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NCT00113035 · ClinicalTrials.gov registry record
Screening Protocol to Evaluate Acid Alpha-Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease
A clinical trial of Pompe Disease, sponsored by Genzyme, a Sanofi Company.
- Completed
- Registry status
- 60
- Enrollment target
- 5
- Study locations
NCT00113035: Completed study of Pompe Disease, sponsored by Genzyme, a Sanofi Company.
NCT00113035 is a study of Pompe Disease that has completed, run by Genzyme, a Sanofi Company. The registered enrollment target is 60 participants, below the 228-participant average among 14 other Pompe Disease trials with a reported enrollment target (74% lower). The trial reports 5 study locations across 5 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00113035, a study of Pompe Disease, has completed, sponsored by Genzyme, a Sanofi Company.
- COMPLETED
- Registry status
- 60 participants
- Enrollment target
- 5
- Study locations
Study 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 primary objective of this study is to identify potential candidates for future clinical studies in Pompe disease.
Conditions Studied
Study Locations (5)
California
- Tower Hematology/Oncology Medical Group - Beverly Hills
District of Columbia
- Children's National Medical Center - Washington D.C.
Missouri
- Washington University Medical Center - St Louis
New York
- Mount Sinai School of Medicine - New York
Pennsylvania
- University of Pittsburgh Medical Center - Pittsburgh
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 60 participants |
| Start Date | 2005-05 |
| Est. Completion | 2005-10 |
What the finished NCT00113035 record still lists
NCT00113035 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 60 participants, a relatively small participant target, below the 228-participant average among 14 other Pompe Disease trials with a reported enrollment target (74% lower).
The record links to 1 condition, with Pompe Disease appearing as the primary indexed condition, and to 0 interventions.
NCT00113035 lists 5 locations in 5 states (California, District of Columbia, Missouri).
Frequently Asked Questions
What is clinical trial NCT00113035 about?
NCT00113035 is a clinical study titled "Screening Protocol to Evaluate Acid Alpha-Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease". 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 ...
What is the current status of trial NCT00113035?
This trial is currently completed. The enrollment target is 60 participants. The study started on 2005-05. Estimated completion is 2005-10.
What conditions does trial NCT00113035 study?
This clinical trial studies the following conditions: Pompe Disease.
Who is sponsoring clinical trial NCT00113035?
This trial is sponsored by Genzyme, a Sanofi Company, which has 196 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT00113035 being conducted?
This trial has 5 study locations across California, District of Columbia, Missouri, New York, Pennsylvania. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT00113035's enrollment target sits among peer trials
60 5th of 14 higher than 10 of 14 other Pompe Disease trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Pompe Disease trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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