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)WithdrawnNCT03196765
What this trial is testing

Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy

Who this might be right for
X-Linked Adrenoleukodystrophy
NeuroVia, Inc.
Not applicableUnknownNCT04605523
What this trial is testing

Neurofilament Light- Chain in Ataxia Telangiectasia

Who this might be right for
Ataxia Telangiectasia
Johann Wolfgang Goethe University Hospital 40
Not applicableActive Not RecruitingNCT03305250
What this trial is testing

Arrhythmia Burden, Risk of Sudden Cardiac Death and Stroke in Patients With Fabry Disease

Who this might be right for
Fabry Disease
University Hospital Birmingham NHS Foundation Trust 169
Not applicableLooking for participantsNCT06806813
What this trial is testing

Italian Anderson Fabry Disease Cardiovascular Registry

Who this might be right for
Fabry Disease
IRCCS Azienda Ospedaliero-Universitaria di Bologna 800
Not applicableEnded earlyNCT05008874
What this trial is testing

Study of Disease Progression in Adults With Inherited Forms of Spastic Paraplegia

Who this might be right for
AMNAMN Gene MutationX-ALD
SwanBio Therapeutics, Inc. 65
Not applicableStudy completedNCT05123768
What this trial is testing

Genetic Etiology in Patients With Cerebral Palsy

Who this might be right for
Cerebral PalsyGenetic DiseaseDevelopmental Delay+1 more
University Medical Centre Ljubljana 150
Large-scale testing (Phase 3)Study completedNCT02536755
What this trial is testing

Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies

Who this might be right for
Gaucher Disease
Genzyme, a Sanofi Company 31
Not applicableEnrolling By InvitationNCT00001215
What this trial is testing

Genetic Studies of Lysosomal Storage Disorders

Who this might be right for
Lysosomal Storage DisordersGaucher DiseaseParkinson Disease
National Human Genome Research Institute (NHGRI) 1,000
Testing effectiveness (Phase 2)Study completedNCT05443685
What this trial is testing

ADX-629 Therapy for Sjogren-Larsson Syndrome

Who this might be right for
Sjogren-Larsson Syndrome
University of Nebraska 8
Testing effectiveness (Phase 2)UnknownNCT05768035
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 applicableStudy completedNCT00001325
What this trial is testing

Metabolic Abnormalities in Children With Epilepsy

Who this might be right for
Generalized EpilepsyInfantile SpasmsMetabolic Disease+2 more
National Institute of Neurological Disorders and Stroke (NINDS) 80
Early research (Phase 1)Study completedNCT05285540
What this trial is testing

Study to Evaluate DT-216 in Adult Patients With Friedreich Ataxia

Who this might be right for
Friedreich Ataxia
Design Therapeutics, Inc. 39
Large-scale testing (Phase 3)Study completedNCT03500094
What this trial is testing

Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)

Who this might be right for
Fabry Disease
Amicus Therapeutics 22
Testing effectiveness (Phase 2)Looking for participantsNCT03952637
What this trial is testing

Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis

Who this might be right for
Lysosomal DiseasesGangliosidosisGM1
National Human Genome Research Institute (NHGRI) 54
Testing effectiveness (Phase 2)Study completedNCT00633139
What this trial is testing

Long-term Metazym Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)

Who this might be right for
Late Infantile Metachromatic Leukodystrophy
Shire 13
Not applicableStudy completedNCT04997772
What this trial is testing

Digital Health Platform Customized for Patients With Gaucher Disease

Who this might be right for
Gaucher DiseasePatient Reported Outcome
Shoshana Vilk 80
Not applicableUnknownNCT03199001
What this trial is testing

Native T1 Mapping by Cardiovascular Magnetic Resonance Imaging in Rare Diseases

Who this might be right for
Fabry Disease
University College, London 400
Not applicableAvailableNCT07762638
What this trial is testing

Post Trial Access to Lucerastat for Patients With Fabry Disease Who Participated in Study ID-069A302

Who this might be right for
Fabry Disease
Idorsia Pharmaceuticals Ltd.
Testing effectiveness (Phase 2)WithdrawnNCT00657748
What this trial is testing

Lithium and Acetate for Canavan Disease

Who this might be right for
Canavan DiseaseInfantileDeficiency Disease+2 more
Assistance Publique - Hôpitaux de Paris
Early research (Phase 1)WithdrawnNCT01787578
What this trial is testing

Safety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD)

Who this might be right for
X-Linked AdrenoleukodystrophyAdrenomyeloneuropathy
Thomas S. Scanlan
Next Page