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Results for “Carbohydrate Metabolism, Inborn Errors”

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Showing 20 of 0 results

Large-scale testing (Phase 3)Study completedNCT01145586
What this trial is testing

A Non-inferiority, Multicenter and Randomized, Multiple-Dose Study About a Treatment to Hypolactasia

Who this might be right for
Lactose Intolerance
Eurofarma Laboratorios S.A. 140
Not applicableStudy completedNCT02141503
What this trial is testing

Clinical Biomarkers in Alpha-mannosidosis

Who this might be right for
Alpha-Mannosidosis
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 11
Not applicableNot Yet RecruitingNCT07666269
What this trial is testing

Morphology in Oral Rare Syndromes & Artificial Intelligence for Clinical Diagnosis

Who this might be right for
Osteogenesis ImperfectaRare Bone DisordersHypophosphatemia+6 more
University Hospital, Bordeaux 240
Not applicableStudy completedNCT06064656
What this trial is testing

A Non-Interventional Study of Clinical Characteristics and Mortality of US Patients With Fibrodysplasia Ossificans Progressiva (FOP)

Who this might be right for
Fibrodysplasia Ossificans Progressiva (FOP)
Regeneron Pharmaceuticals 131
Not applicableUnknownNCT03227042
What this trial is testing

A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

Who this might be right for
Mucopolysaccharidosis Type IIIB
Allievex Corporation 44
Post-approval studies (Phase 4)UnknownNCT02594904
What this trial is testing

Efficacy of Yinzhihuang Oral Liquid on Indirect Bilirubin of Neonates With Glucose-6-phosphate Dehydrogenase Deficiency

Who this might be right for
Neonatal Hyperbilirubinemia
Southern Medical University, China 300
Not applicableStudy completedNCT02765828
What this trial is testing

Identification of Tongue Involvement in Late-Onset Pompe Disease

Who this might be right for
MyopathyNeuropathyGlycogen Storage Disease Type II (Late-onset Pompe Disease)
Duke University 73
Large-scale testing (Phase 3)Enrolling By InvitationNCT06075537
What this trial is testing

An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007

Who this might be right for
Mucopolysaccharidosis II
Denali Therapeutics Inc. 99
Large-scale testing (Phase 3)Active Not RecruitingNCT05208281
What this trial is testing

A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II

Who this might be right for
Mucopolysaccharidosis Type IIMetabolic Diseases
AO GENERIUM 32
Testing effectiveness (Phase 2)Ended earlyNCT01609062
What this trial is testing

Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome

Who this might be right for
Mucopolysaccharidosis IVAMorquio A SyndromeMPS IVA
BioMarin Pharmaceutical 25
Not applicableUnknownNCT03211923
What this trial is testing

Muscle Relaxation in Myopathies With Positive Muscle Phenomena

Who this might be right for
Nemaline Myopathy Type 6Myotonic Dystrophy Type 2McArdle Disease
Radboud University Medical Center 30
Not applicableWithdrawnNCT00875823
What this trial is testing

International Registry for Primary Hyperoxaluria

Who this might be right for
Primary HyperoxaluriaNephrocalcinosisKidney Stones
Mayo Clinic
Not applicableStudy completedNCT02936713
What this trial is testing

Effect of Controlled Diet Combined With a Fermented Milk Product on Gas-related Symptoms

Who this might be right for
Functional Gastrointestinal Disorders
Danone Global Research & Innovation Center 126
Testing effectiveness (Phase 2)Ended earlyNCT03315182
What this trial is testing

Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB

Who this might be right for
Mucopolysaccharidosis Type 3 B
Abeona Therapeutics, Inc 11
Post-approval studies (Phase 4)Looking for participantsNCT06666413
What this trial is testing

China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD

Who this might be right for
Glycogen Storage Disease Type IIPompe's Disease
Genzyme, a Sanofi Company 13
Post-approval studies (Phase 4)Study completedNCT02636413
What this trial is testing

Evaluation of LacTEST for the Diagnosis of Hypolactasia in Adults and Elderly Patients Presenting With Clinical Symptoms of Lactose Intolerance

Who this might be right for
Lactose Intolerance
VenterPharma 1
Not applicableAvailableNCT03775174
What this trial is testing

Expanded Access to Mepsevii

Who this might be right for
MPS VIIMucopolysaccharidosis VIISly Syndrome
Ultragenyx Pharmaceutical Inc
Not applicableUnknownNCT02798718
What this trial is testing

Whole Milk Intake and Cardio-metabolic Risk Factors

Who this might be right for
Lactose MalabsorptionCardiovascular Disease
Huazhong University of Science and Technology 60
Not applicableStudy completedNCT00498420
What this trial is testing

The Natural History of Alpha-Mannosidosis

Who this might be right for
Alpha Mannosidosis
Zymenex A/S 45
Large-scale testing (Phase 3)Active Not RecruitingNCT06031259
What this trial is testing

Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment

Who this might be right for
Hunter Syndrome
Takeda 6
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