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Results for “Carbohydrate Metabolism, Inborn Errors”

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Showing 20 of 0 results

Not applicableStudy completedNCT00076323
What this trial is testing

A Test to Predict the Hemolytic Potential of Drugs in G6PD Deficiency

Who this might be right for
Glucosephosphate Dehydrogenase DeficiencyFavism
Walter Reed Army Institute of Research (WRAIR) 14
Large-scale testing (Phase 3)Active Not RecruitingNCT06149403
What this trial is testing

Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT

Who this might be right for
MPS-IH (Hurler Syndrome)
Orchard Therapeutics 41
Testing effectiveness (Phase 2)Looking for participantsNCT07123155
What this trial is testing

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

Who this might be right for
Pompe Disease
Shionogi 45
Not applicableLooking for participantsNCT04454216
What this trial is testing

GSD VI and GSD IX Natural History

Who this might be right for
Glycogen Storage Disease VIGLYCOGEN STORAGE DISEASE IXa1GLYCOGEN STORAGE DISEASE IXa2+4 more
Duke University 400
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
Early research (Phase 1)Looking for participantsNCT06340685
What this trial is testing

Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency

Who this might be right for
Pyruvate Dehydrogenase Complex Deficiency
Jirair Krikor Bedoyan 6
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 completedNCT05305391
What this trial is testing

Low-grade Inflammation and Gut Symptoms From A2 and Hydrolyzed A1 Milk

Who this might be right for
Milk Intolerance
University of Turku 37
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
Testing effectiveness (Phase 2)Study completedNCT02432768
What this trial is testing

The Effect of Triheptanoin in Adults With McArdle Disease (Glycogen Storage Disease Type V)

Who this might be right for
Glycogen Storage Disease Type V
Rigshospitalet, Denmark 22
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
Early research (Phase 1)Study completedNCT04555486
What this trial is testing

Study to Evaluate Safety, Tolerability, PK and PD of DCR-PHXC in PH Type 3 Patients

Who this might be right for
Primary Hyperoxaluria Type 3
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company 6
Not applicableEnded earlyNCT01067742
What this trial is testing

The Natural History of Mucolipidosis Type IV

Who this might be right for
Mucolipidosis Type IV
Baylor Research Institute 7
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