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Results for “Intellectual Disabilities Nutritional”

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Showing 20 of 0 results

Testing effectiveness (Phase 2)Study completedNCT01506141
What this trial is testing

An Extension Study of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase-IT in Conjunction With Elaprase in Pediatric Participants With Hunter Syndrome and Cognitive Impairment

Who this might be right for
Hunter Syndrome
Takeda 15
Not applicableStudy completedNCT01724905
What this trial is testing

Weight Loss and Maintenance for Individuals With Intellectual Developmental Disabilities (IDD)

Who this might be right for
Weight Loss
Joseph Donnelly 151
Large-scale testing (Phase 3)Ended earlyNCT00748969
What this trial is testing

Clinical Trial of Growth Hormone in MPS I, II, and VI

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis VI
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center 2
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 applicableLooking for participantsNCT01793168
What this trial is testing

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

Who this might be right for
Rare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence+340 more
Sanford Health 20,000
Large-scale testing (Phase 3)Active Not RecruitingNCT03566043
What this trial is testing

CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)

Who this might be right for
Mucopolysaccharidosis Type II (MPS II)
REGENXBIO Inc. 48
Not applicableLooking for participantsNCT05443906
What this trial is testing

Home Exercise for Individuals With Neurodegenerative Disease

Who this might be right for
Neurodegenerative DiseasesLeukodystrophyAtaxia+5 more
Hugo W. Moser Research Institute at Kennedy Krieger, Inc. 30
Testing effectiveness (Phase 2)Study completedNCT02629991
What this trial is testing

Oxytocin vs. Placebo for the Treatment Hyperphagia in Children and Adolescents With Prader-Willi Syndrome

Who this might be right for
Prader-Willi SyndromeHyperphagia
Montefiore Medical Center 23
Not applicableStudy completedNCT00372125
What this trial is testing

Nordic Study on the Effects of Growth Hormone (Norditropin SimpleXx) Treatment in Adults With Prader-Willi Syndrome

Who this might be right for
Prader-Willi Syndrome
Karolinska University Hospital 46
Testing effectiveness (Phase 2)Ended earlyNCT05394064
What this trial is testing

Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN)

Who this might be right for
AMNAMN Gene MutationX-ALD
SwanBio Therapeutics, Inc. 9
Large-scale testing (Phase 3)Study completedNCT02055118
What this trial is testing

Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment

Who this might be right for
Hunter Syndrome
Shire 58
Testing effectiveness (Phase 2)WithdrawnNCT03196765
What this trial is testing

Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy

Who this might be right for
X-Linked Adrenoleukodystrophy
NeuroVia, Inc.
Not applicableStudy completedNCT04007536
What this trial is testing

Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome

Who this might be right for
Mucopolysaccharidosis II
Denali Therapeutics Inc. 18
Early research (Phase 1)WithdrawnNCT05238324
What this trial is testing

Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II

Who this might be right for
Mucopolysaccharidosis II
Homology Medicines, Inc
Not applicableEnded earlyNCT05008874
What this trial is testing

Study of Disease Progression in Adults With Inherited Forms of Spastic Paraplegia

Who this might be right for
AMNAMN Gene MutationX-ALD
SwanBio Therapeutics, Inc. 65
Testing effectiveness (Phase 2)Study completedNCT05443685
What this trial is testing

ADX-629 Therapy for Sjogren-Larsson Syndrome

Who this might be right for
Sjogren-Larsson Syndrome
University of Nebraska 8
Early research (Phase 1)Study completedNCT05504395
What this trial is testing

A Pharmacokinetic and Safety Study of CSTI-500 in Subjects With Prader-Willi Syndrome

Who this might be right for
Prader-Willi Syndrome
ConSynance Therapeutics 10
Not applicableStudy completedNCT00705172
What this trial is testing

Retrospective Observational Study on Efficacy and Safety of Norditropin® in Children With Prader-Willi Syndrome

Who this might be right for
Genetic DisorderPrader-Willi Syndrome
Novo Nordisk A/S 41
Testing effectiveness (Phase 2)Ended earlyNCT03848481
What this trial is testing

CBDV vs Placebo in Children and Adults up to Age 30 With Prader-Willi Syndrome (PWS)

Who this might be right for
Prader-Willi Syndrome
Eric Hollander 6
Early research (Phase 1)WithdrawnNCT01787578
What this trial is testing

Safety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD)

Who this might be right for
X-Linked AdrenoleukodystrophyAdrenomyeloneuropathy
Thomas S. Scanlan
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