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Results for “Acid Maltase Deficiency Disease”

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Showing 12 of 0 results

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
Early research (Phase 1)Not Yet RecruitingNCT07787650
What this trial is testing

S-606001 as Monotherapy in Participants With Late-onset Pompe Disease (LOPD)

Who this might be right for
Pompe Disease
Shionogi 15
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 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
Testing effectiveness (Phase 2)Looking for participantsNCT06391736
What this trial is testing

Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug

Who this might be right for
Pompe Disease (Late-onset)
GeneCradle Inc 33
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
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
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
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07750990
What this trial is testing

An Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD)

Who this might be right for
Pompe Disease
Shionogi 60
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