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 applicableLooking for participantsNCT02132741
What this trial is testing

Optical Coherence Tomography And NEphropathy: The OCTANE Study

Who this might be right for
HealthHypertensionChronic Kidney Disease
University of Edinburgh 150
Not applicableStudy completedNCT04041102
What this trial is testing

Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients

Who this might be right for
GM1 Gangliosidosis
University of Pennsylvania 31
Post-approval studies (Phase 4)Study completedNCT01257737
What this trial is testing

To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders (UCDs)

Who this might be right for
Urea Cycle Disorders
Amgen 88
Not applicableStudy completedNCT00343577
What this trial is testing

Antiproteinuric Agents and Fabry Disease

Who this might be right for
Fabry DiseaseProteinuria
University of Alabama at Birmingham 12
Not applicableStudy completedNCT04649866
What this trial is testing

Coronary Artery Disease in Patients With Friedreich's Ataxia

Who this might be right for
Friedreich Ataxia
University of Pittsburgh 7
Not applicableUnknownNCT00951886
What this trial is testing

The Validity of Forced Expiratory Maneuvers in Ataxia Telangiectasia Studied Longitudinally

Who this might be right for
Ataxia Telangiectasia
Sheba Medical Center 28
Not applicableStudy completedNCT02552628
What this trial is testing

WILSTIM - DBS (WILson STIMulation - Deep Brain Stimulation)

Who this might be right for
Severe DystoniaWilson's Disease
Hospices Civils de Lyon 5
Large-scale testing (Phase 3)Looking for participantsNCT07223944
What this trial is testing

A Gaucher Disease Gene Therapy Trial With FLT201

Who this might be right for
Gaucher Disease Type 1
Spur Therapeutics 45
Not applicableWithdrawnNCT03649919
What this trial is testing

Multi-center Clinical Study on the Diagnosis and Treatment Management of Rare Neurological Disease in Children

Who this might be right for
XlsmaDMDX-ALD+1 more
Children's Hospital of Fudan University
Not applicableLooking for participantsNCT06610019
What this trial is testing

Cardiovascular Multimodality Imaging Study

Who this might be right for
Non-ischemic CardiomyopathyCardiomyopathiesHypertrophic Cardiomyopathy+7 more
Montefiore Medical Center 10,000
Not applicableStudy completedNCT01931644
What this trial is testing

At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions

Who this might be right for
All Diagnosed Health ConditionsADD/ADHDAlopecia Areata+58 more
Sanguine Biosciences 17,667
Not applicableStudy completedNCT05641103
What this trial is testing

PREDIGA 2: Spanish Acronym of "Educational and Diagnostic Project for Gaucher and ASMD"

Who this might be right for
Gaucher DiseaseAcid SphingoMyelinase DeficiencyASMD+2 more
Fundación Española de Hematología y Hemoterapía 122
Not applicableStudy completedNCT01016392
What this trial is testing

Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency

Who this might be right for
Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or Tetrahydrobiopterin (BH4) Deficiency
BioMarin Pharmaceutical 627
Large-scale testing (Phase 3)Study completedNCT02791230
What this trial is testing

Long-term Safety of Tafamidis in Subjects With Transthyretin Cardiomyopathy

Who this might be right for
Transthyretin (TTR) Amyloid Cardiomyopathy
Pfizer 1,733
Early research (Phase 1)Study completedNCT01898364
What this trial is testing

Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.

Who this might be right for
Pompe DiseaseGlycogen Storage Disease Type II (GSD II)Acid Maltase Deficiency
Genzyme, a Sanofi Company 24
Not applicableWithdrawnNCT02778295
What this trial is testing

Biomarker for Patients With Fabry Disease (BioFabry)

Who this might be right for
AngiokeratomasChronic Kidney DiseaseOcular Abnormalities+1 more
CENTOGENE GmbH Rostock
Testing effectiveness (Phase 2)Temporarily pausedNCT04846036
What this trial is testing

The KHENERGYC Study

Who this might be right for
Mitochondrial DiseasesMitochondrial DNA tRNALeu(UUR) m.3243A<G MutationMELAS+1 more
Khondrion BV 24
Not applicableNo Longer AvailableNCT06069934
What this trial is testing

A Second Intermediate-Size Expanded Access Protocol (EAP) for Pridopidine in People With Amyotrophic Lateral Sclerosis (Pridopidine EAP 2)

Who this might be right for
Amyotrophic Lateral Sclerosis
Prilenia
Not applicableWithdrawnNCT01650909
What this trial is testing

The Effects of Sapropterin Dihydrochloride Supplementation on in Vivo Redox Status in Patients With Classical PKU

Who this might be right for
Classical Phenylketonuria(PKU)
Stanford University
Not applicableLooking for participantsNCT00668187
What this trial is testing

A Natural History Study of the Gangliosidoses

Who this might be right for
Tay-Sachs DiseaseSandhoff DiseaseLate Onset Tay-Sachs Disease+2 more
University of Minnesota 52
Next Page