Clinical Trials for X-linked Genetic Diseases

Clinical trials are research studies conducted in an effort to improve overall patient health and care. Each trial involves running supervised tests to determine the effectiveness and safety of new drugs, procedures and/or devices with the aim of answering scientific questions about a disease or condition. Risks and side-effects are also evaluated during these trials with results ranging from being unpleasant, with side effects such as headaches, to more serious or even life-threatening risks. A clinical trial may be separated into phases, or steps, with each step designed to answer a separate research question. This maybe reflected in the trial name.

A brief description of each phase:

• Phase I: Usually designed to evaluate safety, determine a safe dosage range, and identify side effects on a small group of patients.
• Phase II: If Phase I is successful, the trial is then repeated with a larger group to further evaluate its effect and safety.
• Phase III: Trials are conducted on an ever larger group and are compared with the best current treatment while gathering more information on effect and safety.
• Phase IV: These studies monitor long-term side effects after the treatment has been marketed.

Clinical Trials data for X-linked Genetic Diseases is grouped into the following categories:-

• View by trial name
• View by drugs used in X-linked Genetic Diseases clinical trials

Clinical Trials by trial name

The format for each of the following is:

• The name of the trial,
• followed by whether the trial is recruiting patients,
  - The trial may not have started and not yet recruiting, or the trial may have started and do not need any more recruitements, or the trial may be completed.
  - Note**: Please follow the link to determine the current trial status.
• followed by a list of drug/s, if any, used in the trial

ClinicalTrials.gov has listed the following trials for X-linked Genetic Diseases:

• Genetic Studies of X-Linked Lymphoproliferative Disease - This study is currently recruiting patients (Current: 23 Nov 2006)
• DHA and X-Linked Retinitis Pigmentosa - This study is currently recruiting patients (Current: 23 Nov 2006) - docosahexaenoic acid
• Copper Histidine Therapy for Menkes Diseases - This study is currently recruiting patients (Current: 23 Nov 2006) - Copper Histidine
• A Phase IV Study of Recombinant Human Gamma Interferon in Patients With Chronic Granulomatous Diseases of Childhood - This study has been completed (Current: 23 Nov 2006)
• Use of G-CSF to Obtain Blood Cell Precursors - This study is currently recruiting patients (Current: 23 Nov 2006)
• Gene Therapy for Chronic Granulomatous Diseases - Long-Term Follow-up - This study is no longer recruiting patients (Current: 23 Nov 2006) - Gene Therapy Method for CGD
• Analysis of the Nervous System in Patients With Fabry's Disease - This study is currently recruiting patients (Current: 23 Nov 2006)
• Diagnostic Effectiveness of Virtual Bronchoscopy - This study has been completed (Current: 23 Nov 2006)
• Stem Cell Transplant Following Low-Intensity Chemotherapy to Treat Chronic Granulomatous Disease - This study has been completed (Current: 23 Nov 2006)
• Vasodilation in Patients With Fabry's Disease - This study has been completed (Current: 23 Nov 2006)
• Phase II Pilot Study of Aminoimidazole Carboxamide Riboside (AICAR), a Precursor of Purine Synthesis, for Lesch-Nyhan Disease - This study has been completed (Current: 23 Nov 2006) - aminomidazole carboxamide riboside
• Study of Genetic and Molecular Defects in Primary Immunodeficiency Disorders - This study is no longer recruiting patients (Current: 23 Nov 2006)
• Study of Glyceryl Trierucate and Glyceryl Trioleate (Lorenzo's Oil) Therapy in Male Children With Adrenoleukodystrophy - This study is currently recruiting patients (Current: 23 Nov 2006) - glyceryl trierucate,glyceryl trioleate
• Study of Bile Acids in Patients With Peroxisomal Disorders - This study is not yet open for patient recruitment (Current: 23 Nov 2006) - chenodeoxycholic acid,cholic acid,ursodiol
• Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy - This study has been completed (Current: 23 Nov 2006) - glyceryl trierucate,glyceryl trioleate,interferon beta,thalidomide
• Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome) - This study has been completed (Current: 23 Nov 2006)
• Phase III Randomized, Double-Blind Study of Prednisone for Duchenne Muscular Dystrophy - This study has been completed (Current: 23 Nov 2006) - prednisone
• Nutritional Aspects of Rett Syndrome - This study has been completed (Current: 23 Nov 2006)
• Study of Cardiac and Paroxysmal Abnormalities in Rett Syndrome - This study has been completed (Current: 23 Nov 2006)
• Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes - This study has been completed (Current: 23 Nov 2006) - filgrastim
• Phase I/II Study of Monoclonal Factor IX Concentrate for Factor IX Deficiency - This study has been completed (Current: 23 Nov 2006) - monoclonal factor IX replacement therapy
• Study of the Pathogenesis of Rett Syndrome - This study has been completed (Current: 23 Nov 2006) - dextromethorphan,topiramate,Donepezil
• PET Scans in Normal Volunteers and Patients With Fabry Disease - This study has been completed (Current: 23 Nov 2006)
• Gentamicin Treatment of Muscular Dystrophy - This study has been completed (Current: 23 Nov 2006)
• Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation - This study is no longer recruiting patients (Current: 23 Nov 2006)
• Learning and Behavior Problems in Children With Chronic Granulomatous Disease and Related Disorders - This study has been completed (Current: 23 Nov 2006)
• Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies - This study is no longer recruiting patients (Current: 23 Nov 2006) - anti-thymocyte globulin,busulfan,cyclophosphamide,cyclosporine,etoposide,methotrexate,methylprednisolone,prednisone
• Modified Stem Cell Transplantation Procedure for Treating Chronic Granulomatous Disease - This study has been completed (Current: 23 Nov 2006)
• Study of Oral Cholic Acid in Patients With Inborn Errors of Bile Acid Synthesis - This study has been completed (Current: 23 Nov 2006)
• Pilot Study of Allogeneic Bone Marrow Transplantation Plus Cyclosporine and Mycophenolate Mofetil to Induce Mixed Hematopoietic Chimerism in Patients With Primary T-Cell Immunodeficiency Disorders - This study is currently recruiting patients (Current: 23 Nov 2006) - cyclosporine,mycophenolate mofetil
• Study of Total Body Irradiation and Fludarabine Followed By Allogeneic Peripheral Blood Stem Cell or Bone Marrow Transplantation in Combination With Cyclosporine and Mycophenolate Mofetil in Patients With Inherited Disorders - This study is currently recruiting patients (Current: 23 Nov 2006) - cyclosporine,fludarabine,mycophenolate mofetil
• Creatine and Glutamine in Steroid-Naive Duchenne Muscular Dystrophy - This study is no longer recruiting patients (Current: 23 Nov 2006) - Creatine Monohydrate,Glutamine
• A Multicenter Randomized Placebo-Controlled Double-Blind Study to Assess Efficacy and Safety of Glutamine and Creatine Monohydrate in Duchenne Muscular Dystrophy (DMD) - This study has been completed (Current: 23 Nov 2006) - glutamine,creatine monohydrate
• Treatment of Chronic Granulomatous Disease With Allogeneic Stem Cell Transplantation Versus Standard of Care - This study has been completed (Current: 23 Nov 2006) - T-Cell Depleted & CD34+Select/w/StemCell Enriched Product
• Data Collection in Women With Fabry Disease - This study has been completed (Current: 23 Nov 2006)
• An Open-Label Pilot Study of Coenzyme Q10 in Steroid-Treated Duchenne Muscular Dystrophy - This study is no longer recruiting patients (Current: 23 Nov 2006) - Coenzyme Q10
• KUL0401: An Open-Label Pilot Study of Oxatomide in Steroid-Naive Duchenne Muscular Dystrophy - This study is no longer recruiting patients (Current: 23 Nov 2006) - Oxatomide (tinset)
• Posaconazole to Treat Invasive Fungal Infections - This study is currently recruiting patients (Current: 23 Nov 2006) - Posaconazole
• Study Evaluating rFIX; BeneFIX in Severe Hemophilia B - This study is currently recruiting patients (Current: 23 Nov 2006) - BeneFIX
• Alpha-Galactosidase A Replacement Therapy for Fabry Disease - This study has been completed (Current: 23 Nov 2006) - DRX005B
• Effects of CX516 on Functioning in Fragile X Syndrome and Autism - This study has been completed (Current: 23 Nov 2006) - CX516 (Ampalex®)
• Registry of Fabry Disease - A Multicenter Observational Study - This study is currently recruiting patients (Current: 23 Nov 2006)
• Follicle Stimulating Hormone (FSH) to Improve Testicular Development in Men With Hypogonadism - This study is currently recruiting patients (Current: 23 Nov 2006) - gonadotropin releasing hormone (GnRH),follicle stimulating hormone (FSH)
• Dosing Study of Replagal in Patients With Fabry Disease - This study is no longer recruiting patients (Current: 23 Nov 2006) - Replagal
• Independent Studies of Dextromethorphan and of Donepezil Hydrochloride for Rett Syndrome - This study is currently recruiting patients (Current: 23 Nov 2006) - dextromethorphan,donepezil hydrochloride
• Iduronate-2-Sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II) - This study is no longer recruiting patients (Current: 23 Nov 2006) - Iduronate-2-sulfatase enzyme replacement therapy
• An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years With Fabry Disease - This study has been completed (Current: 23 Nov 2006) - Replagal
• National Registry for Ichthyosis and Related Disorders - This study is no longer recruiting patients (Current: 23 Nov 2006)
• A Study of Fabrazyme in Pediatric Patients With Fabry Disease - This study has been completed (Current: 23 Nov 2006) - Fabrazyme (agalsidase beta)
• A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease - This study has been completed (Current: 23 Nov 2006) - Fabrazyme (agalsidase beta)
• A Study of the Safety and Efficacy of Fabrazyme as Compared to Placebo in Patients With Advanced Fabry Disease - This study has been completed (Current: 23 Nov 2006) - Fabrazyme (agalsidase beta)
• Alternative Dosing and Regimen of Replagal to Treat Fabry Disease - This study has been completed (Current: 23 Nov 2006) - Replagal
• Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B - This study is currently recruiting patients (Current: 23 Nov 2006)
• A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease - This study has been completed (Current: 23 Nov 2006) - Fabrazyme (agalsidase beta)
• Replagal Enzyme Replacement Therapy for Children With Fabry Disease - This study is no longer recruiting patients (Current: 23 Nov 2006) - Replagal (Galactosidase Alfa)
• Study Evaluating rFIX; BeneFIX in Hemophilia B - This study is no longer recruiting patients (Current: 23 Nov 2006) - rFIX
• Study Evaluating of Recombinant Human Factor IX (BeneFIX) and a New Formulation of BeneFIX (rFIX-R) in Moderate to Severe Hemophilia B - This study is no longer recruiting patients (Current: 23 Nov 2006) - rFIX,rFIX-R
• Replagal Enzyme Replacement Therapy for Adults With Fabry Disease - This study has been completed (Current: 23 Nov 2006) - Replagal (Agalsidase Alfa),Replagal
• Pentoxifylline in Duchenne Muscular Dystrophy - This study is no longer recruiting patients (Current: 23 Nov 2006) - Pentoxifylline
• Study Evaluating MYO-029 in Adult Muscular Dystrophy - This study is no longer recruiting patients (Current: 23 Nov 2006) - MYO-029
• Study to Collect Data on Fabry Disease Patients With Enhanceable Alpha-Galactosidase A Activity - This study is currently recruiting patients (Current: 23 Nov 2006)
• Efficacy of NovoSeven in Bleeding Prophylaxis in Hemophilia - This study is no longer recruiting patients (Current: 23 Nov 2006) - Activated Recombinant FVII (NovoSeven®)
• High-Dose Prednisone in Duchenne Muscular Dystrophy - This study is currently recruiting patients (Current: 23 Nov 2006) - Prednisone
• Post Marketing Study in Haemophilia B Patients Using Nonafact (Human Coagulation Factor IX) - This study is no longer recruiting patients (Current: 23 Nov 2006) - human coagulation Factor IX
• A Safety and Efficacy Study of Fabrazyme Replacement Therapy in Patients With Cardiac Fabry Disease - This study is currently recruiting patients (Current: 23 Nov 2006) - Agalsidase beta (recombinant form)
• Enzyme Replacement Therapy in Fabry Disease - This study is currently recruiting patients (Current: 23 Nov 2006)
• Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy - This study is not yet open for patient recruitment (Current: 23 Nov 2006)
• Haploidentical Hematopoietic Stem Cell Transplantation (HSCT) for Pediatric Patients With Wiskott-Aldrich Syndrome: A Pilot Study - This study is currently recruiting patients (Current: 23 Nov 2006)
• Prospective Registry of European Hemophilia B Patients Receiving BeneFIX for Usual Use - This study is currently recruiting patients (Current: 23 Nov 2006)
• Neuropathic Pain and Fabry Disease - This study is currently recruiting patients (Current: 23 Nov 2006)
• Stem Cell Transplant for Inborn Errors of Metabolism - This study is currently recruiting patients (Current: 23 Nov 2006) - Busulfan,Cyclophosphamide,ATG
• Study to Describe the Allergic Reactions to Factor IX in Patients With Hemophilia B - This study is currently recruiting patients (Current: 23 Nov 2006)
• A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease - This study is no longer recruiting patients (Current: 23 Nov 2006) - Fabrazyme (agalsidase beta)
• Fabry Disease Registry - This study is currently recruiting patients (Current: 23 Nov 2006) - Fabrazyme (agalsidase beta)
• Test-Retest Reliability of Pulmonary Function Tests in Patients With Duchenne's Muscular Dystrophy - This study is currently recruiting patients (Current: 23 Nov 2006)
• The Effect of Patient Counseling on Adolescent Hemophilia Patient Compliance With Bleeding Logs - This study is no longer recruiting patients (Current: 23 Nov 2006)
• A 12-Week Safety, Pharmacokinetic, and Pharmacodynamic Study of AT1001 in Patients With Fabry Disease - This study is currently recruiting patients (Current: 23 Nov 2006) - AT1001 (migalastat hydrochloride)
• An Open-Label Trial of Donepezil in Fragile X Syndrome - This study is currently recruiting patients (Current: 23 Nov 2006) - Donepezil
• Rapid Infusion of Immune Globulin Intravenous (Human) In Primary Immunodeficiency Patients - This study has been completed (Current: 23 Nov 2006) - Immune Globulin Intravenous (Human) , 10%
• A Study of the Effects of Fabrazyme on Mother's Lactation and on the Growth, Development and Immunologic Response of Their Infants. - This study is currently recruiting patients (Current: 23 Nov 2006) - Fabrazyme (agalsidase beta)
• AT1001 to Treat Fabry Disease - This study is currently recruiting patients (Current: 23 Nov 2006) - AT1001
• A Long Term Safety and Efficacy Study of Fabrazyme Replacement Therapy in Japanese Patients With Fabry Disease. - This study is currently recruiting patients (Current: 23 Nov 2006) - Agalsidase beta (recombinant form)
• Study Evaluating Allergic Reactions to Benefix in Hemophilia B Patients - This study is currently recruiting patients (Current: 23 Nov 2006)
• A Study of the Effectiveness and Safety of Risperidone Versus Placebo in the Treatment of Children With Autistic Disorder and Other Pervasive Developmental Disorders (PDD) - This study is no longer recruiting patients (Current: 23 Nov 2006) - risperidone
• Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy - This study is currently recruiting patients (Current: 23 Nov 2006) - PTC124
• Clinical Study and Gene Mutation Analysis of Adrenoleukodystrophy in Taiwanese Children - This study is currently recruiting patients (Current: 23 Nov 2006)
• Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases. - This study is currently recruiting patients (Current: 23 Nov 2006) - Intravenous immunoglobulin
• A 24-Week Safety and Pharmacodynamic Study of AT1001 in Patients With Fabry Disease - This study is currently recruiting patients (Current: 23 Nov 2006) - AT1001 (migalastat hydrochloride)
• A 12-Week Safety and Pharmacodynamic Study of AT1001 in Patients With Fabry Disease - This study is not yet open for patient recruitment (Current: 23 Nov 2006) - AT1001 (migalastat hydrochloride)
• Effect of Oral Glutamine on Muscle Mass and Function in Duchenne Muscular Dystrophy - This study is currently recruiting patients (Current: 23 Nov 2006) - L-Glutamine
• Genetic and Physical Characteristics of Rett Syndrome - This study is currently recruiting patients (Current: 23 Nov 2006)
• A 12-Week Safety and Pharmacodynamic Study of AT1001 in Female Patients With Fabry Disease - This study is currently recruiting patients (Current: 23 Nov 2006) - AT1001 (migalastat hydrochloride)
• CoQ10 and Prednisone in Non-Ambulatory DMD - This study is currently recruiting patients (Current: 23 Nov 2006) - prednisone,CoQ10
• Biomechanical Analysis of Gait in Individuals With Duchenne Muscular Dystrophy - This study is currently recruiting patients (Current: 23 Nov 2006)
• A Study in Patients With Fabry Disease Who Are on Chronic Hemodialysis Therapy for Treatment of End-Stage Renal Insufficiency. - This study is currently recruiting patients (Current: 23 Nov 2006) - Fabrazyme (agalsidase beta)
• Infliximab to Treat Crohn's-Like Inflammatory Bowel Disease in Chronic Granulomatous Disease - This study is currently recruiting patients (Current: 23 Nov 2006) - Infliximab
• Antiproteinuric Agents and Fabry Disease - This study has been completed (Current: 23 Nov 2006)
• Letrozole Treatment in Normal and GnRH Deficient Women - This study is currently recruiting patients (Current: 23 Nov 2006) - Letrozole,NAL-GLU GnRH antagonist,GnRH
• An Open-Label Maintenance Study of the Enzyme Replacement Therapy Replagal in Patients With Fabry Disease - This study is no longer recruiting patients (Current: 23 Nov 2006) - Replagal agalsidase alfa,Replagal
• Genetic Analysis of Oculocerebrorenal Syndrome of Lowe - This study is no longer recruiting patients (Current: 23 Nov 2006)
• Protein Synthesis in the Brain of Patients With Fragile X Syndrome - This study is currently recruiting patients (Current: 23 Nov 2006)
• Study Comparing On-Demand Treatment With Two Prophylaxis Regimens of BeneFIX in Patients With Severe Hemophilia B - This study is not yet open for patient recruitment (Current: 23 Nov 2006) - BeneFIX
• HSCT for High Risk Inherited Inborn Errors - This study is currently recruiting patients (Current: 23 Nov 2006) - Campath,Clofarabine
• Molecular Analysis of Patients With Neuromuscular Disease - This study is currently recruiting patients (Current: 23 Nov 2006)
• Investigating the Regulation of Reproductive Hormones in Adult Men - This study is currently recruiting patients (Current: 23 Nov 2006) - ketoconazole,gonadotropin releasing hormone (GnRH)
• Gene Therapy for Chronic Granulomatous Disease - This study is currently recruiting patients (Current: 23 Nov 2006) - Phagocyte Oxidase Subunit Transduced CD34 Hematopoietic Stem Cells

Drugs used in Clinical Trials for X-linked Genetic Diseases

The format for each of the following is:

• The drug,
• followed by the name of the trial,
• followed by whether the trial is recruiting patients,
  - The trial may not have started and not yet recruiting, or the trial may have started and do not need any more recruitements, or the trial may be completed.
  - Note**: Please follow the link to determine the current trial status.

ClinicalTrials.gov has listed the following drugs in trials for X-linked Genetic Diseases:

• AT1001 (migalastat hydrochloride) - A 12-Week Safety and Pharmacodynamic Study of AT1001 in Female Patients With Fabry Disease - This study is currently recruiting patients (Current: 23 Nov 2006)
• AT1001 (migalastat hydrochloride) - A 12-Week Safety and Pharmacodynamic Study of AT1001 in Patients With Fabry Disease - This study is not yet open for patient recruitment (Current: 23 Nov 2006)
• AT1001 (migalastat hydrochloride) - A 12-Week Safety, Pharmacokinetic, and Pharmacodynamic Study of AT1001 in Patients With Fabry Disease - This study is currently recruiting patients (Current: 23 Nov 2006)
• AT1001 (migalastat hydrochloride) - A 24-Week Safety and Pharmacodynamic Study of AT1001 in Patients With Fabry Disease - This study is currently recruiting patients (Current: 23 Nov 2006)
• AT1001 - AT1001 to Treat Fabry Disease - This study is currently recruiting patients (Current: 23 Nov 2006)
• ATG - Stem Cell Transplant for Inborn Errors of Metabolism - This study is currently recruiting patients (Current: 23 Nov 2006)
• Activated Recombinant FVII (NovoSeven®) - Efficacy of NovoSeven in Bleeding Prophylaxis in Hemophilia - This study is no longer recruiting patients (Current: 23 Nov 2006)
• Agalsidase beta (recombinant form) - A Long Term Safety and Efficacy Study of Fabrazyme Replacement Therapy in Japanese Patients With Fabry Disease. - This study is currently recruiting patients (Current: 23 Nov 2006)
• Agalsidase beta (recombinant form) - A Safety and Efficacy Study of Fabrazyme Replacement Therapy in Patients With Cardiac Fabry Disease - This study is currently recruiting patients (Current: 23 Nov 2006)
• Aminomidazole Carboxamide Riboside - Phase II Pilot Study of Aminoimidazole Carboxamide Riboside (AICAR), a Precursor of Purine Synthesis, for Lesch-Nyhan Disease - This study has been completed (Current: 23 Nov 2006)
• Anti-Thymocyte Globulin - Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies - This study is no longer recruiting patients (Current: 23 Nov 2006)
• BeneFIX - Study Comparing On-Demand Treatment With Two Prophylaxis Regimens of BeneFIX in Patients With Severe Hemophilia B - This study is not yet open for patient recruitment (Current: 23 Nov 2006)
• BeneFIX - Study Evaluating rFIX; BeneFIX in Severe Hemophilia B - This study is currently recruiting patients (Current: 23 Nov 2006)
• Busulfan - Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies - This study is no longer recruiting patients (Current: 23 Nov 2006)
• Busulfan - Stem Cell Transplant for Inborn Errors of Metabolism - This study is currently recruiting patients (Current: 23 Nov 2006)
• CX516 (Ampalex®) - Effects of CX516 on Functioning in Fragile X Syndrome and Autism - This study has been completed (Current: 23 Nov 2006)
• Campath - HSCT for High Risk Inherited Inborn Errors - This study is currently recruiting patients (Current: 23 Nov 2006)
• Chenodeoxycholic Acid - Study of Bile Acids in Patients With Peroxisomal Disorders - This study is not yet open for patient recruitment (Current: 23 Nov 2006)
• Cholic Acid - Study of Bile Acids in Patients With Peroxisomal Disorders - This study is not yet open for patient recruitment (Current: 23 Nov 2006)
• Clofarabine - HSCT for High Risk Inherited Inborn Errors - This study is currently recruiting patients (Current: 23 Nov 2006)
• CoQ10 - CoQ10 and Prednisone in Non-Ambulatory DMD - This study is currently recruiting patients (Current: 23 Nov 2006)
• Coenzyme Q10 - An Open-Label Pilot Study of Coenzyme Q10 in Steroid-Treated Duchenne Muscular Dystrophy - This study is no longer recruiting patients (Current: 23 Nov 2006)
• Copper Histidine - Copper Histidine Therapy for Menkes Diseases - This study is currently recruiting patients (Current: 23 Nov 2006)
• Creatine Monohydrate - A Multicenter Randomized Placebo-Controlled Double-Blind Study to Assess Efficacy and Safety of Glutamine and Creatine Monohydrate in Duchenne Muscular Dystrophy (DMD) - This study has been completed (Current: 23 Nov 2006)
• Creatine Monohydrate - Creatine and Glutamine in Steroid-Naive Duchenne Muscular Dystrophy - This study is no longer recruiting patients (Current: 23 Nov 2006)
• Cyclophosphamide - Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies - This study is no longer recruiting patients (Current: 23 Nov 2006)
• Cyclophosphamide - Stem Cell Transplant for Inborn Errors of Metabolism - This study is currently recruiting patients (Current: 23 Nov 2006)
• Cyclosporine - Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies - This study is no longer recruiting patients (Current: 23 Nov 2006)
• Cyclosporine - Pilot Study of Allogeneic Bone Marrow Transplantation Plus Cyclosporine and Mycophenolate Mofetil to Induce Mixed Hematopoietic Chimerism in Patients With Primary T-Cell Immunodeficiency Disorders - This study is currently recruiting patients (Current: 23 Nov 2006)
• Cyclosporine - Study of Total Body Irradiation and Fludarabine Followed By Allogeneic Peripheral Blood Stem Cell or Bone Marrow Transplantation in Combination With Cyclosporine and Mycophenolate Mofetil in Patients With Inherited Disorders - This study is currently recruiting patients (Current: 23 Nov 2006)
• DRX005B - Alpha-Galactosidase A Replacement Therapy for Fabry Disease - This study has been completed (Current: 23 Nov 2006)
• Dextromethorphan - Independent Studies of Dextromethorphan and of Donepezil Hydrochloride for Rett Syndrome - This study is currently recruiting patients (Current: 23 Nov 2006)
• Dextromethorphan - Study of the Pathogenesis of Rett Syndrome - This study has been completed (Current: 23 Nov 2006)
• Docosahexaenoic Acid - DHA and X-Linked Retinitis Pigmentosa - This study is currently recruiting patients (Current: 23 Nov 2006)
• Donepezil Hydrochloride - Independent Studies of Dextromethorphan and of Donepezil Hydrochloride for Rett Syndrome - This study is currently recruiting patients (Current: 23 Nov 2006)
• Donepezil - An Open-Label Trial of Donepezil in Fragile X Syndrome - This study is currently recruiting patients (Current: 23 Nov 2006)
• Donepezil - Study of the Pathogenesis of Rett Syndrome - This study has been completed (Current: 23 Nov 2006)
• Etoposide - Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies - This study is no longer recruiting patients (Current: 23 Nov 2006)
• Fabrazyme (agalsidase beta) - A Study in Patients With Fabry Disease Who Are on Chronic Hemodialysis Therapy for Treatment of End-Stage Renal Insufficiency. - This study is currently recruiting patients (Current: 23 Nov 2006)
• Fabrazyme (agalsidase beta) - A Study of Fabrazyme in Pediatric Patients With Fabry Disease - This study has been completed (Current: 23 Nov 2006)
• Fabrazyme (agalsidase beta) - A Study of the Effects of Fabrazyme on Mother's Lactation and on the Growth, Development and Immunologic Response of Their Infants. - This study is currently recruiting patients (Current: 23 Nov 2006)
• Fabrazyme (agalsidase beta) - A Study of the Safety and Efficacy of Fabrazyme as Compared to Placebo in Patients With Advanced Fabry Disease - This study has been completed (Current: 23 Nov 2006)
• Fabrazyme (agalsidase beta) - A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease - This study has been completed (Current: 23 Nov 2006)
• Fabrazyme (agalsidase beta) - A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease - This study has been completed (Current: 23 Nov 2006)
• Fabrazyme (agalsidase beta) - A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease - This study is no longer recruiting patients (Current: 23 Nov 2006)
• Fabrazyme (agalsidase beta) - Fabry Disease Registry - This study is currently recruiting patients (Current: 23 Nov 2006)
• Filgrastim - Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes - This study has been completed (Current: 23 Nov 2006)
• Fludarabine - Study of Total Body Irradiation and Fludarabine Followed By Allogeneic Peripheral Blood Stem Cell or Bone Marrow Transplantation in Combination With Cyclosporine and Mycophenolate Mofetil in Patients With Inherited Disorders - This study is currently recruiting patients (Current: 23 Nov 2006)
• Follicle Stimulating Hormone (Fsh) - Follicle Stimulating Hormone (FSH) to Improve Testicular Development in Men With Hypogonadism - This study is currently recruiting patients (Current: 23 Nov 2006)
• Gene Therapy Method for CGD - Gene Therapy for Chronic Granulomatous Diseases - Long-Term Follow-up - This study is no longer recruiting patients (Current: 23 Nov 2006)
• Glutamine - A Multicenter Randomized Placebo-Controlled Double-Blind Study to Assess Efficacy and Safety of Glutamine and Creatine Monohydrate in Duchenne Muscular Dystrophy (DMD) - This study has been completed (Current: 23 Nov 2006)
• Glutamine - Creatine and Glutamine in Steroid-Naive Duchenne Muscular Dystrophy - This study is no longer recruiting patients (Current: 23 Nov 2006)
• Glyceryl Trierucate - Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy - This study has been completed (Current: 23 Nov 2006)
• Glyceryl Trierucate - Study of Glyceryl Trierucate and Glyceryl Trioleate (Lorenzo's Oil) Therapy in Male Children With Adrenoleukodystrophy - This study is currently recruiting patients (Current: 23 Nov 2006)
• Glyceryl Trioleate - Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy - This study has been completed (Current: 23 Nov 2006)
• Glyceryl Trioleate - Study of Glyceryl Trierucate and Glyceryl Trioleate (Lorenzo's Oil) Therapy in Male Children With Adrenoleukodystrophy - This study is currently recruiting patients (Current: 23 Nov 2006)
• GnRH - Letrozole Treatment in Normal and GnRH Deficient Women - This study is currently recruiting patients (Current: 23 Nov 2006)
• Gonadotropin Releasing Hormone (Gnrh) - Follicle Stimulating Hormone (FSH) to Improve Testicular Development in Men With Hypogonadism - This study is currently recruiting patients (Current: 23 Nov 2006)
• Gonadotropin Releasing Hormone (Gnrh) - Investigating the Regulation of Reproductive Hormones in Adult Men - This study is currently recruiting patients (Current: 23 Nov 2006)
• Human Coagulation Factor Ix - Post Marketing Study in Haemophilia B Patients Using Nonafact (Human Coagulation Factor IX) - This study is no longer recruiting patients (Current: 23 Nov 2006)
• Iduronate-2-sulfatase enzyme replacement therapy - Iduronate-2-Sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II) - This study is no longer recruiting patients (Current: 23 Nov 2006)
• Immune Globulin Intravenous (Human) , 10% - Rapid Infusion of Immune Globulin Intravenous (Human) In Primary Immunodeficiency Patients - This study has been completed (Current: 23 Nov 2006)
• Infliximab - Infliximab to Treat Crohn's-Like Inflammatory Bowel Disease in Chronic Granulomatous Disease - This study is currently recruiting patients (Current: 23 Nov 2006)
• Interferon Beta - Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy - This study has been completed (Current: 23 Nov 2006)
• Intravenous immunoglobulin - Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases. - This study is currently recruiting patients (Current: 23 Nov 2006)
• Ketoconazole - Investigating the Regulation of Reproductive Hormones in Adult Men - This study is currently recruiting patients (Current: 23 Nov 2006)
• L-Glutamine - Effect of Oral Glutamine on Muscle Mass and Function in Duchenne Muscular Dystrophy - This study is currently recruiting patients (Current: 23 Nov 2006)
• Letrozole - Letrozole Treatment in Normal and GnRH Deficient Women - This study is currently recruiting patients (Current: 23 Nov 2006)
• MYO-029 - Study Evaluating MYO-029 in Adult Muscular Dystrophy - This study is no longer recruiting patients (Current: 23 Nov 2006)
• Methotrexate - Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies - This study is no longer recruiting patients (Current: 23 Nov 2006)
• Methylprednisolone - Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies - This study is no longer recruiting patients (Current: 23 Nov 2006)
• Monoclonal Factor Ix Replacement Therapy - Phase I/II Study of Monoclonal Factor IX Concentrate for Factor IX Deficiency - This study has been completed (Current: 23 Nov 2006)
• Mycophenolate Mofetil - Pilot Study of Allogeneic Bone Marrow Transplantation Plus Cyclosporine and Mycophenolate Mofetil to Induce Mixed Hematopoietic Chimerism in Patients With Primary T-Cell Immunodeficiency Disorders - This study is currently recruiting patients (Current: 23 Nov 2006)
• Mycophenolate Mofetil - Study of Total Body Irradiation and Fludarabine Followed By Allogeneic Peripheral Blood Stem Cell or Bone Marrow Transplantation in Combination With Cyclosporine and Mycophenolate Mofetil in Patients With Inherited Disorders - This study is currently recruiting patients (Current: 23 Nov 2006)
• NAL-GLU GnRH antagonist - Letrozole Treatment in Normal and GnRH Deficient Women - This study is currently recruiting patients (Current: 23 Nov 2006)
• Oxatomide (tinset) - KUL0401: An Open-Label Pilot Study of Oxatomide in Steroid-Naive Duchenne Muscular Dystrophy - This study is no longer recruiting patients (Current: 23 Nov 2006)
• PTC124 - Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy - This study is currently recruiting patients (Current: 23 Nov 2006)
• Pentoxifylline - Pentoxifylline in Duchenne Muscular Dystrophy - This study is no longer recruiting patients (Current: 23 Nov 2006)
• Phagocyte Oxidase Subunit Transduced CD34 Hematopoietic Stem Cells - Gene Therapy for Chronic Granulomatous Disease - This study is currently recruiting patients (Current: 23 Nov 2006)
• Posaconazole - Posaconazole to Treat Invasive Fungal Infections - This study is currently recruiting patients (Current: 23 Nov 2006)
• Prednisone - Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies - This study is no longer recruiting patients (Current: 23 Nov 2006)
• Prednisone - CoQ10 and Prednisone in Non-Ambulatory DMD - This study is currently recruiting patients (Current: 23 Nov 2006)
• Prednisone - High-Dose Prednisone in Duchenne Muscular Dystrophy - This study is currently recruiting patients (Current: 23 Nov 2006)
• Prednisone - Phase III Randomized, Double-Blind Study of Prednisone for Duchenne Muscular Dystrophy - This study has been completed (Current: 23 Nov 2006)
• Replagal (Agalsidase Alfa) - Replagal Enzyme Replacement Therapy for Adults With Fabry Disease - This study has been completed (Current: 23 Nov 2006)
• Replagal (Galactosidase Alfa) - Replagal Enzyme Replacement Therapy for Children With Fabry Disease - This study is no longer recruiting patients (Current: 23 Nov 2006)
• Replagal agalsidase alfa - An Open-Label Maintenance Study of the Enzyme Replacement Therapy Replagal in Patients With Fabry Disease - This study is no longer recruiting patients (Current: 23 Nov 2006)
• Replagal - Alternative Dosing and Regimen of Replagal to Treat Fabry Disease - This study has been completed (Current: 23 Nov 2006)
• Replagal - An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years With Fabry Disease - This study has been completed (Current: 23 Nov 2006)
• Replagal - An Open-Label Maintenance Study of the Enzyme Replacement Therapy Replagal in Patients With Fabry Disease - This study is no longer recruiting patients (Current: 23 Nov 2006)
• Replagal - Dosing Study of Replagal in Patients With Fabry Disease - This study is no longer recruiting patients (Current: 23 Nov 2006)
• Replagal - Replagal Enzyme Replacement Therapy for Adults With Fabry Disease - This study has been completed (Current: 23 Nov 2006)
• Rfix-R - Study Evaluating of Recombinant Human Factor IX (BeneFIX) and a New Formulation of BeneFIX (rFIX-R) in Moderate to Severe Hemophilia B - This study is no longer recruiting patients (Current: 23 Nov 2006)
• Rfix - Study Evaluating of Recombinant Human Factor IX (BeneFIX) and a New Formulation of BeneFIX (rFIX-R) in Moderate to Severe Hemophilia B - This study is no longer recruiting patients (Current: 23 Nov 2006)
• Rfix - Study Evaluating rFIX; BeneFIX in Hemophilia B - This study is no longer recruiting patients (Current: 23 Nov 2006)
• Risperidone - A Study of the Effectiveness and Safety of Risperidone Versus Placebo in the Treatment of Children With Autistic Disorder and Other Pervasive Developmental Disorders (PDD) - This study is no longer recruiting patients (Current: 23 Nov 2006)
• T-Cell Depleted & CD34+Select/w/StemCell Enriched Product - Treatment of Chronic Granulomatous Disease With Allogeneic Stem Cell Transplantation Versus Standard of Care - This study has been completed (Current: 23 Nov 2006)
• Thalidomide - Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy - This study has been completed (Current: 23 Nov 2006)
• Topiramate - Study of the Pathogenesis of Rett Syndrome - This study has been completed (Current: 23 Nov 2006)
• Ursodiol - Study of Bile Acids in Patients With Peroxisomal Disorders - This study is not yet open for patient recruitment (Current: 23 Nov 2006)

The trial data on this page has been sourced from

• The U.S. National Library of Medicine (NLM) together with ClinicalTrials.gov

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