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:    "PPM-X syndrome" OR "Mental Retardation, X-Linked"
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Rank Status Study
21 Completed Acamprosate in Youth With Fragile X Syndrome
Conditions: Fragile X Syndrome;   Autism Spectrum Disorders
Intervention: Drug: Acamprosate
22 Completed A Study of RO4917523 in Patients With Fragile X Syndrome
Condition: Fragile X Syndrome
Interventions: Drug: Placebo;   Drug: RO4917523
23 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
24 Completed Phase 2 Study of EPI-743 for Treatment of Rett Syndrome
Condition: Rett Syndrome
Intervention: Drug: EPI-743
25 Recruiting A 6-week, Study of MG01CI Low Dose and High Dose Compared With Placebo in Adults and Adolescents With Fragile X Syndrome
Condition: Fragile X Syndrome
Intervention: Drug: MG01CI extended-release tablet
26 Completed Efficacy and Safety Study of STX209 (Arbaclofen) for Social Withdrawal in Adolescents and Adults With Fragile X Syndrome
Condition: Fragile X Syndrome
Interventions: Drug: arbaclofen;   Drug: placebo
27 Completed Biomarker Testing and DNA Collection in Subjects Participating in Protocol 22001
Condition: Fragile X Syndrome
Intervention: Drug: STX209
28 Unknown  Clinical Study and Gene Mutation Analysis of Adrenoleukodystrophy in Taiwanese Children
Condition: Adrenoleukodystrophy
Intervention: Procedure: blood sampling and medical records
29 Recruiting Molecular Bases of Response to Copper Treatment in Menkes Disease, Related Phenotypes, and Unexplained Copper Deficiency
Conditions: Menkes Disease;   Occipital Horn Syndrome;   Unexplained Copper Deficiency
Intervention: Drug: Copper Histidine
30 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
31 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
32 Completed Study of Cardiac and Paroxysmal Abnormalities in Rett Syndrome
Condition: Rett Syndrome
Intervention:
33 Available Expanded Access for Lorenzo's Oil (GTO/GTE) in Adrenoleukodystrophy
Condition: X-linked Adrenoleukodystrophy
Intervention: Drug: Lorenzo's Oil
34 Completed A Study of RO4917523 in Pediatric Patients With Fragile X Syndrome
Condition: Fragile X Syndrome
Interventions: Drug: Placebo;   Drug: RO4917523
35 Terminated
Has Results
Trial of Dextromethorphan in Rett Syndrome
Condition: Rett Syndrome
Intervention: Drug: Dextromethorphan
36 Recruiting A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Conditions: Hematologic Malignancies;   Certain:;   Inherited Disorders of Metabolism;   Inherited Abnormalities of Erythrocyte Differentiation or Function;   Inherited Abnormalities of Platelets;   Disorders of the Immune System;   Histiocytic Disorders;   X-linked Adrenoleukodystrophy;   Primary Immunodeficiency Diseases;   Bone Marrow Failure;   Beta-thalassemia
Intervention: Drug: A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs)
37 Completed
Has Results
Double-blind Placebo Controlled Study of Oxytocin in Fragile X Syndrome
Condition: Fragile X Syndrome
Interventions: Drug: placebo;   Drug: oxytocin 24IU;   Drug: oxytocin 48IU
38 Recruiting Ganaxolone Treatment in Children With Fragile X Syndrome
Condition: Fragile x Syndrome
Interventions: Drug: Ganaxolone;   Drug: Placebo
39 Completed Riluzole in Fragile X Syndrome
Condition: Fragile X Syndrome
Intervention: Drug: Riluzole
40 Not yet recruiting Analysis of the Glutathione Cycle in Children With Rett Syndrome
Condition: Rett Syndrome
Intervention:

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Indicates status has not been verified in more than two years