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Results for “Lipid Storage Disease”

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Showing 20 of 0 results

Not applicableUnknownNCT01716741
What this trial is testing

Identification of Undiagnosed Gaucher Disease

Who this might be right for
Gaucher Disease
Brigham and Women's Hospital 100
Not applicableStudy completedNCT01632111
What this trial is testing

Pulmonary Involvement in Patients With Fabry Disease

Who this might be right for
Fabry Disease
University of Zurich 110
Not applicableUnknownNCT06116071
What this trial is testing

Biomarkers Related to Bone in Pediatric Gaucher Disease

Who this might be right for
Gaucher DiseaseBone Diseases
Lysosomal and Rare Disorders Research and Treatment Center, Inc. 20
Not applicableUnknownNCT03949920
What this trial is testing

Migalastat in Fabry Disease

Who this might be right for
Fabry Disease
Manchester University NHS Foundation Trust 21
Post-approval studies (Phase 4)Study completedNCT01527318
What this trial is testing

The Effect of Fibrate Therapy in Two Patients With Neutral Lipid Storage Disease With Myopathy (NLSDM)

Who this might be right for
Neutral Lipid Storage Disease
Maastricht University Medical Center 6
Large-scale testing (Phase 3)UnknownNCT04860960
What this trial is testing

Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1

Who this might be right for
Niemann-Pick Disease, Type C1
Cyclo Therapeutics, Inc. 94
Not applicableLooking for participantsNCT03291223
What this trial is testing

Gaucher Disease Outcome Survey (GOS)

Who this might be right for
Gaucher Disease
Shire 1,257
Not applicableActive Not RecruitingNCT06614569
What this trial is testing

Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease

Who this might be right for
GM2 GangliosidosisTay Sachs DiseaseSandhoff Disease
Terence Flotte 7
Early research (Phase 1)WithdrawnNCT01003912
What this trial is testing

Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases

Who this might be right for
Lysosomal Storage DiseasesInborn Errors of Metabolism
Joanne Kurtzberg, MD
Not applicableEnded earlyNCT04094181
What this trial is testing

VPRIV in Participants With Gaucher Disease Previously Treated With Other Enzyme Replacement Therapies or Substrate Reduction Therapies

Who this might be right for
Gaucher Disease
Shire 2
Early research (Phase 1)Looking for participantsNCT04532047
What this trial is testing

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

Who this might be right for
MPS IMPS IIMPS IVA+6 more
University of California, San Francisco 10
Not applicableStudy completedNCT01358370
What this trial is testing

A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype

Who this might be right for
Lysosomal Acid Lipase DeficiencyWolman Disease
Alexion Pharmaceuticals, Inc. 40
Not applicableEnded earlyNCT04836377
What this trial is testing

A Long-Term Follow-up Study of Subjects With Gaucher Disease Who Previously Received AVR-RD-02

Who this might be right for
Type 1 Gaucher Disease
AVROBIO 4
Not applicableLooking for participantsNCT02918032
What this trial is testing

International Registry Study of Neutral Lipid Storage Disease (NLSD) / Triglyceride Deposit Cardiomyovasculopathy (TGCV) and Related Diseases

Who this might be right for
Neutral Lipid Storage Disease
Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan 120
Not applicableStudy completedNCT02969200
What this trial is testing

Fabry: Renal Function During Long-term ERT by 51Cr-EDTA Clearance

Who this might be right for
Fabry Disease
Ulla Feldt-Rasmussen 52
Testing effectiveness (Phase 2)Study completedNCT01363492
What this trial is testing

Safety Study of Replagal® Therapy in Children With Fabry Disease

Who this might be right for
Fabry Disease
Shire 15
Not applicableStudy completedNCT00106912
What this trial is testing

Study to Collect Data on Fabry Disease Patients With Enhanceable Alpha-Galactosidase A Activity

Who this might be right for
Fabry Disease
National Institute of Neurological Disorders and Stroke (NINDS) 35
Large-scale testing (Phase 3)Study completedNCT00672022
What this trial is testing

Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses

Who this might be right for
GM2 GangliosidosesTay-SachsSandhoff Disease
Children's National Research Institute 10
Not applicableStudy completedNCT05702814
What this trial is testing

A Study Measuring Substances Potentially Indicating Bone Problems in Adults With Type 1 Gaucher Condition

Who this might be right for
Gaucher Disease
Takeda 125
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07582484
What this trial is testing

Gene Therapy Trial for CLN6 Batten Disease

Who this might be right for
CLN6Batten DiseaseBatten's Disease+2 more
The Charlotte and Gwenyth Gray Foundation 12
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