Status:
TERMINATED
Immune Tolerance Induction Study
Lead Sponsor:
Genzyme, a Sanofi Company
Conditions:
Pompe Disease
Glycogen Storage Disease Type II (GSD-II)
Eligibility:
All Genders
1+ years
Phase:
PHASE4
Brief Summary
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 I...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
December 14 2008
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
February 18 2020
Estimated Enrollment :
4 Patients enrolled
Trial Details
Trial ID
NCT00701701
Start Date
December 14 2008
End Date
February 18 2020
Last Update
April 7 2022
Active Locations (5)
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1
Louisville, Kentucky, United States
2
Durham, North Carolina, United States
3
Salt Lake City, Utah, United States
4
Norfolk, Virginia, United States