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Results for “Brain Diseases, Metabolic, Inborn”

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Showing 20 of 0 results

Early research (Phase 1)Study completedNCT02750332
What this trial is testing

Bioavailability Food-Effect Study of an Oral Nitisinone Formulation to Treat Hereditary Tyrosinemia (HT-1)

Who this might be right for
Hereditary Tyrosinemia, Type I
Cycle Pharmaceuticals Ltd. 20
Early research (Phase 1)Active Not RecruitingNCT06517914
What this trial is testing

A First-in-Human, Single- and Multiple-Ascending Dose Study of YH35995 in Healthy Adult Male Participants

Who this might be right for
Healthy Participants
Yuhan Corporation 94
Not applicableStudy completedNCT02069509
What this trial is testing

Patient Registry of the European Friedreich's Ataxia Consortium for Translational Studies (EFACTS)

Who this might be right for
Friedreich's Ataxia
European Friedreich's Ataxia Consortium for Translational Studies 1,200
Not applicableStudy completedNCT01178164
What this trial is testing

Prevalence of Fabry's Disease in a Population of Patients With Chronic Pains

Who this might be right for
PainFabry's Disease
University Hospital, Bordeaux 137
Not applicableLooking for participantsNCT06036693
What this trial is testing

MPS (RaDiCo Cohort) (RaDiCo-MPS)

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis III+5 more
Institut National de la Santé Et de la Recherche Médicale, France 1,000
Not applicableStudy completedNCT04310163
What this trial is testing

Interviews and Video Capture in Patients With GM1 Gangliosidosis

Who this might be right for
GM1 Gangliosidosis
LYSOGENE 25
Post-approval studies (Phase 4)Ended earlyNCT01526785
What this trial is testing

Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease

Who this might be right for
Pompe Disease
Genzyme, a Sanofi Company 113
Not applicableLooking for participantsNCT05554835
What this trial is testing

Global Registry and Natural History Study for Mitochondrial Disorders

Who this might be right for
Mitochondrial DiseasesKearns-Sayre SyndromeMIDD+15 more
LMU Klinikum 6,000
Large-scale testing (Phase 3)Study completedNCT06211478
What this trial is testing

Role of Vitamin E in Gaucher Disease Patients

Who this might be right for
Gaucher Disease
Ain Shams University 40
Early research (Phase 1)Study completedNCT00001328
What this trial is testing

Gene Therapy for the Treatment of Brain Tumors

Who this might be right for
Brain NeoplasmNeoplasm Metastasis
National Institute of Neurological Disorders and Stroke (NINDS) 15
Early research (Phase 1)WithdrawnNCT04014712
What this trial is testing

O2 Transport and Utilization in Health and Lung Disease

Who this might be right for
COPDTetrahydrobiopterin DeficiencyOxidative Stress
University of Massachusetts, Amherst
Post-approval studies (Phase 4)Study completedNCT01965912
What this trial is testing

Kuvan®'s Effect on the Cognition of Children With Phenylketonuria

Who this might be right for
Phenylketonuria
BioMarin Pharmaceutical 34
Not applicableUnknownNCT04037189
What this trial is testing

Treatment of Leukemia and Lymphoma in Children With Ataxia Telangiectasia

Who this might be right for
Ataxia TelangiectasiaLeukemiaLymphoma
Rabin Medical Center 250
Not applicableStudy completedNCT06935578
What this trial is testing

RAre, But Not aLone: a Large Italian Network to Empower the Impervious diaGNostic Pathway of Rare cerEbrovascular Diseases (ALIGNED)

Who this might be right for
CADASILCADASIL (Diagnosis)Moya Moya Disease+5 more
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta 500
Not applicableStudy completedNCT01358188
What this trial is testing

Exploration of Immunity in Gaucher Disease

Who this might be right for
Gaucher Disease
O & O Alpan LLC 33
Post-approval studies (Phase 4)Active Not RecruitingNCT05003648
What this trial is testing

Treating Leg Symptoms in Women With X-linked Adrenoleukodystrophy

Who this might be right for
AdrenoleukodystrophyRestless Legs Syndrome
Massachusetts General Hospital 24
Large-scale testing (Phase 3)Study completedNCT01376908
What this trial is testing

Kuvan® in Phenylketonuria Patients Less Than 4 Years Old

Who this might be right for
Phenylketonuria
BioMarin Pharmaceutical 56
Testing effectiveness (Phase 2)Study completedNCT00304512
What this trial is testing

A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease

Who this might be right for
Fabry Disease
Amicus Therapeutics 9
Post-approval studies (Phase 4)Study completedNCT01082328
What this trial is testing

Response to Kuvan® in Subjects With Phenylketonuria (PKU) in a 4 Weeks Testing Period

Who this might be right for
Phenylketonuria
Merck KGaA, Darmstadt, Germany 59
Not applicableLooking for participantsNCT06906367
What this trial is testing

Patients With Fabry Disease (US Specific)

Who this might be right for
Fabry Disease
Amicus Therapeutics 450
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