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.

An Open-Label Extension Study of the Long-Term Safety and Efficacy of Recombinant Human Acid α-Glucosidase (rhGAA) Given as Enyzme Replacement Therapy to a Single Patient With Pompe Disease (Glycogen Storage Disease Type II) Who Were Previously Enrolled in Genzyme-Sponsored Enzyme Replacement Therapy Studies

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00765414

Enrollment

Registered

2008-10-03

Start date

2003-04-30

Completion date

2006-08-31

Last updated

2014-02-05

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Glycogen Storage Disease Type II GSD II, Pompe Disease Late-Onset

Brief 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

BIOLOGICALMyozyme

30 mg/kg qow f and 40 mg/kg qow

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

FEMALE

Age

16 Years to No maximum

Healthy volunteers

Inclusion criteria

* Provide written informed consent prior to participating in any study related procedures; * Currently enrolled in Protocol AGLU01402 * Have the ability to comply with the clinical protocol, which required extensive clinical evaluations for an extended period of time.

Exclusion criteria

* Was pregnant or unwilling to use approved birth control during the course of the study; * Had experienced any unmanageable AEs under Protocol AGLU01402 (as determined and agreed upon by the Principal Investigator and sponsor) due to rhGAA that would preclude continuing ERT; * Was participating in any other investigational study.

Design outcomes

Primary

Measure	Time frame
The objective of this extension study was to monitor the long-term safety and efficacy of a single patient	3 years

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026