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Results for “GAA Deficiency”

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Showing 19 of 0 results

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)Ended earlyNCT02898753
What this trial is testing

VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease

Who this might be right for
Pompe Disease
Valerion Therapeutics, LLC 12
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
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 participantsNCT07354724
What this trial is testing

Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease

Who this might be right for
Late-onset Pompe Disease
Denali Therapeutics Inc. 32
Post-approval studies (Phase 4)Study completedNCT00701129
What this trial is testing

An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease

Who this might be right for
Pompe DiseaseGlycogen Storage Disease Type II
Genzyme, a Sanofi Company 4
Not applicableStudy completedNCT00830583
What this trial is testing

Pompe Prevalence Study in Patients With Muscle Weakness Without Diagnosis

Who this might be right for
Pompe's Disease
Centre Hospitalier Universitaire de Nice 400
Not applicableStudy completedNCT02904395
What this trial is testing

Feasability and Interest of Screening for Infantile Pompe's Diseases at Birth

Who this might be right for
Pompe's Disease
Centre Hospitalier de Cayenne 3,413
Post-approval studies (Phase 4)Ended earlyNCT00701701
What this trial is testing

Immune Tolerance Induction Study

Who this might be right for
Pompe DiseaseGlycogen Storage Disease Type II (GSD-II)Glycogenesis 2 Acid Maltase Deficiency
Genzyme, a Sanofi Company 4
Post-approval studies (Phase 4)Study completedNCT00483379
What this trial is testing

High Dose or High Dose Frequency Study of Alglucosidase Alfa

Who this might be right for
Pompe DiseaseGlycogen Storage Disease Type II (GSD-II)Glycogenesis 2 Acid Maltase Deficiency
Genzyme, a Sanofi Company 13
Not applicableStudy completedNCT05083806
What this trial is testing

MSOT in Pompe Disease

Who this might be right for
Pompe DiseasePompe Disease (Late-onset)Pompe's Disease Juvenile Onset+1 more
University of Erlangen-Nürnberg Medical School 20
Early research (Phase 1)Study completedNCT01898364
What this trial is testing

Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.

Who this might be right for
Pompe DiseaseGlycogen Storage Disease Type II (GSD II)Acid Maltase Deficiency
Genzyme, a Sanofi Company 24
Post-approval studies (Phase 4)Active Not RecruitingNCT07652814
What this trial is testing

Safety and Efficacy of Avalglucosidase Alfa in Patients With Non-classic Pompe Disease Aged ≥ 5 Years

Who this might be right for
Glycogen Storage Disease Type II Late OnsetPompe Disease (Late-onset)Pompe Disease Late-Onset+2 more
Iris Plug 6
Not applicableStudy completedNCT00074932
What this trial is testing

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

Who this might be right for
Glycogen Storage Disease Type IIGlycogenosis 2
Genzyme, a Sanofi Company 9
Not applicableStudy completedNCT02746718
What this trial is testing

Frequency of Pompe's Disease and Neuromuscular Etiologies in Patients With Restrictive Respiratory Failure Associated With Signs of Muscle Weakness

Who this might be right for
Pompe Disease
Centre Hospitalier Universitaire de Nice 50
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