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

Testing effectiveness (Phase 2)Study completedNCT00532844
What this trial is testing

A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction

Who this might be right for
Endothelial Dysfunction
BioMarin Pharmaceutical 52
Not applicableStudy completedNCT05971563
What this trial is testing

Amino Acid Kinetics of GMP-AA in Healthy Human Volunteers

Who this might be right for
Phenylketonurias
Birmingham Women's and Children's NHS Foundation Trust 20
Testing effectiveness (Phase 2)Active Not RecruitingNCT04411654
What this trial is testing

Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)

Who this might be right for
Gaucher Disease, Type 2
Prevail Therapeutics 7
Post-approval studies (Phase 4)Study completedNCT04801303
What this trial is testing

Evaluation of the Effects of Calcitriol's in the Neurological Symptoms of Friedreich's Ataxia Patients

Who this might be right for
Friedreich Ataxia
Berta Alemany 20
Large-scale testing (Phase 3)Study completedNCT01819727
What this trial is testing

An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165

Who this might be right for
Phenylketonuria
BioMarin Pharmaceutical 261
Not applicableStudy completedNCT01999257
What this trial is testing

Efficacy Study of an Online Educational Module Before Carrier Genetic Screening in Persons of Ashkenazi Jewish Descent.

Who this might be right for
Tay Sachs DiseaseCanavan DiseaseFamilial Dysautonomia
McGill University Health Centre/Research Institute of the McGill University Health Centre 60
Large-scale testing (Phase 3)Study completedNCT01411228
What this trial is testing

Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease

Who this might be right for
Gaucher Disease
Pfizer 15
Testing effectiveness (Phase 2)Ended earlyNCT01372228
What this trial is testing

Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders

Who this might be right for
Hurler Syndrome (MPS I)Hurler-Scheie SyndromeHunter Syndrome (MPS II)+9 more
Talaris Therapeutics Inc. 3
Not applicableUnknownNCT05448131
What this trial is testing

ERT in Pompe Disease: Elucidation of Molecular Structures Contributing to Enzyme Uptake and Immunoreactivity

Who this might be right for
Pompe-Disease
Centre for Analytical Biochemistry and Biomedical Mass Spectrometry 50
Testing effectiveness (Phase 2)Study completedNCT01304277
What this trial is testing

This Study is Designed to Evaluate PD/PK and Safety of Replagal Manufactured by Two Different Processes.

Who this might be right for
Fabry Disease
Shire 17
Not applicableLooking for participantsNCT00231400
What this trial is testing

Pompe Disease Registry Protocol

Who this might be right for
Glycogen Storage Disease Type IIPompe Disease
Genzyme, a Sanofi Company 2,000
Not applicableStudy completedNCT02903654
What this trial is testing

Prevalence of Heterozygote Mothers for Pompe's Disease Among Mothers Having Delivered in French Guiana

Who this might be right for
Pompe's Disease
Centre Hospitalier de Cayenne 925
Early research (Phase 1)Study completedNCT00004294
What this trial is testing

Phase I Study of Retrovirally Mediated Transfer of the Human Glucocerebrosidase Gene Into Peripheral Blood Stem Cells for Autologous Transplantation in Patients With Type I Gaucher Disease

Who this might be right for
Gaucher's Disease
National Center for Research Resources (NCRR) 15
Not applicableUnknownNCT01344096
What this trial is testing

Thrombocytopathy in Gaucher Disease Patients

Who this might be right for
Gaucher DiseaseThrombocytopathy
Rabin Medical Center 70
Not applicableStudy completedNCT00446862
What this trial is testing

The Fabrazyme® and Arbs and ACE Inhibitor Treatment (FAACET) Study

Who this might be right for
Fabry DiseaseProteinuria
University of Alabama at Birmingham 36
Early research (Phase 1)Study completedNCT04085666
What this trial is testing

Pharmacodynamics, Safety, Tolerability and Pharmacokinetics of CDX-6114 in Patients With Phenylketonuria (PKU)

Who this might be right for
Phenylketonuria
Société des Produits Nestlé (SPN) 18
Not applicableLooking for participantsNCT06325488
What this trial is testing

Fibrosis, Inflammation, Oxygenation of Renal Tissue In FabrY Disease

Who this might be right for
Fabry DiseaseChronic Kidney Diseases
Caroline Michaela Kistorp 60
Not applicableStudy completedNCT00004753
What this trial is testing

Long-Term Study of Cerebral Glucose Metabolism in Huntington's Disease

Who this might be right for
Huntington's Disease
National Center for Research Resources (NCRR) 90
Early research (Phase 1)Study completedNCT04095273
What this trial is testing

Study to Test How Well Patients With Advanced Solid Tumors Respond to Treatment With the Elimusertib in Combination With Pembrolizumab, to Find the Optimal Dose for Patients, How the Drug is Tolerated and the Way the Body Absorbs, Distributes and Discharges the Drug

Who this might be right for
Advanced Solid Tumors
Bayer 56
Large-scale testing (Phase 3)Study completedNCT03485677
What this trial is testing

Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3

Who this might be right for
Gaucher's Disease Type IGaucher's Disease Type III
Sanofi 57
Next Page