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Results for “Storage Disease”

1,364 trials

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Showing 20 of 1,364 results

Large-scale testing (Phase 3)Study completedNCT00478647
What this trial is testing

Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase

Who this might be right for
Gaucher Disease
Shire 40
Not applicableLooking for participantsNCT07609485
What this trial is testing

Identifying Cellular and Molecular Determinants of Efficacy and Resistance in Patients Undergoing CAR-T Therapy

Who this might be right for
CancerHematologic Malignancy
University Hospital, Lille 700
Not applicableLooking for participantsNCT03564561
What this trial is testing

Natural History of Pompe Disease

Who this might be right for
Glycogen Storage Disease Type II, Adult
Assistance Publique - Hôpitaux de Paris 20
Not applicableUnknownNCT02000310
What this trial is testing

Molecular and Cellular Mechanisms of Lysosomal Storage Diseases

Who this might be right for
Lysosomal Storage Disorders
O & O Alpan LLC 80
Not applicableEnded earlyNCT04008849
What this trial is testing

A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456

Who this might be right for
Inherited Metabolic Disorders (IMD)
Magenta Therapeutics, Inc. 3
Post-approval studies (Phase 4)Study completedNCT00144768
What this trial is testing

A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients

Who this might be right for
Mucopolysaccharidosis IHurler's SyndromeHurler-Scheie Syndrome+1 more
Genzyme, a Sanofi Company 25
Not applicableStudy completedNCT00024908
What this trial is testing

Test of A Model of Representational Knowledge Stored in the Human Prefrontal Cortex

Who this might be right for
Intracranial Central Nervous System DisorderMental DisorderHealthy
National Institute of Neurological Disorders and Stroke (NINDS) 949
Not applicableActive Not RecruitingNCT04848779
What this trial is testing

A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)

Who this might be right for
Glycogen Storage Disease Type II
Sanofi 16
Testing effectiveness (Phase 2)WithdrawnNCT02715505
What this trial is testing

Safety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD)

Who this might be right for
Inherited Metabolic Disorders IMD
Novartis Pharmaceuticals
Testing effectiveness (Phase 2)Ended earlyNCT00467831
What this trial is testing

Pilot Study of a Multi-Drug Regimen for Severe Pulmonary Fibrosis in Hermansky-Pudlak Syndrome

Who this might be right for
Hermansky-Pudlak Syndrome (HPS)Pulmonary FibrosisOculocutaneous Albinism+2 more
National Human Genome Research Institute (NHGRI) 3
Not applicableStudy completedNCT04804566
What this trial is testing

Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients

Who this might be right for
Fabry Disease
Amicus Therapeutics 44
Large-scale testing (Phase 3)Study completedNCT01275066
What this trial is testing

Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Who this might be right for
MPS IV A
BioMarin Pharmaceutical 177
Not applicableUnknownNCT04476550
What this trial is testing

Clinical Specimen Collection From Pompe Disease Patients

Who this might be right for
Pompe Disease
Serhat Gumrukcu, MD PhD 12
Not applicableStudy completedNCT02368340
What this trial is testing

A Longitudinal Study of Hermansky-Pudlak Syndrome Pulmonary Fibrosis

Who this might be right for
Hermansky Pudlak Syndrome
Vanderbilt University 55
Not applicableLooking for participantsNCT06396546
What this trial is testing

'Glycogen Storage Diseases (GSDs) in Indian Children- Establishing an Indian GSD (I-GSD) Registry'

Who this might be right for
Glycogen Storage Diseases
Institute of Liver and Biliary Sciences, India 250
Not applicableLooking for participantsNCT06539169
What this trial is testing

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

Who this might be right for
Alpha-ThalassemiaBeta-ThalassemiaAmyloidosis+14 more
xCures 1,000
Not applicableStudy completedNCT04081272
What this trial is testing

Effect of G6PD Deficiency on Red Blood Cell Storage

Who this might be right for
G6PD Deficiency
Columbia University 40
Post-approval studies (Phase 4)Ended earlyNCT00230607
What this trial is testing

Study of the Effects of Fabrazyme Treatment on Lactation and Infants

Who this might be right for
Fabry DiseaseAlpha Galactosidase A Deficiency
Genzyme, a Sanofi Company 7
Not applicableStudy completedNCT00233870
What this trial is testing

A Long Term Safety and Efficacy Study of Fabrazyme Replacement Therapy in Japanese Patients With Fabry Disease.

Who this might be right for
Fabry Disease
Genzyme, a Sanofi Company 405
Early research (Phase 1)Looking for participantsNCT06567769
What this trial is testing

Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)

Who this might be right for
Sanfilippo Syndrome Type A
GC Biopharma Corp 9
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