157 studies found for:    MENTAL RETARDATION, X-LINKED, WITH OR WITHOUT SEIZURES, ARX-RELATED
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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 Recruiting Combining Lovastatin and a Parent-Implemented Language Intervention for Fragile X Syndrome
Conditions: Fragile X Syndrome;   Genetic Diseases
Interventions: Drug: Lovastatin;   Drug: Placebo
3 Active, not recruiting Triac Trial in MCT8 Patients
Condition: Allan-Herndon-Dudley Syndrome
Intervention: Drug: Triac
4 Not yet recruiting Triac Trial II in MCT8 Patients
Condition: Allan-Herndon-Dudley Syndrome
Intervention: Drug: Triac
5 Recruiting A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy
Condition: X-linked Adrenoleukodystrophy
Intervention: Dietary Supplement: vitamin D3
6 Withdrawn Safety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD)
Conditions: X-Linked Adrenoleukodystrophy;   Adrenomyeloneuropathy
Intervention: Drug: Sobetirome
7 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
8 Completed Exercise Study of Function and Pathology for Women With X-linked Adrenoleukodystrophy
Condition: X-linked Adrenoleukodystrophy
Intervention: Behavioral: exercise training
9 Enrolling by invitation Long-term Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
Conditions: Cerebral Adrenoleukodystrophy (CALD);   Adrenoleukodystrophy (ALD);   X-Linked Adrenoleukodystrophy (X-ALD)
Intervention: Other: Safety and efficacy assessments
10 Recruiting Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)
Conditions: X-Linked Adrenoleukodystrophy (X-ALD);   Cerebral Adrenoleukodystrophy (CALD);   Adrenoleukodystrophy (ALD)
Intervention: Genetic: Allo-HSCT
11 Active, not recruiting A Phase 2/3 Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Childhood Cerebral Adrenoleukodystrophy (CCALD)
Conditions: Childhood Cerebral Adrenoleukodystrophy;   (X-linked Adrenoleukodystrophy Cerebral Childhood)
Interventions: Genetic: Lenti-D Drug Product;   Drug: Busulfan;   Drug: Cyclophosphamide;   Drug: Filgrastim
12 Recruiting Effects of Social Gaze Training on Brain and Behavior in Fragile X Syndrome
Conditions: Fragile X Syndrome;   Intellectual Disability
Intervention: Behavioral: Social gaze training
13 Available Expanded Access for Lorenzo's Oil (GTO/GTE) in Adrenoleukodystrophy
Condition: X-linked Adrenoleukodystrophy
Intervention: Drug: Lorenzo's Oil
14 Terminated
Has Results
Long-term, Safety and Tolerability Study of AFQ056 in Adolescent Patients With Fragile X Syndrome (Open-label)
Condition: Fragile X Syndrome
Intervention: Drug: AFQ056
15 Completed
Has Results
Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome
Condition: Fragile X Syndrome
Interventions: Drug: AFQ056;   Drug: Placebo
16 Terminated
Has Results
Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome
Condition: Fragile X Syndrome
Intervention: Drug: AFQ056
17 Completed Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome
Condition: Fragile X Syndrome
Interventions: Drug: AFQ056;   Drug: Placebo
18 Completed
Has Results
Copper Histidine Therapy for Menkes Diseases
Condition: Kinky Hair Syndrome
Intervention: Drug: Copper Histidine
19 Recruiting Pediatric Patients With Metabolic or Other Genetic Disorders
Conditions: Genetic Disorder;   Asperger Disorder;   Autism Spectrum Disorder;   Fragile X Syndrome;   Developmental Delay
Intervention:
20 Not yet recruiting Glutaminergic Transmission in Autism : Molecular Imaging Exploration
Conditions: Autism;   Fragile-X Syndrome (FXS);   Healthy Volunteers
Interventions: Drug: [18F]FPEB PET imaging;   Other: Biological samples;   Other: MRI

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Indicates status has not been verified in more than two years