CLINICAL TRIALS PROFILE FOR ALGLUCOSIDASE ALFA

All Clinical Trials for alglucosidase alfa

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00158600 ↗	A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease	Completed	Genzyme, a Sanofi Company	Phase 3	2005-09-01	Pompe disease (also known as glycogen storage disease Type II) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by the body's cells to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In patients with Pompe disease, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function. The overall objective is to evaluate the safety, efficacy, and pharmacokinetics (PK) of alglucosidase alfa treatment in patients with late-onset Pompe disease as compared to placebo.
NCT00701129 ↗	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	Completed	Genzyme, a Sanofi Company	Phase 4	2009-10-01	The purpose of this study was to evaluate the efficacy, clinical benefits and safety of a prophylactic immunomodulatory regimen given prior to first treatment with alglucosidase alfa (Myozyme®) in patients with infantile-onset Pompe disease. The objectives were to assess the efficacy of a prophylactic immunomodulatory regimen given prior to first treatment with alglucosidase alfa, as assessed by anti-recombinant human acid alpha-glucosidase (anti-rhGAA) antibody titers, and antibodies that inhibit the activity and/or uptake of alglucosidase alfa; to evaluate the clinical benefit as measured by overall survival, ventilator-free survival, left ventricular mass index (LVMI), gross motor function and development, disability index and the incidence of adverse events (AEs), serious adverse events (SAEs), and clinical laboratory abnormalities.
NCT01380743 ↗	Drug-drug Interaction Study	Completed	Amicus Therapeutics	Phase 2	2011-10-31	This study evaluates drug-drug interactions between AT2220 (duvoglustat) and recombinant human alpha-glucosidase (rhGAA, also known as alglucosidase alfa) in participants with Pompe Disease.
NCT01526785 ↗	A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease	Terminated	Genzyme, a Sanofi Company	Phase 4	2012-03-01	The objective of this study was to evaluate the efficacy and safety of treatment with 4000 litre (L) alglucosidase alfa (Lumizyme®) in Pompe participants.
NCT01898364 ↗	Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.	Completed	Genzyme, a Sanofi Company	Phase 1	2013-07-01	Primary Objective: To evaluate the safety and tolerability of neoGAA in treatment naïve and alglucosidase alfa treated late-onset Pompe disease patients. Secondary Objective: To evaluate the pharmacokinetics, pharmacodynamics of neoGAA in treatment naïve and alglucosidase alfa treated late-onset Pompe disease patients. To evaluate the effect of neoGAA on exploratory efficacy endpoints in treatment naïve and alglucosidase alfa treated late-onset Pompe disease patients.
NCT02782741 ↗	Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease	Active, not recruiting	Genzyme, a Sanofi Company	Phase 3	2016-11-02	Primary Objective: To determine the effect of avalglucosidase alfa treatment on respiratory muscle strength measured by percent (%) predicted forced vital capacity (FVC) in the upright position, as compared to alglucosidase alfa. Secondary Objective: To determine the safety and effect of avalglucosidase alfa treatment on functional endurance (6-minute walk test, inspiratory muscle strength (maximum inspiratory pressure), expiratory muscle strength (maximum expiratory pressure), lower extremity muscle strength (hand-held dynamometry), motor function (Quick Motor Function Test), and health-related quality of life (Short Form-12).

Clinical Trial Conditions for alglucosidase alfa

Condition Name

Condition Name for alglucosidase alfa
Intervention	Trials
Pompe Disease	5
Glycogen Storage Disease Type II-Pompe's Disease	2
Pompe Disease (Late-onset)	2
Glycogen Storage Disease Type II	2

Condition MeSH

Condition MeSH for alglucosidase alfa
Intervention	Trials
Glycogen Storage Disease Type II	11
Glycogen Storage Disease	6
Deficiency Diseases	1

Clinical Trial Locations for alglucosidase alfa

Trials by Country

Trials by Country for alglucosidase alfa
Location	Trials
United States	96
United Kingdom	6
Germany	6
France	6
Australia	5

Trials by US State

Trials by US State for alglucosidase alfa
Location	Trials
North Carolina	7
Florida	5
Arizona	5
New York	5
California	5

Clinical Trial Progress for alglucosidase alfa

Clinical Trial Phase

Clinical Trial Phase for alglucosidase alfa
Clinical Trial Phase	Trials
Phase 4	4
Phase 3	3
Phase 2	2

Clinical Trial Status

Clinical Trial Status for alglucosidase alfa
Clinical Trial Phase	Trials
Completed	7
Active, not recruiting	2
Recruiting	1

Clinical Trial Sponsors for alglucosidase alfa

Sponsor Name

Sponsor Name for alglucosidase alfa
Sponsor	Trials
Genzyme, a Sanofi Company	9
Amicus Therapeutics	2
Klinikum der Universitaet Muenchen	1

Sponsor Type

Sponsor Type for alglucosidase alfa
Sponsor	Trials
Industry	12
Other	2