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Results for “Lysosomal Storage Diseases”

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Showing 20 of 0 results

Testing effectiveness (Phase 2)Study completedNCT00391625
What this trial is testing

Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT)

Who this might be right for
Gaucher Disease
Shire 10
Post-approval studies (Phase 4)Study completedNCT03910621
What this trial is testing

Safety and Efficacy of Miglustat in Chinese NPC Patients

Who this might be right for
Niemann-Pick Disease, Type C
Actelion 17
Testing effectiveness (Phase 2)Looking for participantsNCT07282847
What this trial is testing

Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)

Who this might be right for
Pompe Disease (Late-onset)Pompe Disease Late-OnsetLOPD
AskBio Inc 12
Not applicableStudy completedNCT00852358
What this trial is testing

Intrathecal Enzyme Therapy for Cognitive Decline in MPS I

Who this might be right for
Cognitive DeclineMucopolysaccharidosis IHurler-Scheie Syndrome+2 more
Patricia I. Dickson, M.D. 9
Not applicableStudy completedNCT02838368
What this trial is testing

Investigating Pompe Prevalence in Neuromuscular Medicine Academic Practices

Who this might be right for
Pompe Disease
University of California, Irvine 921
Testing effectiveness (Phase 2)Study completedNCT00467935
What this trial is testing

Scotoma Reduction in AMD Patients Treated With Ranibizumab

Who this might be right for
Macular Degeneration
Retina Research Foundation 20
Testing effectiveness (Phase 2)WithdrawnNCT04094948
What this trial is testing

Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)
Duke University
Testing effectiveness (Phase 2)Study completedNCT00176917
What this trial is testing

Stem Cell Transplantation for Hurler

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis VIMannosidosis+1 more
Masonic Cancer Center, University of Minnesota 41
Not applicableStudy completedNCT01165697
What this trial is testing

Establishment of Biomarkers for Fabry Disease

Who this might be right for
Fabry Disease
Ohio State University 4
Large-scale testing (Phase 3)Study completedNCT00069641
What this trial is testing

Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)

Who this might be right for
Mucopolysaccharidosis II
Shire 96
Not applicableEnded earlyNCT02293408
What this trial is testing

Natural History Study to Characterise the Course of Disease Progression in Participants With Mucopolysaccharidosis Type IIIB

Who this might be right for
MPS IIIB (Sanfilippo B Syndrome)
Alexion Pharmaceuticals, Inc. 30
Not applicableNot Yet RecruitingNCT07485660
What this trial is testing

Care Pathway for Patients With Fabry's Disease (Fabry-PATH)

Who this might be right for
Fabry Disease
University Hospital, Toulouse 200
Not applicableStudy completedNCT02908724
What this trial is testing

Cardiac Involvement in Adult Patients With Fabry Disease; Relation to Enzyme Replacement Therapy

Who this might be right for
Fabry Disease
Ulla Feldt-Rasmussen 66
Not applicableStudy completedNCT01884220
What this trial is testing

Wolman/CESD Natural History Chart Review and Longitudinal Follow-Up

Who this might be right for
Wolman DiseaseCholesterol Ester Storage DiseaseAcid Cholesteryl Ester Hydrolase Deficiency, Type 2
Children's Hospital Medical Center, Cincinnati 4
Large-scale testing (Phase 3)Ended earlyNCT03822013
What this trial is testing

Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD)

Who this might be right for
GM2 GangliosidosisSupportive Care
Tehran University of Medical Sciences 30
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
Testing effectiveness (Phase 2)Active Not RecruitingNCT06545136
What this trial is testing

Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2)

Who this might be right for
Gaucher Disease, Type 1
Spur Therapeutics 75
Not applicableStudy completedNCT04678713
What this trial is testing

Maximal Fat Oxidation and Fuel Use During Exercise

Who this might be right for
Obesity
University of Copenhagen 40
Not applicableAvailableNCT05795361
What this trial is testing

Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome

Who this might be right for
Hunter Syndrome
Takeda
Not applicableUnknownNCT05843851
What this trial is testing

Genetic Newborn Screening for Cystinosis and Primary Hyperoxaluria

Who this might be right for
CystinosisPrimary Hyperoxaluria
Cystinose Stiftung 200,000
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