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Results for “Acid Maltase Deficiency Disease”

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Showing 20 of 0 results

Testing effectiveness (Phase 2)Study completedNCT00976352
What this trial is testing

Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease

Who this might be right for
Pompe Disease
University of Florida 9
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)UnknownNCT02525172
What this trial is testing

Immune Modulation Therapy for Pompe Disease

Who this might be right for
Pompe Disease
National Taiwan University Hospital 8
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
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
Testing effectiveness (Phase 2)Active Not RecruitingNCT04093349
What this trial is testing

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

Who this might be right for
Pompe DiseasePompe Disease (Late-onset)Glycogen Storage Disease Type 2+4 more
Spark Therapeutics, Inc. 4
Not applicableActive Not RecruitingNCT03694561
What this trial is testing

Developing a Management Approach for Patients With "Late-Onset" Pompe Disease

Who this might be right for
Pompe DiseasePompe Disease (Late-onset)GAA Deficiency
Duke University 20
Testing effectiveness (Phase 2)Study completedNCT00051935
What this trial is testing

The Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II

Who this might be right for
Glycogen Storage Disease Type IIPompe DiseaseAcid Maltase Deficiency Disease+1 more
Genzyme, a Sanofi Company 2
Testing effectiveness (Phase 2)WithdrawnNCT01656590
What this trial is testing

High Protein and Exercise Therapy Plus Nocturnal Enteral Feeding in Juvenile-onset Pompe Disease

Who this might be right for
Glycogen Storage Disease Type II
Columbia University
Testing effectiveness (Phase 2)Study completedNCT00765414
What this trial is testing

Extension Study of Long-term Safety and Efficacy of Myozyme for a Single Patient With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored ERT Studies.

Who this might be right for
Pompe Disease Late-OnsetGlycogen Storage Disease Type II GSD II
Genzyme, a Sanofi Company 1
Not applicableStudy completedNCT00001331
What this trial is testing

Genetic and Family Studies of Inherited Muscle Diseases

Who this might be right for
DermatomyositisGlycogen Storage Disease Type IIGlycogen Storage Disease Type VII+2 more
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Post-approval studies (Phase 4)Study completedNCT00455195
What this trial is testing

Late-Onset Treatment Study Extension Protocol

Who this might be right for
Pompe Disease (Late-Onset)Glycogen Storage Disease Type II (GSD-II)Glycogenesis Type II+1 more
Genzyme, a Sanofi Company 81
Not applicableActive Not RecruitingNCT03655223
What this trial is testing

Early Check: Expanded Screening in Newborns

Who this might be right for
Spinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more
RTI International 30,000
Not applicableLooking for participantsNCT00567073
What this trial is testing

Pompe Pregnancy Sub-Registry

Who this might be right for
Glycogen Storage Disease Type II (GSD-II)Pompe Disease (Late-onset)Glycogenesis 2 Acid Maltase Deficiency
Genzyme, a Sanofi Company 20
Not applicableStudy completedNCT00113035
What this trial is testing

Screening Protocol to Evaluate Acid Alpha-Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease

Who this might be right for
Pompe Disease
Genzyme, a Sanofi Company 60
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
Not applicableLooking for participantsNCT01665326
What this trial is testing

Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease

Who this might be right for
Pompe Disease
Duke University 400
Post-approval studies (Phase 4)Not Yet RecruitingNCT06575829
What this trial is testing

Treatment Frequency Reduction in Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)GAA DeficiencyGlycogen Storage Disease Type II+1 more
Erasmus Medical Center 10
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
Testing effectiveness (Phase 2)Study completedNCT00025896
What this trial is testing

Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease

Who this might be right for
Pompe DiseaseGlycogen Storage Disease Type IIAcid Maltase Deficiency Disease+1 more
Genzyme, a Sanofi Company 8
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