Status | Study |
Recruiting |
Study Name: Natural History and Advanced Genetic Study of Pyruvate Dehydrogenase Complex Deficiencies Condition: Pyruvate Dehydrogenase Complex Deficiency Disease Date: 2017-01-27 Interventions: Other: No intervention This is an observational study. The investigators will collect data about exposur |
Enrolling by invitation |
Study Name: Transplantation of Autologous Oral Mucosal Epithelial Stem Cell Sheet for Treating Limbal Stem Cell Deficiency Disease Condition: Limbal Stem Cell Deficiency Date: 2017-01-05 Interventions: Other: cultured autologous oral mucosal epithelial cell sheet a prospective study,Before and after self |
Active, not recruiting |
Study Name: Vitamin D Supplementation in Warfighters Condition: Vitamin D Deficiency Disease Date: 2016-11-02 Interventions: Dietary Supplement: Vitamin D3 As in Arms Other Name |
Completed |
Study Name: Construction of a Health-related Quality of Life (HR-QOL) Questionnaire for Patients With Primary Antibody Deficiency Disease Condition: Immune Deficiency, Antibody Date: 2015-08-31 Interventions: Other: questionnaire survey The HR-QOL PIDD survey will be administered to the patient by the site PI or |
Completed |
Study Name: A Comparison Study of Bypassing Agent Therapy With and Without Tranexamic Acid in Haemophilia A Patients With Inhibitor Condition: Hereditary Factor VIII Deficiency Disease With Inhibitor Date: 2013-02-15 Interventions: Drug: aPCC, aPCC + TXA Feiba 7 |
Recruiting |
Study Name: Evaluate Bacteriophage as a Useful Immunogen in Patients With Primary Immune Deficiency Diseases (PIDD) Condition: Primary Immune Deficiency Diseases Date: 2010-08-05 Interventions: Biological: Bacteriophage OX174 Give first dose of bacteriophage (2 x 109 PFU/kg body weight (0.02 ml/kg |
Withdrawn |
Study Name: Lithium and Acetate for Canavan Disease Condition: Canavan Disease Infantile Deficiency Date: 2008-04-09 Interventions: Drug: Lithium Gluconate (drug) Glyceryl Triacetate GTA (drug) Lithium 1.3 mEq/kg/day (three administrati |
Completed |
Study Name: Safety and Efficacy Study of Flebogamma 5% DIF IGIV in Pediatric Subjects Condition: Primary Immune Deficiency Disease Date: 2008-03-05 Interventions: Biological: Flebogamma 5% DIF Intravenous Immune Globulin (Human) |
Approved for marketing |
Study Name: Alglucosidase Alfa Temporary Access Program Condition: Glycogen Storage Disease Type II (GSD-II) Pompe Disease (Late-Onset) Date: 2007-08-21 Interventions: Biological: alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA]) IV infusion: 20mg/kg q |
Active, not recruiting |
Study Name: Growth and Development Study of Alglucosidase Alfa. Condition: Pompe Disease Glycogen Storage Disease Type II (GSD-II) Ac Date: 2007-06-13 Interventions: Biological: alglucosidase alfa Intravenous (IV) infusion: 20mg/kg every 2 weeks |