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Results for “Lipid Storage Disease”

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Showing 20 of 0 results

Not applicableLooking for participantsNCT06325488
What this trial is testing

Fibrosis, Inflammation, Oxygenation of Renal Tissue In FabrY Disease

Who this might be right for
Fabry DiseaseChronic Kidney Diseases
Caroline Michaela Kistorp 60
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 applicableLooking for participantsNCT05619900
What this trial is testing

Registry of Patients Diagnosed With Lysosomal Storage Diseases

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis IV A+5 more
University of California, San Francisco 250
Testing effectiveness (Phase 2)Active Not RecruitingNCT03770572
What this trial is testing

Gene Therapy for Children With CLN3 Batten Disease

Who this might be right for
CLN3Batten Disease
Neela Therapeutics 7
Testing effectiveness (Phase 2)Active Not RecruitingNCT05228145
What this trial is testing

Gene Therapy Study for Children With CLN5 Batten Disease

Who this might be right for
Neuronal Ceroid Lipofuscinosis CLN5
Neurogene Inc. 6
Testing effectiveness (Phase 2)Study completedNCT03771898
What this trial is testing

Intrathecal SHP611 in Children With Metachromatic Leukodystrophy

Who this might be right for
Metachromatic Leukodystrophy (MLD)
Shire 36
Testing effectiveness (Phase 2)Looking for participantsNCT05791864
What this trial is testing

A First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Tolerability of Gene Therapy With TTX-381 for the Ocular Manifestations Associated With Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease

Who this might be right for
Neuronal Ceroid Lipofuscinosis Type 2
Tern Therapeutics, LLC 16
Not applicableStudy completedNCT05093803
What this trial is testing

Improvement of Physical and Physiological Parameters Through the Use of a Mobile App

Who this might be right for
HypertensionDiet, HealthyBody Weight+3 more
University of Alicante 70
Not applicableLooking for participantsNCT07277361
What this trial is testing

Study of the Quality of Life of Patients With Fabry Disease Aged 65 and Over With and Without Specific Treatment

Who this might be right for
Fabry DiseaseAged 65 Years or OlderAlpha Galactosidase A Deficiency+1 more
Wladimir MAUHIN, Dr 100
Testing effectiveness (Phase 2)UnknownNCT05447494
What this trial is testing

Phase 1/2 Study of CAN103 in Subjects With Gaucher Disease

Who this might be right for
Gaucher Disease, Type 1Gaucher Disease, Type 3
CANbridge (Suzhou) Bio-pharma Co., Ltd. 40
Post-approval studies (Phase 4)Study completedNCT00365131
What this trial is testing

Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.

Who this might be right for
Gaucher Disease Type ICerebroside Lipidosis SyndromeClucocerebrosidase Deficiency Disease+2 more
Genzyme, a Sanofi Company 40
Early research (Phase 1)Study completedNCT02082327
What this trial is testing

Evaluate the Safety of Migalastat Hydrochloride Given Intravenously to Healthy Volunteers

Who this might be right for
Fabry Disease
Amicus Therapeutics 31
Early research (Phase 1)Study completedNCT00418561
What this trial is testing

Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)

Who this might be right for
Metachromatic Leukodystrophy (MLD)
Shire 13
Not applicableStudy completedNCT02956954
What this trial is testing

Follow-up of Myocardial T1 Relaxation Time in Patients With Anderson Fabry Disease

Who this might be right for
Anderson-Fabry Disease
University Hospital, Rouen 26
Not applicableUnknownNCT03683966
What this trial is testing

MigALastat Therapy Adherence Among FABRY Patients: A Prospective Multicentral Observational Study

Who this might be right for
Rare DiseasesFabry DiseaseAdherence, Medication+1 more
Wuerzburg University Hospital 30
Not applicableWithdrawnNCT03145779
What this trial is testing

Evaluation of Phenotypic Variability in Fabry Disease

Who this might be right for
Fabry Disease
Boston Children's Hospital
Large-scale testing (Phase 3)Study completedNCT01124643
What this trial is testing

Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease

Who this might be right for
Fabry Disease
Shire 35
Not applicableLooking for participantsNCT00196742
What this trial is testing

Fabry Disease Registry & Pregnancy Sub-registry

Who this might be right for
Fabry Disease
Genzyme, a Sanofi Company 9,000
Not applicableActive Not RecruitingNCT07506083
What this trial is testing

Natural History in Fabry Disease With IVS4+919G>A Mutations

Who this might be right for
Fabry Disease
Chinese University of Hong Kong 31
Large-scale testing (Phase 3)Looking for participantsNCT05163288
What this trial is testing

A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C

Who this might be right for
Niemann-Pick Disease, Type C
IntraBio Inc 53
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