Crick
HomeTrialsGenesDrugs23andMeGraphBlogContact
  • Home
  • Trials
  • Genes
  • Drugs
  • 23andMe
  • Graph
  • Blog
  • Contact
Crick

An open-source clinical intelligence platform. Explore clinical trials, gene networks, and molecular structures using public data sources.

Data Sources

  • ClinicalTrials.gov
  • OpenTargets
  • ClinVar
  • PubChem

Links

  • Contact
  • Privacy

© 2026 Crick. All rights reserved.

Crick is for educational purposes only. Not medical advice.

Results for “Brain Diseases, Metabolic, Inborn”

Filters

Phase
Early Phase 1
Phase 1
Phase 2
Phase 3
Phase 4
Status
Recruiting
Active, Not Recruiting
Not Yet Recruiting
Completed
Enrolling by Invitation

Showing 20 of 0 results

Not applicableNot Yet RecruitingNCT07046338
What this trial is testing

Lentiviral Hematopoietic Stem Cell Gene Therapy for MLD

Who this might be right for
Metachromatic Leukodystrophy (MLD)
Shenzhen Geno-Immune Medical Institute 10
Not applicableLooking for participantsNCT07551921
What this trial is testing

PheCheck™ Validation Study

Who this might be right for
Phenylketonuria (PKU) and Hyperphenylalaninemia
Aptatek BioSciences, Inc 60
Large-scale testing (Phase 3)Study completedNCT00905268
What this trial is testing

Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia (FRDA) Patients

Who this might be right for
Friedreich's Ataxia
Santhera Pharmaceuticals 232
Large-scale testing (Phase 3)Active Not RecruitingNCT04020055
What this trial is testing

Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease

Who this might be right for
Fabry Disease
Amicus Therapeutics 14
Not applicableUnknownNCT04943991
What this trial is testing

Fabry Disease in High-risk Patients With Left Ventricular Hypertrophy: Prevalence and Implementation of a Clinical Score

Who this might be right for
Fabry DiseaseFabry Disease, Cardiac VariantLysosomal Storage Diseases+2 more
Wuerzburg University Hospital 200
Not applicableLooking for participantsNCT05586243
What this trial is testing

MAGNETIC RESONANCE SPECTROSCOPY BIOMARKERS IN TYPE 3 GAUCHER DISEASE (GD3)

Who this might be right for
Gaucher Disease, Type 3
University of Minnesota 5
Large-scale testing (Phase 3)Study completedNCT02415127
What this trial is testing

Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia

Who this might be right for
Friedreich's Ataxia
Amgen 92
Not applicableStudy completedNCT01773954
What this trial is testing

Repeated Eye Injections of Aflibercept for Treatment of Wet Age Related Macular Degeneration

Who this might be right for
Age-Related Macular DegenerationMacular Degeneration
MidAtlantic Retina 40
Not applicableStudy completedNCT00986973
What this trial is testing

Fluorodeoxyglucose Positron Emission Tomography (FDG PET) Findings in Patients With Phenylketonuria Before and After KUVAN Therapy

Who this might be right for
Phenylketonuria
Children's Hospital of Philadelphia 6
Not applicableLooking for participantsNCT07698743
What this trial is testing

Eating Disorders in Patients With Phenylketonuria

Who this might be right for
Phenylketonuria
Central Hospital, Nancy, France 70
Not applicableStudy completedNCT03418740
What this trial is testing

Neurology Measures in FA Children

Who this might be right for
Friedreich Ataxia
Children's Hospital of Philadelphia 108
Not applicableLooking for participantsNCT06623890
What this trial is testing

Learn More About the Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Who Are Prescribed it by Their Own Doctors

Who this might be right for
Friedreich Ataxia
Biogen 300
Large-scale testing (Phase 3)WithdrawnNCT04418024
What this trial is testing

Efficacy and Safety of AG10 in Subjects with Transthyretin Amyloid Polyneurophathy

Who this might be right for
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
Eidos Therapeutics, a BridgeBio company
Not applicableStudy completedNCT03058848
What this trial is testing

Evaluation of PKU Start

Who this might be right for
PhenylketonuriaInborn Errors of Metabolism
Vitaflo International, Ltd 10
Large-scale testing (Phase 3)Study completedNCT02194985
What this trial is testing

Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease

Who this might be right for
Fabry Disease
Amicus Therapeutics 84
Not applicableUnknownNCT04981210
What this trial is testing

High Risk Screen of Childhood Late-onset Pompe Disease in Pediatric Outpatient Clinics

Who this might be right for
Pompe Disease
National Taiwan University Hospital 1,500
Early research (Phase 1)Looking for participantsNCT06340685
What this trial is testing

Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency

Who this might be right for
Pyruvate Dehydrogenase Complex Deficiency
Jirair Krikor Bedoyan 6
Not applicableStudy completedNCT04126005
What this trial is testing

Natural History Study of Patients With Canavan Disease (CANinform Study)

Who this might be right for
Canavan Disease
Aspa Therapeutics 67
Not applicableWithdrawnNCT01093105
What this trial is testing

An Observational Study of Pediatric Subjects With Globoid Cell Leukodystrophy (GLD)

Who this might be right for
Leukodystrophy, Globoid Cell
Shire
Large-scale testing (Phase 3)Study completedNCT06582524
What this trial is testing

Pegzilarginase in Subjects <24 Months Old With Arginase 1 Deficiency

Who this might be right for
Arginase 1 Deficiency
Immedica Pharma AB 3
Next Page