A Study of rhGAA in Patients With Late-Onset Pompe Disease

Status Completed

Clinical Trial Summary

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, pharmacokinetics (PK) and efficacy of Myozyme treatment.

Clinical Trial Description

n/a

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT00250939
Study type	Interventional
Source	Sanofi
Contact
Status	Completed
Phase	Phase 2
Start date	February 2005
Completion date	November 2006

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See also
Status	Clinical Trial	Phase
Completed	`NCT00158600` - A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease	Phase 3
Recruiting	`NCT04174105` - Gene Transfer Study in Patients With Late Onset Pompe Disease	Phase 1/Phase 2
Active, not recruiting	`NCT04093349` - A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)	Phase 1/Phase 2
Completed	`NCT03285126` - Pompe Gene Therapy- Screening for Eligibility
Recruiting	`NCT05083806` - MSOT in Pompe Disease	N/A
Recruiting	`NCT05272969` - Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe Disease
Active, not recruiting	`NCT03694561` - Developing a Management Approach for Patients With "Late-Onset" Pompe Disease
Completed	`NCT00731081` - Study About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving Myozyme®	N/A
Completed	`NCT00455195` - Late-Onset Treatment Study Extension Protocol	Phase 4
Recruiting	`NCT05951790` - Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease	N/A
Completed	`NCT01288027` - Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa	Phase 4
Recruiting	`NCT00567073` - Pompe Pregnancy Sub-Registry
Completed	`NCT03893240` - Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease	N/A
Withdrawn	`NCT04094948` - Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease	Phase 2
Recruiting	`NCT03911505` - ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD	Phase 3
Recruiting	`NCT06150820` - A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease	N/A
Completed	`NCT03729362` - A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease	Phase 3
Not yet recruiting	`NCT06178432` - Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD)	Early Phase 1
Completed	`NCT00268944` - Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support	Phase 3
Active, not recruiting	`NCT04138277` - A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD	Phase 3