126 studies found for:    "PPM-X syndrome" OR "Mental Retardation, X-Linked"
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Rank Status Study
21 Completed Biomarker Testing and DNA Collection in Subjects Participating in Protocol 22001
Condition: Fragile X Syndrome
Intervention: Drug: STX209
22 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
23 Recruiting Genetic and Physical Characteristics of Rett Syndrome
Condition: Rett Syndrome, MECP2 Duplication dIsorder, and RTT-related Conditions
Intervention:
24 Completed A Study of RO4917523 in Patients With Fragile X Syndrome
Condition: Fragile X Syndrome
Interventions: Drug: Placebo;   Drug: RO4917523
25 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
26 Completed
Has Results
Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase
Conditions: Hunter Syndrome;   Mucopolysaccharidosis II (MPS II)
Intervention: Biological: Idursulfase
27 Completed
Has Results
A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®
Condition: Hunter Syndrome
Interventions: Other: Control;   Drug: Idursulfase IT (1 mg);   Drug: Idursulfase IT (10 mg);   Drug: Idursulfase IT (30 mg)
28 Recruiting Human Placental-Derived Stem Cell Transplantation
Conditions: Mucopolysaccharidosis I;   Mucopolysaccharidosis VI;   Adrenoleukodystrophy;   Niemann-Pick Disease;   Metachromatic Leukodystrophy;   Wolman Disease;   Krabbe's Disease;   Gaucher's Disease;   Fucosidosis;   Batten Disease;   Severe Aplastic Anemia;   Diamond-Blackfan Anemia;   Amegakaryocytic Thrombocytopenia;   Myelodysplastic Syndrome;   Acute Myelogenous Leukemia;   Acute Lymphocytic Leukemia
Intervention: Drug: Human Placental Derived Stem Cell
29 Recruiting Study of Acamprosate in Fragile x Syndrome
Condition: Fragile X Syndrome
Interventions: Drug: acamprosate;   Drug: Placebo
30 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
31 Completed Efficacy, Safety and Tolerability of AFQ056 in Fragile X Patients
Condition: Fragile X Syndrome
Interventions: Drug: AF056;   Drug: Placebo
32 Completed
Has Results
Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)
Condition: Mucopolysaccharidosis II
Interventions: Biological: Iduronate-2-sulfatase enzyme replacement therapy;   Biological: iduronate-2-sulfatase enzyme replacement therapy;   Biological: Placebo
33 Active, not recruiting Extension of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase (IT)in Conjunction With Elaprase in Pediatric Patients With Hunter Syndrome and Cognitive Impairment
Condition: Hunter Syndrome
Intervention: Drug: Idursulfase-IT
34 Active, not recruiting Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
Conditions: Hurler Syndrome (MPS I);   Hurler-Scheie Syndrome With Early Neurologic Involvement and/or Sensitization to Enzyme Replacement Therapy (ERT);   Hunter Syndrome (MPS II);   Sanfilippo Syndrome (MPS III);   Krabbe Disease (Globoid Leukodystrophy);   Metachromatic Leukodystrophy (MLD);   Adrenoleukodystrophy (ALD and AMN);   Sandhoff Disease;   Tay Sachs Disease;   Pelizaeus Merzbacher (PMD);   Niemann-Pick Disease;   Alpha-mannosidosis
Intervention: Biological: Enriched Hematopoetic Stem Cell Transplantation/novel platform technology
35 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)
36 Unknown  Memantine Treatment in Fragile X-Associated Tremor/Ataxia Syndrome
Conditions: Fragile X-Associated Tremor/Ataxia Syndrome;   Fragile X Premutation Carriers
Intervention: Drug: Memantine
37 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
38 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:
39 Recruiting An Open Label, Exploratory Study to Investigate the Treatment Effect of Glatiramer Acetate on Girls Woth Rett Syndrome
Condition: Rett Syndrome
Intervention: Drug: Glatiramer Acetate  (Copaxone®)
40 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

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Indicates status has not been verified in more than two years