1. | | Recruiting | Fludarabine, Low-Dose Total-Body Irradiation, Donor Stem Cell Transplantation, and Immunosuppression Therapy in Treating Patients Who Have Fanconi’s Anemia Conditions: Fanconi's Anemia; adult acute myeloid leukemia; childhood acute myeloid leukemia; Graft Versus Host Disease; Myelodysplastic Syndromes |
2. | | Recruiting | Recombinant Human Factor IX (rFIX; BeneFIX®) in Previously Treated Patients (PTPs) with Hemophilia B Condition: Hemophilia B |
3. | | Recruiting | ReFacto® in Subjects with Hemophilia A Undergoing Major Surgery Condition: Hemophilia A |
4. | | Recruiting | An Investigational Drug Study to Lower Non-Cholesterol Sterol Levels Associated with Sitosterolemia Conditions: Lipid Metabolism, Inborn Errors; Heart Disease |
5. | | Recruiting | Genetic Polymorphisms in the RASSF1A Gene in Children With Wilms' Tumor Conditions: recurrent Wilms' tumor and other childhood kidney tumors; stage I Wilms' tumor; stage II Wilms' tumor; stage III Wilms' tumor; stage IV Wilms' tumor; ... |
6. | | Recruiting | Clinical and Genetic Studies on Holoprosencephaly Condition: Holoprosencephaly |
7. | | Recruiting | Complementary Hyperbaric Oxygen for Brain Tissue Damage Caused by Radiation Therapy Condition: Abnormalities, Radiation Induced |
8. | | Recruiting | Umbilical Cord Blood for Stem Cell Transplantation in Treating Young Patients With Malignant or Nonmalignant Diseases Conditions: Fanconi's Anemia; Cancer |
9. | | Recruiting | Replagal Enzyme Replacement Therapy for Children with Fabry Disease Condition: Fabry Disease |
10. | | Recruiting | Increased Gluconeogenesis is One Cause of Cystic Fibrosis Related Diabetes (CFRD) Conditions: Cystic Fibrosis; Diabetes Mellitus |
11. | | Recruiting | Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry Conditions: Myotonic Dystrophy; Muscular Dystrophy, Facioscapulohumeral; Muscular Dystrophy |
12. | | Recruiting | Natural History of Sickle Cell Disease and Other Hemolytic Disorders Conditions: Hemoglobin SC Disease; Hematologic Diseases |
13. | | Recruiting | Timing of Voluntary Movement in Patients with Tourette Syndrome and Chronic Tic Disorder Conditions: Tourette Syndrome; Tic Disorders |
14. | | Recruiting | A Study of Persons with Gout who Do Not Respond to or Are Allergic to Conventional Therapy Condition: Gout |
15. | | Recruiting | Implitapide in Patients with Heterozygous Familial Hypercholesterolemia (HeFH) on Maximal Concurrent Lipid-Lowering Therapy Condition: Familial Hypercholesterolemia |
16. | | Recruiting | Implitapide in Patients with Homozygous Familial Hypercholesterolemia (HoFH) on Maximal Concurrent Lipid-Lowering Therapy Condition: Familial Hypercholesterolemia |
17. | | Recruiting | Safety and Efficacy Study of Nutropin AQ to Treat Growth Restriction in Children with Cystic Fibrosis Condition: Cystic Fibrosis |
18. | | Recruiting | Phase 1 Trial of Idebenone to Treat Patients with Friedreich's Ataxia Condition: Friedreich Ataxia |
19. | | Recruiting | Genetics and Clinical Characteristics of Bardet-Biedl Syndrome Condition: Bardet-Biedl Syndrome |
20. | | Recruiting | Infant Learning Project Condition: Craniosynostosis |
21. | | Recruiting | Compassionate Use of Stanate (TM) Stannsoporfin Conditions: Neonatal Jaundice; Jaundice; Hyperbilirubinemia |
22. | | Recruiting | Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B Condition: Hemophilia B |
23. | | Recruiting | A Test to Predict the Hemolytic Potential of Drugs in G6PD Deficiency Conditions: Glucosephosphate Dehydrogenase Deficiency; Favism |
24. | | Recruiting | Pirfenidone in Children and Young Adults with Neurofibromatosis type I and Progressive Plexiform Neurofibromas Conditions: Neurofibromatosis 1; Neurofibroma, Plexiform |
25. | | Recruiting | Alternative Dosing and Regimen of Replagal to Treat Fabry Disease Condition: Fabry Disease |
26. | | Recruiting | Family Health After Predictive Huntington Disease (HD) Testing Condition: Huntington Disease |
27. | | Recruiting | Expanded access use of Myozyme (alglucosidase alfa) in patients with infantile-onset Pompe disease Conditions: Glycogen Storage Disease Type II; Glycogenosis 2 |
28. | | Recruiting | Kidney Transplantation in Patients with Cystinosis Condition: Cystinosis |
29. | | Recruiting | Sirolimus to Prevent GVHD after Stem Cell Transplant for Blood Cancers Conditions: Hematologic Neoplasms; Neural Tube Defects; Myeloproliferative Disorders |
30. | | Recruiting | The Research Registry for Neonatal Lupus Conditions: Neonatal lupus; Systemic Lupus Erythematosus; Sjogren's Syndrome; Congenital heart block |
31. | | Recruiting | Safety and Efficacy of Recombinant Adeno-Associated Virus Containing the CFTR Gene in the Treatment of Cystic Fibrosis Condition: Cystic Fibrosis |
32. | | Recruiting | Diabetes Therapy to Improve BMI and Lung Function in CF Conditions: Cystic Fibrosis; Diabetes Mellitus |
33. | | Recruiting | Atorvastatin Therapy to Improve Endothelial Function in Sickle Cell Disease Condition: Sickle Cell Disease |
34. | | Not yet recruiting | Silent Cerebral Infarct Multi-Center Clinical Trial Conditions: Sickle Cell Anemia; Stroke |
35. | | Recruiting | The Role of Susceptibility to Thrombosis in the Pseudotumor Cerebri of Nephropathic Cystinosis: A Case-Control Study Conditions: Pseudotumor Cerebri; Cystinosis |
36. | | Recruiting | An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years with Fabry Disease Condition: Fabry Disease |
37. | | Recruiting | Prenatal Screening For Smith-Lemli-Opitz Syndrome Conditions: Smith-Lemli-Opitz Syndrome; Pregnancy |
38. | | Recruiting | Assessing the Results of Lip Surgery in Patients With Cleft Lip and Palate Conditions: Cleft Lip; Cleft Palate |
39. | | Recruiting | Perinatal Infections in Pakistan Conditions: Bacterial Vaginosis; Fetal Membranes, Premature Rupture; Chorioamnionitis |
40. | | Recruiting | Iron Balance Study of DFO and GT56-252 in Patients with Transfusional Iron Overload Secondary to Beta-Thalassemia Condition: beta-Thalassemia |
41. | | Not yet recruiting | Dextromethorphan and Donepezil Hydrochloride for Rett Syndrome Condition: Rett Syndrome |
42. | | Recruiting | Anakinra to Treat Patients with Neonatal Onset Multisystem Inflammatory Disease Conditions: Nervous System Malformations; Arthropathy, Neurogenic; Urticaria; Papilledema |
43. | | Recruiting | Evaluation of Autosomal Recessive Polycystic Kidney Disease and Congenital Hepatic Fibrosis Condition: Polycystic Kidney, Autosomal Recessive |
44. | | Recruiting | Cardiac Function in Patients with Hereditary Hemochromatosis Condition: Hemochromatosis |
45. | | Not yet recruiting | Clinical Trial to Slow the Progression of ADPKD Conditions: Kidney, Polycystic; Kidney, Polycystic, Autosomal Dominant |
46. | | Recruiting | Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients with MPS VI Condition: Mucopolysaccharidosis VI |
47. | | Recruiting | Home Based Massage and Relaxation for Sickle Cell Pain Condition: Sickle Cell Disease |
48. | | Recruiting | Treatment of Self-Injurious Behavior in Individuals with Prader-Willi Syndrome Conditions: Prader-Willi Syndrome; Self-Injurious Behavior |
49. | | Recruiting | Investigating the Effect of Vitamin A Supplementation on Retinitis Pigmentosa Condition: Retinitis Pigmentosa |
50. | | Recruiting | Follicle Stimulating Hormone (FSH) to Improve Testicular Development in Men with Hypogonadism Conditions: Hypogonadism; Kallmann Syndrome |