16 studies found for:    MENTAL RETARDATION, X-LINKED 1
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Rank Status Study
1 Terminated The Natural History of Reproductive and Overall Health in Girls and Women With a Pre-Mutation in the FMR1 Gene; Creation of a Patient Registry
Conditions: Fragile X Syndrome;   FMR1 Premutation;   Primary Ovairan Insufficiency;   Premature Ovarian Failure;   Premature Menopause
Intervention:
2 Completed HSCT for High Risk Inherited Inborn Errors
Conditions: Adrenoleukodystrophy;   Metachromatic Leukodystrophy;   Globoid Cell Leukodystrophy;   Tay Sachs Disease;   Sandhoffs Disease;   Wolman Disease;   I-Cell Disease;   Sanfilippo Syndrome;   GM1 Gangliosidosis
Interventions: Drug: Clofarabine;   Procedure: Total body Irradiation;   Drug: Melphalan;   Biological: Hematopoietic Stem Cell Transplantation;   Drug: Alemtuzumab;   Drug: mycophenylate mofetil;   Device: Cyclosporine A;   Drug: Hydroxyurea
3 Completed
Has Results
Stem Cell Transplant for Inborn Errors of Metabolism
Conditions: Adrenoleukodystrophy;   Metachromatic Leukodystrophy;   Globoid Cell Leukodystrophy;   Gaucher's Disease;   Fucosidosis;   Wolman Disease;   Niemann-Pick Disease;   Batten Disease;   GM1 Gangliosidosis;   Tay Sachs Disease;   Sandhoff Disease
Interventions: Procedure: Stem Cell Transplant;   Drug: Busulfan, Cyclophosphamide, Antithymocyte Globulin
4 Completed Treatment of Rett Syndrome With rhIGF-1 (Mecasermin [rDNA]Injection)
Condition: Rett Syndrome
Intervention: Drug: rhIGF-1
5 Recruiting Biobanking of Rett Syndrome and Related Disorders
Conditions: Rett Syndrome;   MECP2 Duplication;   CDKL5;   FOXG1 Disorders
Intervention:
6 Recruiting Treatment of Rett Syndrome With Recombinant Human IGF-1
Condition: Rett Syndrome
Interventions: Drug: Recombinant Human Insulin Growth Factor 1 (rhIGF-1);   Drug: Placebo
7 Recruiting Genetics of Pediatric-Onset Motor Neuron and Neuromuscular Diseases
Conditions: Spinal Muscular Atrophy;   Charcot-Marie-Tooth Disease;   Muscular Dystrophy;   Spinal Muscular Atrophy With Respiratory Distress 1;   Amyotrophic Lateral Sclerosis;   Motor Neuron Disease;   Neuromuscular Disease;   Peroneal Muscular Atrophy;   Fragile X Syndrome
Intervention: Other: sample collection
8 Completed
Has Results
Safety and Tolerability of the D1 Dopamine Receptor Antagonist Ecopipam in Patients With Lesch-Nyhan Disease
Condition: Lesch-Nyhan Disease
Intervention: Drug: Ecopipam
9 Recruiting Study to Assess Safety and Efficacy of Fingolimod in Children With Rett Syndrome
Condition: Rett's Syndrome
Intervention: Drug: fingolimod (FTY720)
10 Recruiting Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter Syndrome
Condition: Mucopolysaccharidosis II
Intervention: Biological: AGT-182
11 Recruiting A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy
Condition: X-linked Adrenoleukodystrophy
Intervention: Dietary Supplement: vitamin D3
12 Completed 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);   Dietary Supplement: Placebo
13 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
14 Completed Clinical Study to Assess the Pharmacokinetics, Safety and Tolerability of Single and Multiple Oral Doses of AFQ056 in Children With Fragile X Syndrome (FXS)
Condition: Fragile X Syndrome
Intervention: Drug: AFQ056
15 Recruiting Combined Treatment of Minocycline and Lovastatin to Treat Individuals With Fragile X Syndrome
Condition: Fragile X Syndrome
Interventions: Drug: Minocycline, then Minocycline/Lovastatin;   Drug: Lovastatin, then Minocycline/Lovastatin
16 Completed Genetic and Physical Characteristics of Rett Syndrome
Conditions: Rett Syndrome;   MECP2 Duplication Disorder;   Rett-related Disorder
Intervention:

Indicates status has not been verified in more than two years