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
120 studies found for:    "MECP2 duplication syndrome" OR "Mental Retardation, X-Linked"
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Rank Status Study
21 Recruiting EXPLAIN -FragilE X Registry: An exPlorative Longitudinal Study for chAracterIzation, Treatment Pathways and patieNt-related Outcomes
Condition: Fragile-X Syndrome
Intervention:
22 Recruiting Study to Assess Safety and Efficacy of Fingolimod in Children With Rett Syndrome
Condition: Rett's Syndrome
Intervention: Drug: fingolimod (FTY720)
23 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
24 Completed Study of Cardiac and Paroxysmal Abnormalities in Rett Syndrome
Condition: Rett Syndrome
Intervention:
25 Completed Aripiprazole in Fragile X Syndrome
Conditions: Fragile X Syndrome;   Behavioral Problems Associated With Fragile X Syndrome
Intervention: Drug: Aripiprazole
26 Completed A Study to Test the Possibility of Cross Reaction Induced by the Idursulfase Drug to GSK2788723
Condition: Mucopolysaccharidosis II
Interventions: Drug: Idursulfase;   Drug: GSK2788723
27 Recruiting Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment
Condition: Hunter Syndrome
Interventions: Biological: idursulfase-IT;   Other: No IT treatment
28 Suspended Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy
Condition: Adrenoleukodystrophy
Interventions: Drug: glyceryl trierucate;   Drug: glyceryl trioleate
29 Enrolling by invitation Brain Connectivity in Neurodevelopmental Disorders in Response to Treatment
Conditions: Autism Spectrum Disorders;   Psychosis;   Fragile X Syndrome
Intervention:
30 Unknown  Safety and Efficacy Study of Antioxidants for the Treatment of the Fragile X Syndrome
Condition: Fragile X Syndrome
Interventions: Dietary Supplement: Ascorbic Acid (Vitamin C) and Alpha-tocopherol (Vitamin E);   Other: Placebo
31 Unknown  Add-on Pilot Trial of Minocycline to Treat Fragile X Syndrome
Condition: Fragile X Syndrome
Intervention: Drug: Minocycline
32 Unknown  Validation of Saliva in the Esoterix Genetic Laboratories Fragile X Assay
Condition: Fragile X Syndrome
Intervention:
33 Completed Safety and Efficacy of Hunterase
Condition: Hunter Syndrome
Intervention: Biological: Hunterase
34 Completed Exercise Study of Function and Pathology for Women With X-linked Adrenoleukodystrophy
Condition: X-linked Adrenoleukodystrophy
Intervention: Behavioral: exercise training
35 Withdrawn Trial of Kuvan in Lesch-Nyhan Disease
Condition: Behavioral Manifestations of Lesch-Nyhan Disease
Intervention: Drug: sapropterin
36 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
37 Terminated An Open Label Extension Study of STX209 in Subjects With Fragile X Syndrome
Condition: Fragile X Syndrome
Intervention: Drug: Arbaclofen
38 Completed Biomarker and DNA Collection in Subjects Participating in Protocol 22003
Condition: Fragile X Syndrome
Intervention: Drug: STX209
39 Completed To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients
Condition: Mucopolysaccharidosis II
Interventions: Drug: GC1111_0.5mg/kg;   Drug: GC1111_1.0mg/kg;   Drug: Elaprase_0.5mg/kg
40 Completed A Study of RO4917523 in Patients With Fragile X Syndrome
Condition: Fragile X Syndrome
Interventions: Drug: Placebo;   Drug: RO4917523

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Indicates status has not been verified in more than two years