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
  • About
  • Privacy

© 2026 Crick. All rights reserved.

Crick is for educational purposes only. Not medical advice.

Results for “Brain Diseases, Metabolic, Inborn”

1,523 trials

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 1,523 results

Not applicableUnknownNCT01898325
What this trial is testing

Assessment Of Liver and Spleen Fibrosis in Patients With Gaucher Disease Using Fibroscan

Who this might be right for
Fibrosis
Rambam Health Care Campus 40
Not applicableActive Not RecruitingNCT05526664
What this trial is testing

Omics Gaucher Study: Multiomic Approach

Who this might be right for
Gaucher Disease
CENTOGENE GmbH Rostock 25
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
Testing effectiveness (Phase 2)Study completedNCT03516487
What this trial is testing

Safety and Tolerability of SYNB1618 in Healthy Adult Volunteers and Adult Subjects With Phenylketonuria

Who this might be right for
PhenylketonuriaHealthy
Synlogic 70
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07173933
What this trial is testing

Phase I/II Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of GC310 Injection in Patients With Wilson's Disease (WD)

Who this might be right for
Wilson Disease
GeneCradle Inc 15
Early research (Phase 1)Study completedNCT02536911
What this trial is testing

The Effects of Hepatic Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate

Who this might be right for
Gaucher Disease
Genzyme, a Sanofi Company 24
Not applicableLooking for participantsNCT06933212
What this trial is testing

Effect of the Mediterranean Diet in Patients Affected by CADASIL and Cerebral Amyloid Angiopathy.

Who this might be right for
Cerebral Amyloid AngiopathyCAA - Cerebral Amyloid Angiopathy
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta 86
Not applicableStudy completedNCT03432871
What this trial is testing

Nicotinamide Riboside and Mitochondrial Biogenesis

Who this might be right for
Mitochondrial DiseasesMitochondrial MyopathiesProgressive External Ophthalmoplegia+4 more
Cambridge University Hospitals NHS Foundation Trust 13
Not applicableStudy completedNCT00728364
What this trial is testing

Anderson-Fabry Disease in Chronic Kidney Disease Patients Not on Renal Replacement Therapy

Who this might be right for
Focus of Study: Prevalence of Fabry Disease in CKD Population
Klinikum Wels-Grieskirchen 4,353
Not applicableStudy completedNCT06762509
What this trial is testing

The Clinical Study of Botulinum Toxin Type A Injection in the Treatment of Wilson Disease

Who this might be right for
Wilson Disease
The First Affiliated Hospital of Anhui University of Traditional Chinese Medicine 60
Not applicableStudy completedNCT06539598
What this trial is testing

Respiratory Training in Friedreich's Ataxia

Who this might be right for
Friedreich Ataxia
University of Florida 1
Testing effectiveness (Phase 2)Ended earlyNCT03746587
What this trial is testing

Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3

Who this might be right for
Gaucher Disease, Type 1Gaucher Disease, Type 3
ZevraDenmark 39
Not applicableStudy completedNCT05083806
What this trial is testing

MSOT in Pompe Disease

Who this might be right for
Pompe DiseasePompe Disease (Late-onset)Pompe's Disease Juvenile Onset+1 more
University of Erlangen-Nürnberg Medical School 20
Not applicableNot Yet RecruitingNCT07241832
What this trial is testing

Multifaceted Assessment of Patients With Wilson's Disease in a Low-Resource Setting in Upper Egypt: Service Integration, Psychosocial Burden, Dietary Practices, and the Geo-Spatial Disease Map

Who this might be right for
Wilson's Disease
Assiut University 66
Testing effectiveness (Phase 2)Study completedNCT00391625
What this trial is testing

Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT)

Who this might be right for
Gaucher Disease
Shire 10
Large-scale testing (Phase 3)Study completedNCT03566017
What this trial is testing

Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease

Who this might be right for
Fabry Disease
Chiesi Farmaceutici S.p.A. 97
Early research (Phase 1)Ended earlyNCT05222178
What this trial is testing

Safety and Efficacy of HMI-103 in Participants With Classical PKU Due to PAH Deficiency

Who this might be right for
PhenylketonuriasPAH DeficiencyPhenylketonuria
Homology Medicines, Inc 3
Testing effectiveness (Phase 2)Looking for participantsNCT05768035
What this trial is testing

Safety and Efficacy of SMART101 in Adult Patients With Hematological Malignancies After Haploidentical HSCT With Post-transplant Cyclophosphamide

Who this might be right for
Hematological Malignancies
Smart Immune SAS 40
Not applicableEnded earlyNCT01904708
What this trial is testing

Moderate Intensity Exercise and Phenylketonuria

Who this might be right for
PhenylketonuriaPKU
Oregon Health and Science University 2
Not applicableLooking for participantsNCT06573723
What this trial is testing

Institutional Registry of Rare Diseases

Who this might be right for
Rare DiseasesAmyloidosisSarcoidosis+23 more
Hospital Italiano de Buenos Aires 380
Load More Results