Biotinidase deficiency Clinical Trials

• Prepubertal Children with Growth Failure Associated with Primary IGF-1 Deficiency
  ClinicalTrials.gov Identifier: NCT00125164 - Active, not recruiting
  Insulin-Like Growth Factor-1 Deficiency; Primary IGF-1 Deficiency
  
• Recombinant Human Insulin-Like Growth Factor (rhIGF-1)Treatment of Short Stature Associated with IGF-1 Deficiency
  ClinicalTrials.gov Identifier: NCT00125190 - Active, not recruiting
  Insulin-Like Growth Factor-1 Deficiency; Primary IGF-1 Deficiency; IGF-1
  
• Gene Therapy for Patients with Leukocyte Adherence Deficiency (Follow-Up of Phase 1 Trial)
  ClinicalTrials.gov Identifier: NCT00023010 - Active, not recruiting
  Leukocyte Adhesion Deficiency Syndrome
  
• 4-PBA: Will it increase the level of Alpha 1-Antitrypsin(AAT) in persons with AAT deficiency?
  ClinicalTrials.gov Identifier: NCT00067756 - Active, not recruiting
  alpha 1-Antitrypsin Deficiency
  
• Phase I Study of Retrovirus-Mediated Transfer of cDNA for Human CD18 into Peripheral Blood Repopulating Cells of Patients With Leukocyte Adherence Deficiency
  ClinicalTrials.gov Identifier: NCT00004470 - Active, not recruiting
  Leukocyte Adhesion Deficiency Syndrome
  
• Open Label Phase 3 Study of NABI-IGIV 10% [(HUMAN)] In Subjects With Primary Immune Deficiency Disorders (PIDD)
  ClinicalTrials.gov Identifier: NCT00538915 - Recruiting
  Primary Immune Deficiency Disorders; Antibody Deficiency Syndromes
  
• Bezafibrate Trial in CPT2 Deficiency
  ClinicalTrials.gov Identifier: NCT00336167 - Recruiting
  Carnitine Palmitoyl Transferase 2 Deficiency
  
• PET Imaging of GABA Receptors in Succinic Semialdehyde Dehydrogenase Deficiency
  ClinicalTrials.gov Identifier: NCT00246870 - Recruiting
  Succinic Semialdehyde Dehydrogenase Deficiency; SSADH Deficiency
  
• Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin Deficiency
  ClinicalTrials.gov Identifier: NCT00377416 - Active, not recruiting
  Alpha 1-Antitrypsin Deficiency
  
• Efficacy and Safety of a High Dosage Compared to the Label Dosage of Somatropin in Early Pubertal Stage Children With Growth Hormone Deficiency
  ClinicalTrials.gov Identifier: NCT00191165 - Active, not recruiting
  Growth Hormone Deficiency
  
• A Multi-Dose Study to Assess Tolerability, Safety and Pharmacology of hGH-ViaDerm™ System in Adults With GH-Deficiency
  ClinicalTrials.gov Identifier: NCT00455260 - Recruiting
  Growth Hormone Deficiency
  
• A Clinical Study of the PK, Efficacy and Safety of Omr-IgG-am IGIV in Subjects With Primary Immune Deficiency Diseases
  ClinicalTrials.gov Identifier: NCT00468273 - Recruiting
  Immunologic Deficiency Syndromes
  
• Ferinject® Assessment in Patients With IRon Deficiency and Chronic Heart Failure (FAIR-HF)
  ClinicalTrials.gov Identifier: NCT00520780 - Recruiting
  Chronic Heart Failure; Iron Deficiency; Iron Deficiency Anemia; Anaemia
  
• Helicobacter Pylori and Iron Deficiency: Prevalence of Association and Effect of Therapy
  ClinicalTrials.gov Identifier: NCT00475527 - Active, not recruiting
  Anemia, Iron-Deficiency; Helicobacter Pylori
  
• Zemaira in Subjects With Emphysema Due to API Deficiency
  ClinicalTrials.gov Identifier: NCT00261833 - Recruiting
  Alpha1-Proteinase Inhibitor Deficiency
  
• Comparative Results of Maxillary Deficiency Treatment by Tongue Plate and Facemask in Growing Patients
  ClinicalTrials.gov Identifier: NCT00519415 - Recruiting
  Orthodontic Angle Class Three; Maxillary Deficiency
  
• A Study of the Efficacy and Safety of Genz-112638 in Type 1 Gaucher Patients
  ClinicalTrials.gov Identifier: NCT00358150 - Active, not recruiting
  Gaucher Disease, Type 1; Cerebroside Lipidosis Syndrome; Glucocerebrosidase Deficiency Disease; Glucosylceramide Beta-Glucosidase Deficiency Disease; Gaucher Disease, Non-Neuronopathic Form
  
• H. Pylori Eradication Trial to Reduce Iron Deficiency in Children
  ClinicalTrials.gov Identifier: NCT00364104 - Recruiting
  Iron Deficiency
  
• Consequence of Lifetime Isolated Growth Hormone Deficiency
  ClinicalTrials.gov Identifier: NCT00149708 - Recruiting
  Growth Hormone Deficiency
  
• Recombinant Human Antithrombin (rhAT) in Patients with Hereditary Antithrombin Deficiency Undergoing Surgery or Delivery
  ClinicalTrials.gov Identifier: NCT00110513 - Recruiting
  Antithrombin III Deficiency
  
• Vitamin D Deficiency, Insulin Resistance and FGF-23
  ClinicalTrials.gov Identifier: NCT00491322 - Recruiting
  Vitamin D Deficiency
  
• Estrogen Deficiency and Cardiovascular Disease in Premenopausal Women
  ClinicalTrials.gov Identifier: NCT00572858 - Recruiting
  Estrogen Deficiency
  
• Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease
  ClinicalTrials.gov Identifier: NCT00364858 - Active, not recruiting
  Gaucher Disease, Type 1; Cerebroside Lipidosis Syndrome; Glucocerebrosidase Deficiency Disease; Glucosylceramide Beta-Glucosidase Deficiency Disease; Gaucher Disease, Non-Neuronopathic Form
  
• A Single Dose Study to Evaluate the PK-PD Response and Safety of PHA-794428 in Children With Growth Hormone Deficiency
  ClinicalTrials.gov Identifier: NCT00314938 - Recruiting
  Growth Hormone Deficiency
  
• A Study To Evaluate The Dose Response And Safety Of PHA-794428 In Adults With Growth Hormone Deficiency
  ClinicalTrials.gov Identifier: NCT00308464 - Recruiting
  Growth Hormone Deficiency
  
• Characterization of Hyperparathyroidism and Vitamin D Deficiency in Obesity
  ClinicalTrials.gov Identifier: NCT00288873 - Active, not recruiting
  Vitamin D Deficiency; Secondary Hyperparathyroidism; Obesity
  
• Molecular and Clinical Studies of Primary Immunodeficiency diseases
  ClinicalTrials.gov Identifier: NCT00006319 - Recruiting
  Immunologic Deficiency Syndrome
  
• rhGH and rhIGF-1 Combination Therapy in Children With Short Stature Associated With IGF-1 Deficiency
  ClinicalTrials.gov Identifier: NCT00572156 - Not yet recruiting
  Insulin-Like Growth Factor-1 Deficiency
  
• Treatment of Adults With Growth Hormone Deficiency
  ClinicalTrials.gov Identifier: NCT00294619 - Recruiting
  Pituitary Disorders; Adult Growth Hormone Deficiency
  
• Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin Deficiency
  ClinicalTrials.gov Identifier: NCT00430768 - Recruiting
  Alpha 1-Antitrypsin Deficiency
  
• The Use of High Resolution Chest Computed Tomography in Alpha-1 Antitrypsin Deficiency
  ClinicalTrials.gov Identifier: NCT00532805 - Recruiting
  Alpha-1 Antitrypsin Deficiency
  
• Pharmacokinetics of Leptin Administration During Fasting
  ClinicalTrials.gov Identifier: NCT00140205 - Active, not recruiting
  energy deficiency due to short-term fasting
  
• Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults with Partial Ornithine Transcarbamylase Deficiency
  ClinicalTrials.gov Identifier: NCT00004498 - Active, not recruiting
  Ornithine Transcarbamylase Deficiency Disease
  
• Safety and Efficacy of PRO 542 in the Treatment of HIV-Infected Patients
  ClinicalTrials.gov Identifier: NCT00055185 - Active, not recruiting
  HIV Infections; Acquired Immune Deficiency Syndrome
  
• Growth Hormone and Chromosome 18q- Abnormal Growth
  ClinicalTrials.gov Identifier: NCT00134420 - Active, not recruiting
  Chromosome 18; Growth Hormone Deficiency
  
• International Collaborative Gaucher Group (ICGG) Gaucher Registry
  ClinicalTrials.gov Identifier: NCT00358943 - Recruiting
  Gaucher Disease; Cerebroside Lipidosis Syndrome; Glucocerebrosidase Deficiency Disease; Glucosylceramide Beta-Glucosidase Deficiency Disease
  
• Dietary Cholesterol and Defects in Cholesterol Synthesis in Mevalonate Kinase Deficiency
  ClinicalTrials.gov Identifier: NCT00260299 - Recruiting
  Mevalonic Aciduria; Mevalonate Kinase Deficiency; Immune System Diseases; Periodic Fever Syndromes, Hereditary; Lipid Metabolism, Inborn Errors
  
• Genetic Basis of Primary Immunodeficiencies
  ClinicalTrials.gov Identifier: NCT00001788 - Recruiting
  Immunologic Deficiency Syndrome
  
• Examining Genetic Differences Among People With 21-Hydroxylase Deficiency
  ClinicalTrials.gov Identifier: NCT00542841 - Recruiting
  21-Hydroxylase Deficiency
  
• The Prevalence of Vitamin D Deficiency and Effects of Vitamin D Supplementation in HIV-1 Infected Patients
  ClinicalTrials.gov Identifier: NCT00306410 - Recruiting
  Vitamin D Deficiency; HIV Infections
  
• Effects of Growth Hormone Administration on Cardiovascular Risk in Cured Acromegalics With Growth Hormone Deficiency
  ClinicalTrials.gov Identifier: NCT00182091 - Recruiting
  Acromegaly; Growth Hormone Deficiency; Pituitary Disease
  
• Iron Supplementation Among Adolescent Girls in India
  ClinicalTrials.gov Identifier: NCT00198848 - Recruiting
  Iron deficiency anemia
  
• Treatment of Anemia in the 2nd Year of Life. Comparison of the Efficacy of Two Different Iron Preparations.
  ClinicalTrials.gov Identifier: NCT00248716 - Recruiting
  Iron Deficiency; Anemia
  
• Growth Hormone Administration and Its Effects on Cardiovascular Risk Factors in Growth Hormone Deficient Women
  ClinicalTrials.gov Identifier: NCT00136032 - Active, not recruiting
  Growth Hormone Deficiency; Pituitary Tumor; Pituitary Disease
  
• Detection and Characterization of Host Defense Defects
  ClinicalTrials.gov Identifier: NCT00001355 - Recruiting
  Immunologic Deficiency Syndrome; Infection
  
• Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies
  ClinicalTrials.gov Identifier: NCT00006054 - Active, not recruiting
  Immunologic Deficiency Syndromes; Chediak-Higashi Syndrome; Common Variable Immunodeficiency; Graft Versus Host Disease; ...
  
• Treatment of Children and Adolescents With Growth Failure Associated With Primary Insulin-Like Growth Factor-1 (IGF-1) Deficiency
  ClinicalTrials.gov Identifier: NCT00330668 - Recruiting
  Primary IGF-1 Deficiency; Growth Disorders
  
• The Effect of Early Iron Deficiency on Developmental Brain and Behavior in Infants
  ClinicalTrials.gov Identifier: NCT00515918 - Not yet recruiting
  Iron Deficiency
  
• Ig NextGen 10% in Patients With Primary Immune Deficiency (PID)
  ClinicalTrials.gov Identifier: NCT00405184 - Recruiting
  Primary Immune Deficiency (PID)
  
• Zinc Deficiency in HIV - 1
  ClinicalTrials.gov Identifier: NCT00149552 - Active, not recruiting
  HIV; alcohol & drug use; zinc; zinc deficiency
  
• Prevention of Iron Deficiency in 2nd Year of Life
  ClinicalTrials.gov Identifier: NCT00479102 - Not yet recruiting
  Iron Deficiency; Anemia
  
• Effects of Growth Hormone on Glucose and Protein Metabolism in Children With Growth Hormone Deficiency
  ClinicalTrials.gov Identifier: NCT00362063 - Recruiting
  Growth Hormone Deficiency
  
• Use of Labeled Glucose to Study Lymphocyte Replication and Survival in HIV-Infected Patients
  ClinicalTrials.gov Identifier: NCT00001651 - Recruiting
  HIV Infection; Immunologic Deficiency Syndrome
  
• Endocrine Dysfunction and Growth Hormone Deficiency in Children with Optic Nerve Hypoplasia.
  ClinicalTrials.gov Identifier: NCT00140413 - Recruiting
  Optic Nerve Hypoplasia; Growth Hormone Deficiency; Septo-Optic dysplasia; Hypopituitarism
  
• Growth Hormone Deficiency in Chronic Heart Failure: an Observational Study
  ClinicalTrials.gov Identifier: NCT00511927 - Recruiting
  Ischemic Heart Disease; Dilative Cardiomyopathy; Heart Failure; Growth Hormone Deficiency
  
• Prevention of Vitamin D Deficiency
  ClinicalTrials.gov Identifier: NCT00494104 - Recruiting
  Vitamin D Deficiency
  
• Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)
  ClinicalTrials.gov Identifier: NCT00410566 - Recruiting
  Acid Sphingomyelinase Deficiency; Niemann-Pick Disease
  
• Study of Genetic and Molecular Defects in Primary Immunodeficiency Disorders
  ClinicalTrials.gov Identifier: NCT00004341 - Active, not recruiting
  X-linked agammaglobulinemia; X-linked hyper IgM syndrome; Wiskott-Aldrich Syndrome; Leukocyte Adhesion Deficiency Syndrome
  
• Investigation of the Human Immune Response in Normal Subjects and Patients with Disorders of the Immune System and Cancer
  ClinicalTrials.gov Identifier: NCT00001582 - Recruiting
  Communicable Disease; Immunologic Deficiency Syndrome; Neoplasm
  
• Interest of Biofer ® Compound Used in Blédilait Follow on Milk in Infant With Latent Iron Deficiency
  ClinicalTrials.gov Identifier: NCT00554814 - Not yet recruiting
  Latent Iron Deficiency
  
• Iodine Status in Pregnant Women and Their Newborns: is Congenital Hypothyroidism Related to Iodine Deficiency in Pregnancy?
  ClinicalTrials.gov Identifier: NCT00505479 - Recruiting
  Congenital Hypothyroidism; Pregnancy; Iodine Deficiency
  
• Rold of Leptin in the Neuroendocrine and Immune Response to Fasting
  ClinicalTrials.gov Identifier: NCT00140231 - Recruiting
  energy deficiency associated with short-term fasting
  
• Aralast alpha1-Proteinase Inhibitor Surveillance Study
  ClinicalTrials.gov Identifier: NCT00313144 - Active, not recruiting
  Alpha 1-Antitrypsin Deficiency
  
• Somatropin Treatment in Patients With SHOX Deficiency and Turner Syndrome
  ClinicalTrials.gov Identifier: NCT00190658 - Active, not recruiting
  Failure to Thrive
  
• Predictive Markers in GHD and TS Children Treated With SAIZEN®
  ClinicalTrials.gov Identifier: NCT00256126 - Active, not recruiting
  Growth Hormone Deficiency
  
• Efficacy and Safety of Testogel® in Men With Partial Androgen Deficiency of Aging Males (PADAM)
  ClinicalTrials.gov Identifier: NCT00185198 - Active, not recruiting
  Hypogonadism
  
• A Trial of the Pharmacokinetics, Safety, and Tolerability of Subcutaneous Gamunex® in Primary Immunodeficiency
  ClinicalTrials.gov Identifier: NCT00389324 - Active, not recruiting
  Immunologic Deficiency Syndrome
  
• Iron Therapy in Colo-Rectal Neoplasm and Iron Deficiency Anemia: Intravenous Iron Sucrose Versus Oral Ferrous Sulphate.
  ClinicalTrials.gov Identifier: NCT00199277 - Not yet recruiting
  Colorectal Neoplasm; Iron deficiency anemia
  
• Relationship Between Folic Acid and Warfarin Metabolism and Effect
  ClinicalTrials.gov Identifier: NCT00162409 - Recruiting
  Folic Acid Deficiency
  
• Does Treatment of Hypovitaminosis D Increase Calcium Absorption?
  ClinicalTrials.gov Identifier: NCT00581828 - Active, not recruiting
  Osteoporosis; Osteopenia; Vitamin D Deficiency; Hypoparathyroidism; Hypercalciuria; Hypercalcemia
  
• Randomized, Double-Blind, Placebo-Controlled Study of the Safety, Efficacy and Pharmacokinetics of Myozyme in Patients with Late-Onset Pompe Disease.
  ClinicalTrials.gov Identifier: NCT00158600 - Active, not recruiting
  Pompe Disease (Late-onset); Glycogen storage disease type II (GSD-II); Acid Maltase Deficiency Disease; Glycogenosis 2
  
• Gastrointestinal Evaluation in Young Men With Iron Deficiency Anemia
  ClinicalTrials.gov Identifier: NCT00498511 - Not yet recruiting
  Anemia, Iron-Deficiency
  
• Safety and Efficacy of Intravenous Immunoglobulin IgPro10 in Patients With Primary Immunodeficiencies (PID)
  ClinicalTrials.gov Identifier: NCT00322556 - Active, not recruiting
  Agammaglobulinemia; IgG Deficiency; Common Variable Immunodeficiency
  
• Umbilical Cord Clamping and Infant Iron Status
  ClinicalTrials.gov Identifier: NCT00298051 - Active, not recruiting
  Iron-Deficiency Anemia
  
• Sunlight Exposures Effect on Serum Vitamin D Levels
  ClinicalTrials.gov Identifier: NCT00473317 - Active, not recruiting
  Vitamin D Deficiency
  
• Single-Dose Postpartum Vitamin A Supplementation of Mothers and Neonates
  ClinicalTrials.gov Identifier: NCT00198718 - Active, not recruiting
  Vitamin A Deficiency; HIV
  
• Vitamin D Administration in the Nursing Home
  ClinicalTrials.gov Identifier: NCT00204919 - Active, not recruiting
  - Vitamin D Deficiency; - Osteoporosis
  
• Vitamin D Deficiency Causes Immune Dysfunction and Enables or Perpetuates the Development of Rheumatoid Arthritis
  ClinicalTrials.gov Identifier: NCT00279461 - Not yet recruiting
  Arthritis, Rheumatoid; Vitamin D Deficiency
  
• A Study to Assess the Effectiveness of an Investigational Drug in Elderly Subjects With Low Vitamin D Levels.
  ClinicalTrials.gov Identifier: NCT00242476 - Active, not recruiting
  Vitamin D Deficiency
  
• Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency
  ClinicalTrials.gov Identifier: NCT00355264 - Active, not recruiting
  Tetrahydrobiopterin Deficiencies; Hyperphenylalaninemia, Non-Phenylketonuric
  
• Effect of Vitamin C on Iron Absorption
  ClinicalTrials.gov Identifier: NCT00570895 - Enrolling by invitation
  Iron Deficiency
  
• Immune Regulation in Patients with Common Variable Immunodeficiency and Related Syndromes
  ClinicalTrials.gov Identifier: NCT00001244 - Recruiting
  Agammaglobulinemia; Common Variable Immunodeficiency; Immunologic Deficiency Syndrome
  
• Genetic Analysis of Immune Disorders
  ClinicalTrials.gov Identifier: NCT00001467 - Recruiting
  Healthy; Immunologic Deficiency Syndrome; Job's Syndrome; Severe Combined Immunodeficiency
  
• Testosterone and Growth Hormone for Bone Loss in Men
  ClinicalTrials.gov Identifier: NCT00080483 - Recruiting
  Hypopituitarism; Hypogonadism; Growth Hormone Deficiency
  
• Evaluation of rhGH Replacement Therapy in Patients With Pseudohypoparathyroidism Type Ia (PHP Ia)
  ClinicalTrials.gov Identifier: NCT00497484 - Active, not recruiting
  Pseudohypoparathyroidism; Growth Hormone Deficiency, Dwarfism
  
• Growth Hormone and Endothelial Function in Children
  ClinicalTrials.gov Identifier: NCT00373386 - Active, not recruiting
  Growth Hormone Deficiency; Panhypopituitarism; Short Stature
  
• Assessment of Anaemia Attributable to Schistosomiasis in School Children in Kenya: Mechanisms and Effect of Treatment
  ClinicalTrials.gov Identifier: NCT00414479 - Active, not recruiting
  Anaemia; Schistosomiasis Infection; Malaria; Iron Deficiency
  
• Effect of Intravenous Ferrous Sucrose on Exercise Capacity in Chronic Heart Failure
  ClinicalTrials.gov Identifier: NCT00125996 - Recruiting
  Chronic Heart Failure; Anaemia; Iron Deficiency
  
• Cortical Excitability in SSADH Deficiency Patients
  ClinicalTrials.gov Identifier: NCT00132366 - Recruiting
  
  
• Penicillamine Chelation for Children With Lead Poisoning
  ClinicalTrials.gov Identifier: NCT00552630 - Active, not recruiting
  Lead Poisoning; Vitamin D Deficiency
  
• Optimization of Selenoprotein P in Chinese Subjects
  ClinicalTrials.gov Identifier: NCT00428649 - Recruiting
  Selenium Deficiency
  
• Iron Deficiency in Single Ventricle Patients
  ClinicalTrials.gov Identifier: NCT00459225 - Recruiting
  Anemia
  
• Use of G-CSF to Obtain Blood Cell Precursors
  ClinicalTrials.gov Identifier: NCT00001405 - Recruiting
  Chronic Granulomatous Disease; Healthy; Immunologic Disease; Leukocyte Adhesion Deficiency Syndrome; Severe Combined Immunodeficiency
  
• Mutation Analysis of 17βhydroxysteroid Dehydrogenase 3 Deficiency
  ClinicalTrials.gov Identifier: NCT00173654 - Recruiting
  Pseudohermaphroditism
  
• Relative Bioavailability of Ferric Pyrophosphate From an Apple Juice Drink
  ClinicalTrials.gov Identifier: NCT00474682 - Recruiting
  Iron Deficiency
  
• GTA-Glyceryltriacetate for Canavan Disease
  ClinicalTrials.gov Identifier: NCT00278707 - Active, not recruiting
  Infantile Canavan Disease; Deficiency Disease, Aspartoacylase
  
• Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome
  ClinicalTrials.gov Identifier: NCT00001596 - Recruiting
  Albinism; Inborn Errors of Metabolism; Oculocutaneous Albinism; Platelet Storage Pool Deficiency; Pulmonary Fibrosis
  
• Efficacy and Safety Study of Protein C Concentrate in Subjects With Severe Congenital Protein C Deficiency
  ClinicalTrials.gov Identifier: NCT00157118 - Completed
  Protein C Deficiency
  
• Treatment of Deficient Subclass or Anti-Polysaccharide Antibody Response
  ClinicalTrials.gov Identifier: NCT00522821 - Recruiting
  IgG Deficiency; Infections
  
• Late-Onset Treatment Study Extension Protocol
  ClinicalTrials.gov Identifier: NCT00455195 - Active, not recruiting
  Pompe Disease (Late-Onset); Glycogen Storage Disease Type II (GSD-II); Glycogenesis 2; Acid Maltase Deficiency