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Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
NCT01288027
First received: January 27, 2011
Last updated: August 20, 2014
Last verified: August 2014

January 27, 2011
August 20, 2014
June 2011
December 2013   (final data collection date for primary outcome measure)
Percent reduction from baseline in tissue glycogen content in muscle biopsy samples. [ Time Frame: Week 26 ] [ Designated as safety issue: No ]
Same as current
Complete list of historical versions of study NCT01288027 on ClinicalTrials.gov Archive Site
  • Glycogen distribution [ Time Frame: Baseline and Week 26 ] [ Designated as safety issue: No ]
  • Muscle fiber morphology [ Time Frame: Baseline and Week 26 ] [ Designated as safety issue: No ]
  • Lysosomal inclusions [ Time Frame: Baseline and Week 26 ] [ Designated as safety issue: No ]
  • change from baseline in intact muscle and fatty replacement [ Time Frame: Week 26 ] [ Designated as safety issue: No ]
Same as current
Not Provided
Not Provided
 
Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa
A Phase 4 Prospective Exploratory Muscle Biopsy, Biomarker, and Imaging Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa

This is an open-label, multicenter study of patients with late-onset Pompe disease naïve to treatment with enzyme replacement therapy (ERT). The primary purpose of this study is to evaluate glycogen clearance in muscle tissue samples collected pre and post alglucosidase alfa treatment in patients with Late-Onset Pompe disease.

Not Provided
Interventional
Phase 4
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
  • Pompe Disease (Late-Onset)
  • Glycogen Storage Disease Type II (GSD II)
  • Glycogenesis 2 Acid Maltase Deficiency
Biological: alglucosidase alfa
Intravenous (IV) infusion of 20mg/kg every other week (qow)for 24 weeks
Experimental: alglucosidase alfa
Intravenous (IV) infusion of 20mg/kg every other week (qow) for 24 weeks.
Intervention: Biological: alglucosidase alfa
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
16
December 2013
December 2013   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • The patient has confirmed acid α-glucosidase [GAA] enzyme deficiency from any tissue source and/or confirmed GAA gene mutations and without known cardiac hypertrophy.
  • The patient is able to ambulate a distance without stopping and without an assistive device. Use of assistive device for community ambulation is appropriate.
  • The patient has a certain forced vital capacity (FVC) in upright position.
  • The patient, if female and of childbearing potential, must have a negative pregnancy test (urine beta-human chorionic gonadotropin [β-hCG]) at baseline.

Exclusion Criteria:

  • The patient has had previous treatment with enzyme replacement therapy (ERT).
  • The patient is wheelchair dependent.
  • The patient requires invasive-ventilation (non-invasive ventilation is allowed).
  • The patient is participating in another clinical study using investigational treatment.
  • The patient cannot submit to magnetic resonance imaging (MRI) examination because of a formal contraindication such as a pacemaker, implanted ferromagnetic metals, etc.
  • The patient, in the opinion of the Investigator, is unable to adhere to the requirements of the study.
Both
18 Years and older
No
Contact information is only displayed when the study is recruiting subjects
United States,   Germany,   Netherlands,   United Kingdom
 
NCT01288027
AGLU07310, 2010-020611-36
Yes
Sanofi ( Genzyme, a Sanofi Company )
Genzyme, a Sanofi Company
Not Provided
Study Director: Medical Monitor Genzyme, a Sanofi Company
Sanofi
August 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP