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 “Sphingolipidoses”

547 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 547 results

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
Not applicableEnded earlyNCT03896607
What this trial is testing

Liver and the Spleen Stifness in Adult Patients With Gaucher's Disease Using Ultrasound Shear Wave Elastography

Who this might be right for
Gaucher DiseaseLiver FibrosisSpleen; Fibrosis
HaEmek Medical Center, Israel 18
Large-scale testing (Phase 3)Active Not RecruitingNCT03614234
What this trial is testing

Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients

Who this might be right for
Fabry Disease
Chiesi Farmaceutici S.p.A. 29
Testing effectiveness (Phase 2)Study completedNCT01685216
What this trial is testing

Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease

Who this might be right for
Gaucher Disease, Type 3
Shire 7
Large-scale testing (Phase 3)Active Not RecruitingNCT05222906
What this trial is testing

Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3

Who this might be right for
Gaucher's Disease Type III
Sanofi 43
Testing effectiveness (Phase 2)Study completedNCT00813865
What this trial is testing

A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients

Who this might be right for
Gaucher DiseaseType 1 Gaucher DiseaseGaucher Disease, Type 1
Amicus Therapeutics 8
Testing effectiveness (Phase 2)Study completedNCT00084084
What this trial is testing

Replagal Enzyme Replacement Therapy for Children With Fabry Disease

Who this might be right for
Fabry Disease
Shire 17
Testing effectiveness (Phase 2)Ended earlyNCT00526071
What this trial is testing

Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study

Who this might be right for
Fabry Disease
Amicus Therapeutics 23
Not applicableNo Longer AvailableNCT03639844
What this trial is testing

BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study

Who this might be right for
Hurler SyndromeInherited Metabolic DisorderLysosomal Storage Disorder+2 more
Bellicum Pharmaceuticals
Not applicableLooking for participantsNCT06813443
What this trial is testing

Characterization of Patients With Cardiomyopathy to Identify Critical Patients Candidates for Cardiac Transplantation

Who this might be right for
CardiomyopathiesAmyloidosis CardiacFabry Disease+14 more
IRCCS Azienda Ospedaliero-Universitaria di Bologna 700
Not applicableUnknownNCT05761834
What this trial is testing

FASHION Fabry Disease Hypertrophic Cardiomyopathy and Infammation

Who this might be right for
Fabry Disease
Fondazione Policlinico Universitario Agostino Gemelli IRCCS 150
Post-approval studies (Phase 4)Study completedNCT04120506
What this trial is testing

Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV)

Who this might be right for
Gaucher Disease, Type 1
Shaare Zedek Medical Center 15
Not applicableLooking for participantsNCT05843552
What this trial is testing

Extracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher Disease

Who this might be right for
Gaucher Disease
University of Minnesota 30
Large-scale testing (Phase 3)Active Not RecruitingNCT05280548
What this trial is testing

Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease

Who this might be right for
Fabry Disease
Sanofi 104
Not applicableEnded earlyNCT04008849
What this trial is testing

A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456

Who this might be right for
Inherited Metabolic Disorders (IMD)
Magenta Therapeutics, Inc. 3
Not applicableLooking for participantsNCT04880356
What this trial is testing

Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.

Who this might be right for
Inherited DiseaseRare DiseasesMetabolic Disease+3 more
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta 100
Not applicableUnknownNCT04324775
What this trial is testing

Comparison of Acute Effect of Different Manual Techniques on Muscle Fascia Stiffness

Who this might be right for
Manual Lymphatic DrainageSwedish MassageMuscle Fascia Stiffness
Marmara University 30
Not applicableAvailableNCT04316637
What this trial is testing

Early Access Program With Arimoclomol in US Patients With NPC

Who this might be right for
Niemann-Pick Disease, Type C
ZevraDenmark
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 applicableWithdrawnNCT01331642
What this trial is testing

Biomarker for Gaucher Disease: BioGaucher (BioGaucher)

Who this might be right for
SplenomegalyHepatomegaly
CENTOGENE GmbH Rostock
Load More Results