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Results for “X-Linked Intellectual Disability”

381 trials

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Showing 20 of 381 results

Not applicableNo Longer AvailableNCT02233257
What this trial is testing

Expanded Access for Lorenzo's Oil (GTO/GTE) in Adrenoleukodystrophy

Who this might be right for
X-linked Adrenoleukodystrophy
University of Minnesota
Large-scale testing (Phase 3)WithdrawnNCT00935753
What this trial is testing

Trial of Kuvan in Lesch-Nyhan Disease

Who this might be right for
Behavioral Manifestations of Lesch-Nyhan Disease
University of California, San Diego
Testing effectiveness (Phase 2)Study completedNCT02952534
What this trial is testing

Rucaparib in Patients With Metastatic Castration-resistant Prostate Cancer and Homologous Recombination Gene Deficiency

Who this might be right for
Metastatic Castration Resistant Prostate Cancer
pharmaand GmbH 277
Not applicableLooking for participantsNCT06147414
What this trial is testing

Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders

Who this might be right for
Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, IncludingSickle Cell DiseaseCystic Fibrosis+12 more
Assistance Publique - Hôpitaux de Paris 550
Large-scale testing (Phase 3)Active Not RecruitingNCT02616484
What this trial is testing

Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency:

Who this might be right for
Pyruvate Dehydrogenase Complex Deficiency
Saol Therapeutics Inc 34
Not applicableStudy completedNCT03161171
What this trial is testing

Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-III

Who this might be right for
Mucopolysaccharidosis Type IMucopolysaccharidosis Type IIMucopolysaccharidosis Type III+2 more
University Hospital Heidelberg 34
Large-scale testing (Phase 3)Active Not RecruitingNCT04348136
What this trial is testing

An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II

Who this might be right for
Mucopolysaccharidosis II
JCR Pharmaceuticals Co., Ltd. 27
Not applicableLooking for participantsNCT04880356
What this trial is testing

Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.

Who this might be right for
Inherited DiseaseRare DiseasesMetabolic Disease+3 more
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta 100
Not applicableStudy completedNCT02738281
What this trial is testing

Natural History of Rett Syndrome & Related Disorders

Who this might be right for
Rett SyndromeMECP2 Duplication dIsorderCDKL5 Disorder+1 more
University of Alabama at Birmingham 1,044
Post-approval studies (Phase 4)Study completedNCT02455622
What this trial is testing

Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age

Who this might be right for
Hunter Syndrome
Shire 21
Not applicableStudy completedNCT00299312
What this trial is testing

Genetic and Physical Characteristics of Rett Syndrome

Who this might be right for
Rett SyndromeMECP2 Duplication DisorderRett-related Disorder
University of Alabama at Birmingham 10
Large-scale testing (Phase 3)Study completedNCT00007020
What this trial is testing

Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid

Who this might be right for
Infantile Refsum's DiseaseZellweger SyndromeAdrenoleukodystrophy+2 more
Mirum Pharmaceuticals, Inc. 85
Testing effectiveness (Phase 2)Study completedNCT01520363
What this trial is testing

Placebo Controlled Trial of Dextromethorphan in Rett Syndrome

Who this might be right for
Rett Syndrome
Hugo W. Moser Research Institute at Kennedy Krieger, Inc. 57
Testing effectiveness (Phase 2)WithdrawnNCT04823052
What this trial is testing

Investigation of Sulindac (HLX-0201) and Gaboxadol (HLX-0206) in Male Fragile X Syndrome Patients Aged 13-40

Who this might be right for
Fragile X Syndrome
Healx Limited
Large-scale testing (Phase 3)Ended earlyNCT04988867
What this trial is testing

An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome

Who this might be right for
Rett Syndrome
ACADIA Pharmaceuticals Inc. 15
Not applicableLooking for participantsNCT06338267
What this trial is testing

Validating Innovative Biosensors for Rett Autonomic Symptom Tracking

Who this might be right for
Rett Syndrome
Rett Syndrome Research Trust 30
Large-scale testing (Phase 3)Enrolling By InvitationNCT05594992
What this trial is testing

An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) Subjects

Who this might be right for
Mucopolysaccharidosis II
JCR Pharmaceuticals Co., Ltd. 80
Large-scale testing (Phase 3)Study completedNCT01282268
What this trial is testing

Efficacy and Safety Study of STX209 (Arbaclofen) for Social Withdrawal in Adolescents and Adults With Fragile X Syndrome

Who this might be right for
Fragile X Syndrome
Seaside Therapeutics, Inc. 125
Not applicableStudy completedNCT01449240
What this trial is testing

Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome

Who this might be right for
Hunter Syndrome
Shire 10
Not applicableLooking for participantsNCT05939232
What this trial is testing

Registry of X-linked Adrenoleukodystrophy

Who this might be right for
X-linked Adrenoleukodystrophy
Beijing Tiantan Hospital 200
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