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Results for “Inherited Metabolic Disorder of Nervous System”

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Showing 20 of 0 results

Not applicableActive Not RecruitingNCT05692596
What this trial is testing

The Pancreas Interception Center (PIC) for Early Detection, Prevention, and Novel Therapeutics

Who this might be right for
Pancreatic Ductal AdenocarcinomaPancreatic CystChronic Pancreatitis+10 more
H. Lee Moffitt Cancer Center and Research Institute 317
Large-scale testing (Phase 3)Looking for participantsNCT06953583
What this trial is testing

Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia

Who this might be right for
Friedreich Ataxia
Biogen 255
Early research (Phase 1)Ended earlyNCT00215527
What this trial is testing

Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I

Who this might be right for
Mucopolysaccharidosis ILysosomal Storage DiseasesSpinal Cord Compression
Patricia I. Dickson, M.D. 4
Large-scale testing (Phase 3)Active Not RecruitingNCT05701774
What this trial is testing

Open-Label Extension Study of DCCR in Patients With Prader-Willi Syndrome

Who this might be right for
Prader-Willi Syndrome
Soleno Therapeutics, Inc. 83
Large-scale testing (Phase 3)Ended earlyNCT03643562
What this trial is testing

Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System

Who this might be right for
Niemann-Pick Type C Disease
Mandos LLC 27
Large-scale testing (Phase 3)Looking for participantsNCT07223203
What this trial is testing

TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy

Who this might be right for
Hereditary Transthyretin-Mediated Amyloidosis With PolyneuropathyhATTR-PN
Alnylam Pharmaceuticals 125
Not applicableStudy completedNCT00414583
What this trial is testing

Stroke in Young Fabry Patients (sifap1): Frequency of Fabry Disease in Young Stroke Patients

Who this might be right for
Cerebrovascular Accident
CENTOGENE GmbH Rostock 5,000
Large-scale testing (Phase 3)Study completedNCT05184088
What this trial is testing

Efficacy of [18F]Florbetaben PET for Diagnosis of Cardiac AL Amyloidosis

Who this might be right for
Cardiac AmyloidosisAL AmyloidosisATTR Amyloidosis
Lantheus Germany GmbH 244
Not applicableLooking for participantsNCT07235709
What this trial is testing

Effect of Agalsidase Alfa on Cardiac Inflammation in Patients With Fabry Disease: A [18F]-FDG PET-CMR Study

Who this might be right for
Fabry Disease
Yonsei University 25
Not applicableLooking for participantsNCT04675749
What this trial is testing

Quality of Life in Women With X-linked Adrenoleukodystrophy

Who this might be right for
X-linked Adrenoleukodystrophy
Leipzig University Medical Center 200
Not applicableStudy completedNCT01864304
What this trial is testing

Fat Distribution and Glucose Metabolism in Williams Syndrome

Who this might be right for
Williams Syndrome
Massachusetts General Hospital 24
Not applicableActive Not RecruitingNCT06239064
What this trial is testing

Early Genetic Identification of Obesity

Who this might be right for
Obesity, ChildhoodHyperphagiaRetinopathy+5 more
Rolfs Consulting und Verwaltungs-GmbH (RCV) 1,000
Not applicableLooking for participantsNCT05954416
What this trial is testing

FARD (RaDiCo Cohort) (RaDiCo-FARD)

Who this might be right for
Inherited Epidermolysis BullosaIchthyosisEctodermal Dysplasia+6 more
Institut National de la Santé Et de la Recherche Médicale, France 900
Not applicableLooking for participantsNCT05489549
What this trial is testing

Subclinical Transthyretin Cardiac Amyloidosis in V122I TTR Carriers

Who this might be right for
Amyloidosis, HereditaryAmyloidosis CardiacAmyloidosis, Familial+2 more
University of Texas Southwestern Medical Center 500
Large-scale testing (Phase 3)Study completedNCT01104649
What this trial is testing

Efficacy of Riluzole in Hereditary Cerebellar Ataxia

Who this might be right for
Cerebellar Ataxia
S. Andrea Hospital 60
Testing effectiveness (Phase 2)Study completedNCT03962114
What this trial is testing

Effects of Vitamin B3 in Patients With Ataxia Telangiectasia

Who this might be right for
Ataxia TelangiectasiaATM Gene Mutation
Radboud University Medical Center 24
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07410455
What this trial is testing

An Open-label, Phase 2 Pilot Study on the Efficacy and Safety of Piclidenoson in Patients With Lowe Syndrome

Who this might be right for
Lowe Syndrome
Can-Fite BioPharma 5
Not applicableLooking for participantsNCT02795052
What this trial is testing

Neurologic Stem Cell Treatment Study

Who this might be right for
Neurologic DisordersNervous System DiseasesNeurodegenerative Diseases+23 more
MD Stem Cells 500
Not applicableStudy completedNCT04577170
What this trial is testing

Pulsatility Index, Vasomotor Reactivity and Leukoencephalopathy in Fabry Patients

Who this might be right for
Fabry Disease
National and Kapodistrian University of Athens 45
Not applicableStudy completedNCT06419231
What this trial is testing

Effectiveness of a Butyrate Formulation and Butyrate + Polyphenol Formulation on Gut Health, Permeability and Associated Symptoms

Who this might be right for
Gut Health
Supplement Formulators, Inc. 143
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