Comment Period Extended to 3/23/2015 for Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials
123 studies found for:    "Renpenning syndrome" OR "Mental Retardation, X-Linked"
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Rank Status Study
1 Enrolling by invitation Genetic Disease Gene Identification
Conditions: Congenital Vertical Talus;   Familial Encephalopathy With Neuroserpin Inclusion Bodies;   Idiopathic Generalised Epilepsy;   Familial Dementia;   X-Linked Mental Retardation
Intervention:
2 Recruiting Triac Trial in MCT8 Patients
Condition: Allan-Herndon-Dudley Syndrome
Intervention: Drug: Triac
3 Completed
Has Results
Safety, Tolerability and Efficacy Study of STX209 in Subjects With Fragile X Syndrome
Condition: Fragile X Syndrome
Interventions: Drug: STX209;   Drug: Placebo
4 Completed Study of Cardiac and Paroxysmal Abnormalities in Rett Syndrome
Condition: Rett Syndrome
Intervention:
5 Completed Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome
Condition: Fragile X Syndrome
Interventions: Drug: AFQ056;   Drug: Placebo
6 Completed Study of the Pathogenesis of Rett Syndrome
Condition: Rett Syndrome
Interventions: Drug: dextromethorphan;   Drug: topiramate;   Drug: Donepezil
7 Completed
Has Results
Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome
Condition: Hunter Syndrome
Intervention: Other: No treatment
8 Recruiting Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
Conditions: Mucopolysaccharidosis;   Hurler Syndrome;   Hunter Syndrome;   Maroteaux-Lamy Syndrome;   Sly Syndrome;   Alpha Mannosidosis;   Fucosidosis;   Aspartylglucosaminuria;   Adrenoleukodystrophy (ALD);   Krabbe Disease;   Metachromatic Leukodystrophy (MLD);   Sphingolipidoses;   Peroxisomal Disorders
Interventions: Drug: Campath-1H;   Drug: Cyclophosphamide;   Drug: Busulfan;   Procedure: Allogeneic stem cell transplantation;   Drug: Cyclosporine A;   Drug: Mycophenolate Mofetil
9 Suspended A Study to Assess the Tolerability of a Single Dose of STX107 in Adults With Fragile X Syndrome
Condition: Fragile X Syndrome
Intervention: Drug: STX107
10 Terminated
Has Results
Clinical Trial of Growth Hormone in MPS I, II, and VI
Conditions: Mucopolysaccharidosis I;   Mucopolysaccharidosis II;   Mucopolysaccharidosis VI
Intervention: Drug: Somatropin (DNA origin)
11 Completed Predictors of Caregiver Adaptation to Pervasive Developmental Disorders
Conditions: Autism;   Asperger's Syndrome;   Rett's Disorder
Intervention:
12 Unknown  Independent Studies of Dextromethorphan and of Donepezil Hydrochloride for Rett Syndrome
Condition: Rett Syndrome
Interventions: Drug: dextromethorphan;   Drug: donepezil hydrochloride
13 Completed Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy
Condition: Adrenoleukodystrophy
Interventions: Drug: glyceryl trierucate;   Drug: glyceryl trioleate;   Drug: interferon beta;   Drug: thalidomide
14 Unknown  Validation of Saliva in the Esoterix Genetic Laboratories Fragile X Assay
Condition: Fragile X Syndrome
Intervention:
15 Completed Safety and Efficacy of Hunterase
Condition: Hunter Syndrome
Intervention: Biological: Hunterase
16 Unknown  Add-on Pilot Trial of Minocycline to Treat Fragile X Syndrome
Condition: Fragile X Syndrome
Intervention: Drug: Minocycline
17 Completed Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of Central Nervous System (CNS) Involvement and Who Are Currently Receiving Treatment With Elaprase®
Condition: Hunter Syndrome
Interventions: Behavioral: Neurobehavioral testing;   Other: Visual and auditory assessments
18 Completed Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome)
Condition: Mucopolysaccharidosis II
Intervention: Genetic: lymphocyte gene therapy
19 Terminated Long-term, Safety and Tolerability Study of AFQ056 in Adolescent Patients With Fragile X Syndrome (Open-label)
Condition: Fragile X Syndrome
Intervention: Drug: AFQ056
20 Completed
Has Results
Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy
Conditions: Hunter Syndrome;   Mucopolysaccharidosis II;   MPS II
Intervention: Biological: Idursulfase

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Indicates status has not been verified in more than two years