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Immune Tolerance Induction Study

TerminatedPhase 4

Sponsor: Genzyme, a Sanofi Company

Conditions: Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Glycogenesis 2 Acid Maltase Deficiency

Interventions: Myozyme® (alglucosidase alfa), Myozyme® (alglucosidase alfa)

Countries: United States, Israel

An exploratory, open-labeled study of participants with Pompe disease, who had 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 participants who were then receiving Myozyme® therapy were enrolled into the study, and were 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 participants were followed for a minimum of 18 months on treatment or, if a participant was \<6 months of age at the time of enrollment, until the participant was 2 years of age. Both cross-reacting immunologic material (CRIM)-negative and CRIM-positive participants were eligible for Regimen A depending if they met the required criteria. Regimen B, however, was limited to CRIM-negative participants.

Eligibility overview

Sex: ALL

Age: 1 Month to —

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria

Inclusion Criteria:

* The participant (and/or participant's legal guardian if participant was \< 18 years) provided written informed consent prior to any study-related procedures that were performed.
* The participants had 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 participant (and/or legal guardian) had ability to comply with clinical protocol.
* If the participant was CRIM-positive, he/she had received at least 6 consecutive months of Myozyme® infusions (20 mg/kg qow).
* If the participant was CRIM-negative, he/she had received at least 1 Myozyme® infusion prior to enrollment.
* Regimen A only: The participants exhibits clinical decline; The participant had 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 participant was CRIM-negative AND The participant did not exhibit clinical decline; OR all of the following: The participant was CRIM-negative AND The participant exhibited clinical decline AND The participant did not exhibit high anti-rhGAA antibody titers and had not tested positive for antibodies that inhibit enzymatic activity and/or uptake of Myozyme®.

Exclusion Criteria:

* The participant had a clinical condition unrelated to Pompe disease that would interfere with program assessments.
* The participant was at risk of reactivation or was a carrier of Hepatitis B or Hepatitis C.
* The participant was at risk of reactivation or had positive serology suggestive of active infection for cytomegalovirus, Herpes simplex, JC virus, Parvovirus or Epstein Barr virus.
* The participant was at risk of reactivation of tuberculosis or had regular contact with individuals who were being actively treated for tuberculosis.
* The participant had low serum albumin.
* The participant had a major congenital abnormality.
* The participant had used any investigational product (other than alglucosidase alfa) within 30 days prior to study enrollment.
* The participant was pregnant or lactating.
* The participant has had or was required to have any live vaccination within one month prior to enrollment.

Locations (5)

Louisville, Kentucky, United States
Durham, North Carolina, United States
Salt Lake City, Utah, United States
Norfolk, Virginia, United States
Haifa, Israel