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Results for “Lipid Storage Disease”

651 trials

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Showing 20 of 651 results

Early research (Phase 1)Study completedNCT05343715
What this trial is testing

PK/PD Study of 2 Agalsidase Formulations in Single Dose of 1 mg/kg Administered to Healthy Volunteers as IV Infusion

Who this might be right for
Fabry Disease
Bio Sidus SA 24
Not applicableEnded earlyNCT03886714
What this trial is testing

Screening for Fabry Disease in Renal Transplantation

Who this might be right for
Fabry DiseaseNephropathy
University Hospital, Montpellier 592
Not applicableUnknownNCT04761952
What this trial is testing

N-3 Polyunsaturated Fatty Acids Prevent Postoperative Recurrence of Crohn's Disease

Who this might be right for
Inflammatory Bowel DiseasesCrohn DiseasePolyunsaturated Acid Lipidosis
Sixth Affiliated Hospital, Sun Yat-sen University 236
Not applicableEnded earlyNCT00149318
What this trial is testing

Enzyme Replacement Therapy in Fabry Disease

Who this might be right for
Fabry Disease
Mario Negri Institute for Pharmacological Research 2
Not applicableStudy completedNCT05687474
What this trial is testing

Baby Detect : Genomic Newborn Screening

Who this might be right for
Congenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more
Centre Hospitalier Universitaire de Liege 6,824
Early research (Phase 1)Active Not RecruitingNCT04737460
What this trial is testing

Study for the Treatment for CLN7 Disease

Who this might be right for
CLN7
Benjamin Greenberg 4
Post-approval studies (Phase 4)Study completedNCT01997489
What this trial is testing

Ophthalmic Findings During 10-year Enzyme Substitution of Danish Fabry Patients.

Who this might be right for
Fabry Disease
Rigshospitalet, Denmark 39
Post-approval studies (Phase 4)Study completedNCT00028262
What this trial is testing

Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis

Who this might be right for
Infantile Neronal Ceroid Lipofuscinosis
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 10
Not applicableLooking for participantsNCT03190837
What this trial is testing

A Long-term Follow-up Study of Gaucher Disease

Who this might be right for
Gaucher Disease
Duke University 200
Not applicableStudy completedNCT00005111
What this trial is testing

PET Scans in Normal Volunteers and Patients With Fabry Disease

Who this might be right for
Fabry DiseaseHealthy
National Institute of Neurological Disorders and Stroke (NINDS) 24
Not applicableStudy completedNCT04708301
What this trial is testing

Characterisation of Heart Involvement in Fabry Disease With T1 Mapping

Who this might be right for
Fabry Disease
Manchester University NHS Foundation Trust 200
Testing effectiveness (Phase 2)Ended earlyNCT02193867
What this trial is testing

Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency

Who this might be right for
Lysosomal Acid Lipase Deficiency
Alexion Pharmaceuticals, Inc. 10
Testing effectiveness (Phase 2)Study completedNCT00196716
What this trial is testing

The Safety and Efficacy of Fabrazyme in Patients With Fabry Disease

Who this might be right for
Fabry Disease
Genzyme, a Sanofi Company 21
Large-scale testing (Phase 3)Study completedNCT03442972
What this trial is testing

Glucagon-like Peptide-2 Mediated Secretion of Stored Enteral Lipids

Who this might be right for
Hyperlipidemias
University Health Network, Toronto 21
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
Testing effectiveness (Phase 2)Study completedNCT00004488
What this trial is testing

Phase II Randomized Study of Alendronate Sodium for Osteopenia in Patients With Gaucher's Disease

Who this might be right for
Gaucher's DiseaseOsteopenia
Children's Hospital Medical Center, Cincinnati 82
Early research (Phase 1)Study completedNCT02939547
What this trial is testing

Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)

Who this might be right for
Niemann-Pick Disease, Type C1
Cyclo Therapeutics, Inc. 13
Early research (Phase 1)Study completedNCT01917708
What this trial is testing

Bone Marrow Transplant With Abatacept for Non-Malignant Diseases

Who this might be right for
Hurler SyndromeFanconi AnemiaGlanzmann Thrombasthenia+12 more
Emory University 10
Not applicableStudy completedNCT04916977
What this trial is testing

Global Burden and Treatment Trajectiories in Italian Patients With Fabry Disease

Who this might be right for
Fabry Disease
Chiesi Italia 200
Not applicableStudy completedNCT06257901
What this trial is testing

A Co-designed Physical Activity Intervention in Fabry Disease

Who this might be right for
Fabry Disease
Brunel University 41
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