| 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 |
