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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 participantsNCT07241234
What this trial is testing

Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria

Who this might be right for
Phenylketonuria
Agios Pharmaceuticals, Inc. 20
Not applicableUnknownNCT02497807
What this trial is testing

The Pathophysiology of Tourette Syndrome: a Multimodal Study

Who this might be right for
Tourette Syndrome
Second Affiliated Hospital, Zhejiang University, School of Medicine 40
Early research (Phase 1)WithdrawnNCT00679744
What this trial is testing

A Phase I Study of Pyrimethamine in Patients With GM2 Gangliosidosis

Who this might be right for
G(M2) GangliosideTay-Sachs Disease GangliosideSandhoff Disease Ganglioside
Exsar Corporation
Not applicableStudy completedNCT01971957
What this trial is testing

Sjogren-Larsson Syndrome: Natural History, Clinical Variation and Evaluation of Biochemical Markers

Who this might be right for
Sjogren-Larsson Syndrome (SLS)
University of Nebraska 20
Not applicableUnknownNCT04758130
What this trial is testing

Getting Global Rare Disease Insights Through Technology Study

Who this might be right for
Metabolic DiseaseFabry DiseaseGaucher Disease+2 more
M.A.G.I.C. Clinic LTD 150
Testing effectiveness (Phase 2)WithdrawnNCT05146284
What this trial is testing

Study to Assess PXL770 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)

Who this might be right for
Adrenomyeloneuropathy
Poxel SA
Testing effectiveness (Phase 2)Study completedNCT06051448
What this trial is testing

Promoting Resilience in Stress Management (PRISM) and Clinical-focused Narrative (CFN) Pilot in Adults With Primary Mitochondrial Disease (PMD).

Who this might be right for
Mitochondrial DiseasesMitochondrial MyopathiesMitochondrial Encephalomyopathies+2 more
Children's Hospital of Philadelphia 16
Post-approval studies (Phase 4)Study completedNCT00364858
What this trial is testing

Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease

Who this might be right for
Gaucher Disease, Type 1Cerebroside Lipidosis SyndromeGlucocerebrosidase Deficiency Disease+2 more
Genzyme, a Sanofi Company 95
Not applicableLooking for participantsNCT04012658
What this trial is testing

A Registered Cohort Study on Wilson's Disease

Who this might be right for
Wilson's Disease
Wan-Jin Chen 2,000
Not applicableEnded earlyNCT03810508
What this trial is testing

A Natural History Study of Charcot-Marie-Tooth 4J (CMT4J)

Who this might be right for
Charcot-Marie-Tooth DiseaseHereditary Motor and Sensory Neuropathy IV
Neurogene Inc. 21
Not applicableLooking for participantsNCT06941025
What this trial is testing

Maternal and Postnatal Outcomes Study (MOS): A Global Observational Registry Assessing the Safety of Elfabrio® in Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding

Who this might be right for
Fabry DiseasePregnancyPregnancy Complications
Chiesi Farmaceutici S.p.A. 10
Post-approval studies (Phase 4)Study completedNCT00074984
What this trial is testing

The Safety and Efficacy of Fabrazyme (Agalsidase Beta) as Compared to Placebo in Patients With Advanced Fabry Disease

Who this might be right for
Fabry Disease
Genzyme, a Sanofi Company 82
Not applicableStudy completedNCT02824068
What this trial is testing

Long-term Outcome in Late-onset Pompe Disease Treated Beyond 36 Months (STIG-Pompe-Study)

Who this might be right for
Pompe Disease
LMU Klinikum 112
Not applicableStudy completedNCT01610089
What this trial is testing

Nitric Oxide Flux and Ureagenesis in Argininosuccinate Synthetase Deficiency (ASSD)(Citrullinemia I)

Who this might be right for
Citrullinemia
Baylor College of Medicine 6
Testing effectiveness (Phase 2)Looking for participantsNCT07399704
What this trial is testing

Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

Who this might be right for
GM2 GangliosidosisNiemann-Pick Type C Disease
Azafaros B.V. 21
Not applicableStudy completedNCT00056186
What this trial is testing

Transitional Life Events in Patients With Friedreich's Ataxia: Implications for Genetic Counseling

Who this might be right for
Friedreich Ataxia
National Human Genome Research Institute (NHGRI) 40
Not applicableStudy completedNCT01019629
What this trial is testing

Fabry Screening Study

Who this might be right for
Fabry Disease
Baylor Research Institute 2,724
Large-scale testing (Phase 3)Study completedNCT05529992
What this trial is testing

Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease

Who this might be right for
Gaucher Disease
Takeda 20
Testing effectiveness (Phase 2)Study completedNCT02675465
What this trial is testing

First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221

Who this might be right for
Pompe Disease
Amicus Therapeutics 29
Early research (Phase 1)Active Not RecruitingNCT02359565
What this trial is testing

Pembrolizumab in Treating Younger Patients With Recurrent, Progressive, or Refractory High-Grade Gliomas, Diffuse Intrinsic Pontine Gliomas, Hypermutated Brain Tumors, Ependymoma or Medulloblastoma

Who this might be right for
Constitutional Mismatch Repair Deficiency SyndromeLynch SyndromeMalignant Glioma+8 more
National Cancer Institute (NCI) 71
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