Salt Lake City, Utah


Purpose:

An exploratory, open-labeled study of patients with Pompe disease, who have previously received Myozyme (alglucosidase alfa) treatment, to evaluate the efficacy, safety and clinical benefit of 2 Immune Tolerance Induction (ITI) regimens in combination with Myozyme. Eligible patients who are currently receiving Myozyme therapy will be enrolled into the study, and will be followed for a minimum of 18 months on-study (a 6-month ITI treatment module and a 12-month follow-up module on Myozyme alone). Eligible patients will be followed for a minimum of 18 months on treatment or, if a patient is <6 months of age at the time of enrollment, until the patient is 2 years of age. Both cross-reacting immunologic material (CRIM)-negative and CRIM-positive patients can be eligible for Regimen A depending if they meet the required criteria. Regimen B however, is limited to CRIM-negative patients.


Criteria:

Inclusion Criteria: - The patient (and/or patient's legal guardian if patient is < 18years) must provide written informed consent prior to any study-related procedures that are performed; - The patient must have a confirmed diagnosis of Pompe disease defined as a documented acid α-glucosidase (GAA) enzyme deficiency from any tissue source or 2 GAA gene mutations; - The patient (and/or legal guardian) must have ability to comply with clinical protocol; - If the patient is Cross-reacting immunologic material (CRIM)-positive, he/she must have received at least 6 consecutive months of Myozyme infusions (20mg/kg qow) - If the patient is CRIM-negative, he/she must have received at least 1 Myozyme infusion prior to enrollment - Regimen A only: The patient exhibits clinical decline; The patient has persistent high anti-recombinant human acid α-glucosidase (anti-rhGAA) antibody titers and/or tested positive for antibodies that inhibit enzymatic activity and/or uptake of Myozyme; - Regimen B only: The patient is CRIM-negative AND The patient does not exhibit clinical decline; OR ALL OF THE FOLLOWING: The patient is CRIM-negative AND The patient exhibits clinical decline AND The patient does NOT exhibit high anti-rhGAA antibody titers and has NOT tested positive for antibodies that inhibit enzymatic activity and/or uptake of Myozyme. Exclusion Criteria: - The patient has a clinical condition unrelated to Pompe disease that would interfere with program assessments; - The patient is at risk of reactivation or is a carrier of Hepatitis B or Hepatitis C; - The patient is at risk of reactivation or has positive serology suggestive of active infection for cytomegalovirus, Herpes simplex, JC virus, Parvovirus or Epstein Barr virus; - The patient is at risk of reactivation of tuberculosis or has regular contact with individuals who are being actively treated for tuberculosis; - The patient has low serum albumin; - The patient has a major congenital abnormality; - The patient has used any investigational product (other than alglucosidase alfa) within 30 days prior to study enrollment; - The patient is pregnant or lactating; - The patient has had or is required to have any live vaccination within one month prior to enrollment.


NCT ID:

NCT00701701


Primary Contact:

Study Director
Medical Monitor
Genzyme, a Sanofi Company

Medical Information
Phone: 800-745-4447
Email: medinfo@genzyme.com


Backup Contact:

Email: MedInfo@genzyme.com
Medical Information
Phone: 617-252-7832


Location Contact:

Salt Lake City, Utah
United States



There is no listed contact information for this specific location.

Site Status: Recruiting


Data Source: ClinicalTrials.gov

Date Processed: January 18, 2018

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