124 studies found for:    "Renpenning syndrome" OR "Mental Retardation, X-Linked"
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Study Details
Participant Details
Identifiers
Dates
Rank Status Study
1 Enrolling by invitation Genetic Disease Gene Identification
Conditions: Congenital Vertical Talus;   Familial Encephalopathy With Neuroserpin Inclusion Bodies;   Idiopathic Generalised Epilepsy;   Familial Dementia;   X-Linked Mental Retardation
Intervention:
2 Not yet recruiting Triac Trial II in MCT8 Patients
Condition: Allan-Herndon-Dudley Syndrome
Intervention: Drug: Triac
3 Recruiting Triac Trial in MCT8 Patients
Condition: Allan-Herndon-Dudley Syndrome
Intervention: Drug: Triac
4 Completed A Single-Dose Study in Normal Volunteers to Assess the Safety, Tolerability and Pharmacokinetics of STX107
Condition: Fragile X Syndrome
Intervention: Drug: STX107
5 Active, not recruiting Pilot Study of the Effects of the Desipramine on the Neurovegetative Parameters of the Child With Rett Syndrome
Condition: Rett Syndrome
Interventions: Drug: Administration of a high dose of desipramine;   Drug: Administration of a low dose of desipramine;   Drug: Administration of a placebo
6 Suspended Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy
Condition: Adrenoleukodystrophy
Interventions: Drug: glyceryl trierucate;   Drug: glyceryl trioleate
7 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
8 Enrolling by invitation Brain Connectivity in Neurodevelopmental Disorders in Response to Treatment
Conditions: Autism Spectrum Disorders;   Psychosis;   Fragile X Syndrome
Intervention:
9 Completed Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome
Condition: Fragile X Syndrome
Intervention: Drug: AFQ056
10 Enrolling by invitation Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Patients With Hunter Syndrome That Have Completed Study HGT-HIT-094
Condition: Hunter Syndrome
Intervention: Drug: Idursulfase-IT
11 Terminated Long-term, Safety and Tolerability Study of AFQ056 in Adolescent Patients With Fragile X Syndrome (Open-label)
Condition: Fragile X Syndrome
Intervention: Drug: AFQ056
12 Completed Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome)
Condition: Mucopolysaccharidosis II
Intervention: Genetic: lymphocyte gene therapy
13 Completed A Clinical Trial for AMN: Validation of Biomarkers of Oxidative Stress, Efficacy and Safety of a Mixture of Antioxidants
Condition: Adrenomyeloneuropathy
Interventions: Drug: N-acetylcysteine;   Drug: lipoic acid;   Drug: vitamin E
14 Completed The Effect of Bezafibrate on the Level of Very Long Chain Fatty Acids (VLCFA) in X-linked Adrenoleukodystrophy (X-ALD)
Conditions: X-linked Adrenoleukodystrophy;   Adrenomyeloneuropathy
Intervention: Drug: Bezafibrate
15 Recruiting Placebo Controlled Trial of Dextromethorphan in Rett Syndrome
Condition: Rett Syndrome
Interventions: Drug: dextromethorphan;   Drug: placebo
16 Recruiting UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Conditions: Adrenoleukodystrophy;   Batten Disease;   Mucopolysaccharidosis II;   Leukodystrophy, Globoid Cell;   Leukodystrophy, Metachromatic;   Neimann Pick Disease;   Pelizaeus-Merzbacher Disease;   Sandhoff Disease;   Tay-Sachs Disease;   Brain Diseases, Metabolic, Inborn
Intervention: Biological: DUOC-01
17 Completed Study of Cardiac and Paroxysmal Abnormalities in Rett Syndrome
Condition: Rett Syndrome
Intervention:
18 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
19 Terminated An Open Label Extension Study of STX209 in Subjects With Fragile X Syndrome
Condition: Fragile X Syndrome
Intervention: Drug: Arbaclofen
20 Completed Biomarker and DNA Collection in Subjects Participating in Protocol 22003
Condition: Fragile X Syndrome
Intervention: Drug: STX209

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Indicates status has not been verified in more than two years