Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.

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 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.

View on ClinicalTrials.gov ↗

View your shortlist →

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

Pompe Disease

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

FieldValue
Enrollment Target 60 participants
Start Date 2005-05
Est. Completion 2005-10

Sponsor

Genzyme, a Sanofi Company

196 total trials

Interested in This Trial?

Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT00113035

The ClinicalTrials.gov registry entry for NCT00113035 describes a study currently listed as completed, categorized as an unspecified phase. The registered enrollment target is 60 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 228-participant average among 14 other Pompe Disease trials with a reported enrollment target (74% lower). The listed sponsor is Genzyme, a Sanofi Company, which has 196 total studies on file at ClinicalTrials.gov.

The record links to 1 condition, with Pompe Disease appearing as the primary indexed condition, and to 0 interventions.

NCT00113035 reports 5 study locations spanning 5 distinct geographic areas - top geographies include 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

Similar trials for Pompe Disease

Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.

Explore more on PlainTrial

Pompe Disease

All registered trials for this condition

California

All trials with a study site in California

Recruiting now

Find trials open to new participants near you

Rankings

Top sponsors, conditions, and phases

Read our methodology - how this data is sourced, computed, and verified.

Related

Browse all → | Rankings →
Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.