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.

Glycogen Storage Disease Type II GSD II Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00765414
• Other study ID #: AGLU02103
• Status: Completed
• Phase: Phase 2
• First received: October 2, 2008
• Last updated: February 4, 2014
• Start date: April 2003
• Est. completion date: August 2006

Study information

• Verified date: February 2014
• Source: Sanofi
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 1
• Est. completion date: August 2006
• Est. primary completion date: April 2006
• Accepts healthy volunteers: No
• Gender: Female
• Age group: 16 Years and older

Eligibility

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.

Study Design

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

Related Conditions & MeSH terms

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

Intervention

Biological:
• Myozyme
30 mg/kg qow f and 40 mg/kg qow

Locations

Country	Name	City	State
United States	University of Texas Health Science Center at San Antonio	San Antonio	Texas

Sponsors (1)

Lead Sponsor	Collaborator
Genzyme, a Sanofi Company

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The objective of this extension study was to monitor the long-term safety and efficacy of a single patient		3 years	No