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OpenTrials
Completed

NCT Number: NCT00765414

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.

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Key information

Conditions

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

Age range

16 year and older

Sex eligibility

Female

Study type

Interventional

Phase

Phase 2

Primary location

University of Texas Health Science Center at San Antonio

San Antonio, Texas, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Myozyme

Biological

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

Other names: Alglucosidase alfa

Primary outcomes

  1. The objective of this extension study was to monitor the long-term safety and efficacy of a single patient

    Time frame: 3 years

Sponsors and collaborators

Lead sponsor

Genzyme, a Sanofi Company

Industry

Registry information

Official study 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

Important dates

Study start
2003
Primary completion
2006
Study completion
2006
First posted
Oct 3, 2008
Registry last updated
Feb 5, 2014

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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