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 “Lysosomal Storage Disorders”

1,069 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 1,069 results

Testing effectiveness (Phase 2)Enrolling By InvitationNCT07393373
What this trial is testing

Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia

Who this might be right for
Hypochondroplasia
QED Therapeutics, a BridgeBio company 135
Testing effectiveness (Phase 2)Ended earlyNCT05116046
What this trial is testing

Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia

Who this might be right for
Achondroplasia
Pfizer 35
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 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
Testing effectiveness (Phase 2)Study completedNCT01102686
What this trial is testing

Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)

Who this might be right for
Gangliosidoses, GM2Sandhoff DiseaseTay-Sachs Disease
The Hospital for Sick Children 20
Testing effectiveness (Phase 2)Ended earlyNCT04273269
What this trial is testing

A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis

Who this might be right for
GM1 Gangliosidosis
LYSOGENE 5
Not applicableStudy completedNCT00351156
What this trial is testing

Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease

Who this might be right for
Gaucher Disease
Amicus Therapeutics 50
Early research (Phase 1)Study completedNCT02422654
What this trial is testing

Taste Evaluation of Different Liquid Formulations With Eliglustat

Who this might be right for
Gaucher Disease
Genzyme, a Sanofi Company 8
Not applicableLooking for participantsNCT04613089
What this trial is testing

Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD Database

Who this might be right for
Neuronal Ceroid LipofuscinosisBatten DiseaseCLN1 Disease+12 more
Universitätsklinikum Hamburg-Eppendorf 500
Not applicableLooking for participantsNCT05473637
What this trial is testing

Taiwan Associated Genetic and Nongenetic Small Vessel Disease

Who this might be right for
Cerebral Small Vessel DiseasesCadasilHTRA1-Related Autosomal Dominant Cerebral Angiopathy+5 more
National Taiwan University Hospital 500
Testing effectiveness (Phase 2)Study completedNCT02675465
What this trial is testing

First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221

Who this might be right for
Pompe Disease
Amicus Therapeutics 29
Not applicableNot Yet RecruitingNCT07516587
What this trial is testing

Study to Investigate the Efficacy of a Digestive Health Dietary Supplement on Post-meal Bloating in Healthy Participants

Who this might be right for
Digestive HealthAbdominal DiscomfortGas and Bloating
Arrae 100
Large-scale testing (Phase 3)Study completedNCT00158600
What this trial is testing

A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)Glycogen Storage Disease Type II (GSD-II)Acid Maltase Deficiency Disease+1 more
Genzyme, a Sanofi Company 90
Not applicableEnrolling By InvitationNCT06605612
What this trial is testing

Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders

Who this might be right for
Inclusion Body MyositisMyotonic DystrophyLimb-girdle and Facioscapulohumeral Muscular Dystrophies+9 more
LMU Klinikum 108
Early research (Phase 1)WithdrawnNCT00679744
What this trial is testing

A Phase I Study of Pyrimethamine in Patients With GM2 Gangliosidosis

Who this might be right for
G(M2) GangliosideTay-Sachs Disease GangliosideSandhoff Disease Ganglioside
Exsar Corporation
Not applicableUnknownNCT05719337
What this trial is testing

Multi-Modality Echocardiographic Techniques in Pathological Left Ventricular Hypertrophy Adults

Who this might be right for
Left Ventricular Hypertrophy
First Hospital of China Medical University 660
Large-scale testing (Phase 3)Study completedNCT05529992
What this trial is testing

Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease

Who this might be right for
Gaucher Disease
Takeda 20
Testing effectiveness (Phase 2)Study completedNCT03406962
What this trial is testing

MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)

Who this might be right for
Inherited Metabolic Disorders (IMD)
Magenta Therapeutics, Inc. 8
Not applicableStudy completedNCT00071903
What this trial is testing

The Role of Susceptibility to Thrombosis in the Pseudotumor Cerebri of Nephropathic Cystinosis: A Case-Control Study

Who this might be right for
Pseudotumor CerebriCystinosis
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 18
Post-approval studies (Phase 4)Study completedNCT00299000
What this trial is testing

Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI

Who this might be right for
Mucopolysaccharidosis VIMaroteaux-Lamy Syndrome
BioMarin Pharmaceutical 4
Next Page