120 studies found for:    "PPM-X syndrome" OR "Mental Retardation, X-Linked"
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Rank Status Study
21 Completed Iduronate-2-Sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)
Condition: Mucopolysaccharidosis II
Intervention: Drug: Iduronate-2-sulfatase enzyme replacement therapy
22 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
23 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
24 Completed Efficacy and Safety Study of STX209 (Arbaclofen) for the Treatment of Social Withdrawal in Children With Fragile X Syndrome
Condition: Fragile X Syndrome
Interventions: Drug: arbaclofen;   Drug: Placebo
25 Completed Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid
Conditions: Infantile Refsum's Disease;   Zellweger Syndrome;   Adrenoleukodystrophy;   Peroxisomal Disorders;   Cholestasis
Intervention: Drug: Cholic Acids
26 Unknown  The Psychiatric and Cognitive Phenotypes in Velocardiofacial Syndrome
Conditions: Velocardiofacial Syndrome;   Williams Syndrome;   Fragile X Syndrome
Intervention: Drug: methylphenidate, fluoxetin, risperidone
27 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
28 Completed A Study of RO4917523 in Patients With Fragile X Syndrome
Condition: Fragile X Syndrome
Interventions: Drug: Placebo;   Drug: RO4917523
29 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
30 Completed
Has Results
Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome
Condition: Hunter Syndrome
Intervention: Other: No treatment
31 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
32 Unknown  Independent Studies of Dextromethorphan and of Donepezil Hydrochloride for Rett Syndrome
Condition: Rett Syndrome
Interventions: Drug: dextromethorphan;   Drug: donepezil hydrochloride
33 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
34 Completed Riluzole in Fragile X Syndrome
Condition: Fragile X Syndrome
Intervention: Drug: Riluzole
35 Recruiting Ganaxolone Treatment in Children With Fragile X Syndrome
Condition: Fragile x Syndrome
Interventions: Drug: Ganaxolone;   Drug: Placebo
36 Completed Exercise Study of Function and Pathology for Women With X-linked Adrenoleukodystrophy
Condition: X-linked Adrenoleukodystrophy
Intervention: Behavioral: exercise training
37 Withdrawn Trial of Kuvan in Lesch-Nyhan Disease
Condition: Behavioral Manifestations of Lesch-Nyhan Disease
Intervention: Drug: sapropterin
38 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
39 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
40 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

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Indicates status has not been verified in more than two years