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Results for “Infantile-onset Pompe Disease”

25 trials

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Showing 20 of 25 results

Not applicableApproved For MarketingNCT00074919
What this trial is testing

Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-onset Pompe Disease

Who this might be right for
Glycogen Storage Disease Type IIGlycogenosis 2
Genzyme, a Sanofi Company
Post-approval studies (Phase 4)Study completedNCT03687333
What this trial is testing

Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment

Who this might be right for
Glycogen Storage Disease Type II
Genzyme, a Sanofi Company 10
Not applicableLooking for participantsNCT06833489
What this trial is testing

Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases

Who this might be right for
Rare Genetic Muscle DiseasesMuscular Dystrophy, DuchenneMuscular Dystrophy, Becker+2 more
Assistance Publique Hopitaux De Marseille 50
Post-approval studies (Phase 4)Ended earlyNCT01597596
What this trial is testing

A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease

Who this might be right for
Pompe Disease (Infantile-Onset)Glycogen Storage Disease Type II (GSD II)Glycogenosis 2+1 more
Genzyme, a Sanofi Company 4
Testing effectiveness (Phase 2)Active Not RecruitingNCT03019406
What this trial is testing

Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa

Who this might be right for
Glycogen Storage Disease Type II-Pompe's Disease
Genzyme, a Sanofi Company 22
Large-scale testing (Phase 3)Study completedNCT00059280
What this trial is testing

The Safety and Efficacy of rhGAA in Patients With Infantile-onset Pompe Disease

Who this might be right for
Glycogen Storage Disease Type II
Genzyme, a Sanofi Company 16
Testing effectiveness (Phase 2)Study completedNCT00053573
What this trial is testing

RhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease)

Who this might be right for
Glycogen Storage Disease Type IIPompe DiseaseAcid Maltase Deficiency Disease+1 more
Genzyme, a Sanofi Company 20
Not applicableEnrolling By InvitationNCT07072676
What this trial is testing

The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.

Who this might be right for
Inclusion Body MyositisMyotonic Dystrophy 1Myotonic Dystrophy 2+10 more
LMU Klinikum 30
Large-scale testing (Phase 3)Active Not RecruitingNCT04910776
What this trial is testing

Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa

Who this might be right for
Glycogen Storage Disease Type II
Sanofi 17
Not applicableStudy completedNCT05687474
What this trial is testing

Baby Detect : Genomic Newborn Screening

Who this might be right for
Congenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more
Centre Hospitalier Universitaire de Liege 6,824
Not applicableAvailableNCT04327973
What this trial is testing

Expanded Access for ATB200/AT2221 for the Treatment of IOPD

Who this might be right for
Pompe Disease Infantile-Onset
Amicus Therapeutics
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
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
Post-approval studies (Phase 4)Study completedNCT00486889
What this trial is testing

Growth and Development Study of Alglucosidase Alfa

Who this might be right for
Pompe DiseaseGlycogen Storage Disease Type II (GSD-II)Acid Maltase Deficiency Disease
Genzyme, a Sanofi Company 12
Not applicableStudy completedNCT02950298
What this trial is testing

Pompe Telemedicine Developmental Study

Who this might be right for
Pompe DiseaseGlycogen Storage Disease II
Duke University 22
Testing effectiveness (Phase 2)Study completedNCT00763932
What this trial is testing

Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) Studies

Who this might be right for
Pompe Disease Infantile-OnsetGlycogen Storage Disease Type II
Genzyme, a Sanofi Company 7
Early research (Phase 1)Looking for participantsNCT04532047
What this trial is testing

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

Who this might be right for
MPS IMPS IIMPS IVA+6 more
University of California, San Francisco 10
Large-scale testing (Phase 3)Study completedNCT00125879
What this trial is testing

Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602

Who this might be right for
Glycogen Storage Disease Type II
Genzyme, a Sanofi Company 16
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
Not applicableUnknownNCT05567627
What this trial is testing

Clinical Exploration of Adeno-associated Virus (AAV) Expressing Human Acid Alpha- Glucosidase (GAA) Gene Therapy for Patients With Infantile-onset Pompe Disease

Who this might be right for
Infantile-onset Pompe Disease
Seventh Medical Center of PLA General Hospital 6
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