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NCT00765414 · ClinicalTrials.gov registry record · Phase 2

Extension Study of Long-term Safety and Efficacy of Myozyme for a Single Patient With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored ERT Studies.

A Phase 2 study, sponsored by Genzyme, a Sanofi Company.

Completed
Registry status
Phase 2
Development phase
1
Enrollment target

NCT00765414: Completed Phase 2 study, sponsored by Genzyme, a Sanofi Company.

NCT00765414 is a Phase 2 study that has completed, run by Genzyme, a Sanofi Company. The registered enrollment target is 1 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (99% lower). According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT00765414, a Phase 2 study, has completed, sponsored by Genzyme, a Sanofi Company.

COMPLETED
Registry status
Phase 2
Development phase
1 participants
Enrollment target

Study Summary

This extension study was to monitor the long-term safety and efficacy of rhGAA treatment in a single patient with infantile-onset Pompe disease who were previously treated with rhGAA in a Genzyme study.

Interventions

  • BIOLOGICAL Myozyme

Trial Details

FieldValue
Enrollment Target 1 participants
Start Date 2003-04
Est. Completion 2006-08
Phase Phase 2
Genzyme, a Sanofi Company

196 total trials

What the finished NCT00765414 record still lists

NCT00765414 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 1 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (99% lower).

The record links to 0 conditions, and to 1 intervention - of which Myozyme is the first listed.

NCT00765414 does not publish any study locations in the registry export this page uses.

Frequently Asked Questions

What is clinical trial NCT00765414 about?

NCT00765414 is a clinical study titled "Extension Study of Long-term Safety and Efficacy of Myozyme for a Single Patient With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored ERT Studies.". This extension study was to monitor the long-term safety and efficacy of rhGAA treatment in a single patient with infantile-onset Pompe disease who were previously treated with rhGAA in a Genzyme study.

What is the current status of trial NCT00765414?

This trial is currently completed. It is a Phase 2 study. The enrollment target is 1 participants. The study started on 2003-04. Estimated completion is 2006-08.

What interventions are being tested in trial NCT00765414?

The interventions under investigation include: Myozyme (BIOLOGICAL).

Who is sponsoring clinical trial NCT00765414?

This trial is sponsored by Genzyme, a Sanofi Company, which has 196 total clinical trials registered on ClinicalTrials.gov.

How this trial's enrollment target compares

Where NCT00765414's enrollment target sits among peer trials

1 2001st of 2000 the lowest of 2,000 other Phase 2 trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Phase 2 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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Source: ClinicalTrials.gov NCT00765414, the US trial registry maintained by the National Library of Medicine. NCT00765414 (small enrollment · none site footprint · completed) retrieved and formatted by PlainTrial, see methodology.