
- Conditions
- Heart Failure, Vitamin D Deficiency, Cardiomyopathy, Congestive Heart Failure
- Trial name
- NCT01636570
- Testing
- Vitamin D3 (cholecalciferol)
- Where
- 2 sites in United States
- Volunteers
- 40 people
- Sponsor
- Heidi Moretti, MS, RD
HIV Infections, Hepatitis C
Treatment of Hepatitis C in Hemophilic Patients With HIV
Pegylated interferon (PEG-interferon) and ribavirin are accepted treatments for hepatitis C virus (HCV) infection. However, HCV infection progresses differently in patients who are coinfected with HIV and in hemophiliacs.
- Conditions
- HIV Infections, Hepatitis C, Hemophilia
- Trial name
- NCT00055341
- Testing
- Peginterferon alfa-2a, Ribavirin
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Lung Diseases, Respiratory Distress Syndrome, Newborn
Epidemiology of Surfactant Protein-B Deficiency
The purpose of this study is to test the hypothesis that excess, rare, functionally disruptive single nucleotide polymorphisms (SNPs) characterize genes (e.g., the surfactant protein-B gene)(SFTPB) and gene networks (e.g., the…
- Conditions
- Lung Diseases, Respiratory Distress Syndrome, Newborn, Pulmonary Surfactant, Lung Diseases, Interstitial
- Trial name
- NCT00014859
- Where
- United States
- Volunteers
- 5,176 people, healthy volunteers welcome
- Sponsor
- Washington University School of Medicine
Congenital Protein C Deficiency
A Study of TAK-662 for Japanese Patients With Congenital Protein C Deficiency
Pharmacokinetic Part: This study is for Japanese participants with congenital protein C deficiency. The main aim of this study is to check how much TAK-662 stays in their blood over time.
- Trial name
- NCT04984889
- Testing
- TAK-662
- Where
- 4 sites in Japan
- Volunteers
- 5 people
- Sponsor
- Takeda