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Results for “Lipid Storage Disease”

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Showing 20 of 0 results

Not applicableLooking for participantsNCT04388969
What this trial is testing

World Data on Ambroxol for Patients With GD and GBA Related PD

Who this might be right for
Gaucher DiseaseParkinson DiseaseGBA Gene Mutation
Shaare Zedek Medical Center 300
Large-scale testing (Phase 3)Ended earlyNCT04958642
What this trial is testing

Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease

Who this might be right for
Niemann-Pick Disease, Type C
Mandos LLC 66
Not applicableEnded earlyNCT00004442
What this trial is testing

Study of Bile Acids in Patients With Peroxisomal Disorders

Who this might be right for
Infantile Refsum's DiseaseZellweger SyndromeBifunctional Enzyme Deficiency+1 more
University of Cincinnati 25
Not applicableStudy completedNCT06484478
What this trial is testing

A Real-world Wearables Study in Fabry Disease.

Who this might be right for
Fabry Disease
Royal Free Hospital NHS Foundation Trust 100
Not applicableUnknownNCT01791452
What this trial is testing

Novel Association of Cholesterol Ester Storage Disease Due to Lysosomal Acid Lipase Deficiency and Non-Alcoholic Fatty Liver Disease: A Prospective Clinical Study

Who this might be right for
Non-alcoholic Fatty Liver DiseaseCholesterol Ester Storage Disease
Assy Nimer
Large-scale testing (Phase 3)Ended earlyNCT00654433
What this trial is testing

ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases

Who this might be right for
Inherited Metabolic DiseasesLysosomal Storage DisordersPeroxisomal Storage Diseases+2 more
Aldagen 40
Very early researchLooking for participantsNCT06272149
What this trial is testing

An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease

Who this might be right for
Type II Gaucher Disease
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine 6
Not applicableLooking for participantsNCT07575347
What this trial is testing

Periodontal Disease in Rare Renal Disorders (PERIO-RA-RE)

Who this might be right for
Periodontal DiseasePeriodontitisCKD+6 more
Stefan Lujinschi 100
Testing effectiveness (Phase 2)Study completedNCT02912793
What this trial is testing

Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients

Who this might be right for
Niemann-Pick Disease, Type C1
Cyclo Therapeutics, Inc. 12
Not applicableActive Not RecruitingNCT00787865
What this trial is testing

Diffusion Tensor Imaging (DTI) in Infants With Krabbe Disease

Who this might be right for
Krabbe Disease
University of Pittsburgh 100
Not applicableUnknownNCT01716728
What this trial is testing

Identification of Undiagnosed Lysosomal Acid Lipase Deficiency

Who this might be right for
Lysosomal Acid Lipase DeficiencyCholesterol Ester Storage Disease
Massachusetts General Hospital 60
Large-scale testing (Phase 3)Study completedNCT00712348
What this trial is testing

Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase

Who this might be right for
Gaucher Disease
Pfizer 31
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
Not applicableStudy completedNCT02699190
What this trial is testing

LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies

Who this might be right for
LeukodystrophyWhite Matter Disease4H Syndrome+61 more
Children's Hospital of Philadelphia 236
Testing effectiveness (Phase 2)Looking for participantsNCT06818838
What this trial is testing

A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gaucher Disease

Who this might be right for
Gaucher Disease Type 1
Lingyi Biotech Co., Ltd. 12
Early research (Phase 1)Ended earlyNCT04669535
What this trial is testing

A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease

Who this might be right for
Tay-Sachs DiseaseSandhoff Disease
Terence Flotte 9
Not applicableStudy completedNCT01899950
What this trial is testing

Longitudinal Study of Cognition With Niemann-Pick Disease, Type C

Who this might be right for
Niemann-Pick Disease, Type C
Mayo Clinic 70
Testing effectiveness (Phase 2)UnknownNCT01951989
What this trial is testing

Intra-monocyte Imiglucerase Kinetics in Gaucher Disease

Who this might be right for
Gaucher Disease
University Hospital, Clermont-Ferrand 60
Large-scale testing (Phase 3)Study completedNCT03445650
What this trial is testing

RESET Trial - Part 1 - A Phase 3 Trial in Subjects With Sjögren-Larsson Syndrome (SLS)

Who this might be right for
Sjogren-Larsson Syndrome
Aldeyra Therapeutics, Inc. 11
Testing effectiveness (Phase 2)Active Not RecruitingNCT02171104
What this trial is testing

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

Who this might be right for
Mucopolysaccharidosis DisordersHurler SyndromeHunter Syndrome+26 more
Masonic Cancer Center, University of Minnesota 149
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