NCT00125879

Brief 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 overall objective of this study is to evaluate the long-term safety and efficacy of Myozyme treatment in patients with infantile-onset Pompe disease.

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
16

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Jun 2005

Shorter than P25 for phase_2

Geographic Reach
7 countries

14 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 1, 2005

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

August 1, 2005

Completed
1 day until next milestone

First Posted

Study publicly available on registry

August 2, 2005

Completed
10 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2006

Completed
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2006

Completed
Last Updated

February 6, 2014

Status Verified

February 1, 2014

Enrollment Period

1 year

First QC Date

August 1, 2005

Last Update Submit

February 4, 2014

Conditions

Glycogen Storage Disease Type II

Keywords

Glycogen Storage Disease Type IIGSD-IIPompe DiseasePompe Disease (Late-onset)Acid Maltase Deficiency DiseaseGlycogenosis 2Glycogen Storage Disease Type II (GSD-II)

Outcome Measures

Primary Outcomes (1)

  • Long-term Safety and Efficacy

    52 weeks

Study Arms (1)

1

EXPERIMENTAL
Biological: Myozyme

Interventions

MyozymeBIOLOGICAL

20 mg/kg qow or 40 mg/kg qow

Also known as: alglucosidase alfa
1

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • The patient's legal guardian(s) must provide written informed consent prior to any study-related procedures being performed
  • The patient and his/her legal guardian(s) must have the ability to comply with the clinical protocol
  • The patient must have completed Protocol AGLU01602.

You may not qualify if:

  • Patient has experienced any unmanageable adverse event (AE) in Protocol AGLU01602 due to Myozyme that would preclude continuing treatment with Myozyme

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (14)

University of Alabama

Birmingham, Alabama, 35233, United States

Location

Shands Hospital at the University of Florida

Gainesville, Florida, 32610, United States

Location

Miami Children's Hospital

Miami, Florida, 33155, United States

Location

Emory University Medical Genetics

Decatur, Georgia, 30033, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

CHU Amiens

Amiens, 80054, France

Location

CHU Cote de Nacre

Caen, 14033, France

Location

Universitats-Kinderklinik Mainz

Mainz, 55131, Germany

Location

Rambam Medical Center

Haifa, 35254, Israel

Location

San Gerardo Hospital

Monza, 20052, Italy

Location

Erasmus MC University

Rotterdam, 3015 GJ, Netherlands

Location

Tzu-Chi General Hospital

Hualien City, 970, Taiwan

Location

Chi-Mei Medical Center Dept of Pediatrics

Tainan, 710, Taiwan

Location

Related Publications (1)

  • Kishnani PS, Goldenberg PC, DeArmey SL, Heller J, Benjamin D, Young S, Bali D, Smith SA, Li JS, Mandel H, Koeberl D, Rosenberg A, Chen YT. Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants. Mol Genet Metab. 2010 Jan;99(1):26-33. doi: 10.1016/j.ymgme.2009.08.003.

    PMID: 19775921DERIVED

MeSH Terms

Conditions

Glycogen Storage Disease Type II

Interventions

GAA protein, human

Condition Hierarchy (Ancestors)

Lysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGlycogen Storage DiseaseCarbohydrate Metabolism, Inborn ErrorsLysosomal Storage DiseasesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Medical Monitor

    Genzyme, a Sanofi Company

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY

Study Record Dates

First Submitted

August 1, 2005

First Posted

August 2, 2005

Study Start

June 1, 2005

Primary Completion

June 1, 2006

Study Completion

December 1, 2006

Last Updated

February 6, 2014

Record last verified: 2014-02

Locations

DE(1)IL(1)FR(2)NL(1)IT(1)TW(2)US(6)