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An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease

Information source: Sanofi
ClinicalTrials.gov processed this data on August 23, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Pompe Disease; Glycogen Storage Disease Type II

Intervention: Alglucosidase Alfa (Biological); Methotrexate (Drug); Rituximab (Drug)

Phase: Phase 4

Status: Completed

Sponsored by: Genzyme, a Sanofi Company

Official(s) and/or principal investigator(s):
Medical Monitor, Study Director, Affiliation: Genzyme Coorporation


The purpose of this study was to evaluate the efficacy, clinical benefits and safety of a prophylactic immunomodulatory regimen given prior to first treatment with alglucosidase alfa (Myozyme®) in patients with infantile-onset Pompe disease. The objectives were to assess the efficacy of a prophylactic immunomodulatory regimen given prior to first treatment with alglucosidase alfa, as assessed by anti-recombinant human acid alpha-glucosidase (anti-rhGAA) antibody titers, and antibodies that inhibit the activity and/or uptake of alglucosidase alfa; to evaluate the clinical benefit as measured by overall survival, ventilator-free survival, left ventricular mass index (LVMI), gross motor function and development, disability index and the incidence of adverse events (AEs), serious adverse events (SAEs), and clinical laboratory abnormalities.

Clinical Details

Official title: An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme®-Naive, CRIM(-) Patients With Infantile-onset Pompe Disease

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

Primary outcome:

Change From Baseline in Number of Patients With Anti-Recombinant Human Acid Alfa-glucosidase (Anti-rhGAA) Immunoglobulin G (IgG) Antibody at End of Study

Number of Patients With Recombinant Human Acid Alfa-glucosidase (rhGAA) Inhibitory Antibody at End of Study

Number of Patients Who Survived at End of Study

Number of Patients With Normal/Abnormal Left Ventricular Mass (LVM) Z-Score and LVM Index at End of Study

Number of Patients With Ventilator Use at End of Study

Gross Motor Disability Assessed by Gross Motor Function Measure-88 (GMFM-88) at End of Study

Motor Development Status Assessed by Alberta Infantile Motor Scale (AIMS) at End of Study

Disability Index Assessed by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI) at End of Study


Minimum age: N/A. Maximum age: N/A. Gender(s): Both.


Inclusion Criteria:

- The patient's legal guardian(s) must have provided written informed consent prior to

any study-related procedures being performed

- The patient must have had a clinical diagnosis of Pompe disease as defined by

documented acid alpha-glucosidase (GAA) deficiency (deficient endogenous GAA activity) in skin fibroblasts, muscle, or blood, or 2 GAA mutations. Consent was also sought from the biological parent(s) for parental GAA mutational analysis, but was not a requirement for study eligibility

- The patient must have not received Myozyme® or any rhGAA therapies prior to

enrollment in the study

- The patient must be CRIM negative via Western Blot analysis performed on skin

fibroblasts or via 2 known CRIM negative mutations (in which case CRIM status was to be confirmed by Western Blot analysis after enrollment)

- The patient's legal guardian(s) must have the ability to comply with the clinical

protocol Exclusion Criteria:

- The patient had any medical condition that, in the opinion of the Investigator, could

be exacerbated/precipitated by or interfere with the study regimen or assessments; such conditions may include but were not limited to human immunodeficiency virus, cancer, Hepatitis B, Hepatitis C, Cytomegalovirus, Herpes Simplex, John Cunningham (JC) virus, Parvovirus, or Epstein Barr virus or tuberculosis

- The patient had used any investigational product within 30 days prior to study


- The patient had or was required to have any live vaccination within 1 month prior to


Locations and Contacts

Kosair Children's Hospital, Louisville, Kentucky 40202, United States

Duke University Medical Center, Durham, North Carolina 27710, United States

Additional Information

Starting date: October 2009
Last updated: April 9, 2014

Page last updated: August 23, 2015

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