Immune Tolerance Induction Study
| Status: | Active, not recruiting |
|---|---|
| Conditions: | Diabetes |
| Therapuetic Areas: | Endocrinology |
| Healthy: | No |
| Age Range: | Any |
| Updated: | 4/17/2018 |
| Start Date: | December 14, 2008 |
| End Date: | January 2020 |
An Exploratory Study of the Safety and Efficacy of Immune Tolerance Induction (ITI) in Patients With Pompe Disease Who Have Previously Received Myozyme
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.
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.
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.
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