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Results for “Brain Diseases, Metabolic, Inborn”

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Showing 20 of 0 results

Early research (Phase 1)Looking for participantsNCT06150716
What this trial is testing

Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)

Who this might be right for
Pelizaeus-Merzbacher Disease
Ionis Pharmaceuticals, Inc. 24
Not applicableStudy completedNCT00030134
What this trial is testing

Data Collection in Women With Fabry Disease

Who this might be right for
Fabry Disease
National Institute of Neurological Disorders and Stroke (NINDS) 60
Not applicableStudy completedNCT02742298
What this trial is testing

Pompe Disease QMUS and EIM

Who this might be right for
Pompe Disease
Duke University 25
Large-scale testing (Phase 3)Study completedNCT03425539
What this trial is testing

Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease

Who this might be right for
Fabry Disease
Idorsia Pharmaceuticals Ltd. 118
Not applicableLooking for participantsNCT05444127
What this trial is testing

Oral Health and Wilson's Disease: SOMAWI

Who this might be right for
Wilson Disease
Fondation Ophtalmologique Adolphe de Rothschild 150
Early research (Phase 1)Not Yet RecruitingNCT07479953
What this trial is testing

Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis Clinical Trial

Who this might be right for
GM1 GangliosidosesGM1 Gangliosidosis, Type IGM1 Gangliosidosis, Type 2
Tippi Mackenzie 5
Testing effectiveness (Phase 2)Ended earlyNCT01237041
What this trial is testing

Free Fatty Acids, Body Weight, and Growth Hormones Secretion in Children

Who this might be right for
ObesityShort StatureGrowth Hormone Deficiency
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 37
Not applicableEnrolling By InvitationNCT07159581
What this trial is testing

Gene Therapy for Wilson Disease Evaluated by 64Cu PET/CT

Who this might be right for
Wilson Disease
Thomas Damgaard Sandahl 5
Not applicableLooking for participantsNCT06560736
What this trial is testing

Development of Novel Psychological Assessment Tools and Anxiety Intervention for Phenylketonuria

Who this might be right for
Phenylketonurias
University of Missouri-Columbia 30
Not applicableStudy completedNCT02179333
What this trial is testing

Preliminary Study of the Scale To Assess Ataxia and Neurologic Dysfunction (STAND)

Who this might be right for
Spinocerebellar Ataxia - All Sub-typesFriedreich's Ataxia
University of South Florida 30
Not applicableStudy completedNCT04086511
What this trial is testing

PANDA: PKU Amino Acid Evaluation

Who this might be right for
Phenylketonuria (PKU)
Nutricia Research 37
Not applicableStudy completedNCT02934399
What this trial is testing

Dynamic Hormone Diagnostics in Endocrine Disease

Who this might be right for
Adrenal InsufficiencyCongenital Adrenal HyperplasiaCushing Syndrome+3 more
Haukeland University Hospital 528
Not applicableUnknownNCT03278899
What this trial is testing

Prospectively Assess Disease Progression in Male Children With X-ALD

Who this might be right for
X-Linked Adrenoleukodystrophy
NeuroVia, Inc. 100
Early research (Phase 1)Active Not RecruitingNCT07255599
What this trial is testing

Evaluation of BH4 Responsiveness in Our PKU Patients

Who this might be right for
Phenylketonuria (PKU)
Sohag University 20
Not applicableLooking for participantsNCT06838143
What this trial is testing

Ilaris NIS in Korea

Who this might be right for
Hereditary Periodic Fever SyndromesCryopyrin-associated Periodic Syndromes (CAPS)Colchicine Resistance Familial Mediterranean Fever (crFMF)+3 more
Novartis Pharmaceuticals 25
Not applicableUnknownNCT05679076
What this trial is testing

A Qualitative Study on the Pain and Quality of Life of Patients With Fabry Disease

Who this might be right for
Fabry Disease
Yonsei University 10
Not applicableStudy completedNCT01314560
What this trial is testing

Study of the Pathophysiological Mechanisms Involved in Bleeding Events

Who this might be right for
Oculocerebrorenal Syndrome
Assistance Publique - Hôpitaux de Paris 30
Post-approval studies (Phase 4)Active Not RecruitingNCT06901323
What this trial is testing

Effect of L-carnitine Supplementation on Phenylalanine and Brain-derived Neurotrophic Factor Levels in Infants and Children With Phenylketonuria

Who this might be right for
Phenylketonuria
Mansoura University 176
Not applicableStudy completedNCT02555579
What this trial is testing

Simplified Diet Approach in Phenylketonuria

Who this might be right for
Phenylketonuria
University of Nebraska 33
Not applicableStudy completedNCT00465062
What this trial is testing

Evaluate and Characterize the Effect of Pharmacological Chemicals on Blood From Patients With Gaucher Disease

Who this might be right for
Gaucher Disease
National Institute of Neurological Disorders and Stroke (NINDS) 50
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