A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease
| Status: | Terminated |
|---|---|
| Conditions: | Diabetes |
| Therapuetic Areas: | Endocrinology |
| Healthy: | No |
| Age Range: | Any |
| Updated: | 4/21/2016 |
| Start Date: | August 2012 |
| End Date: | December 2014 |
A Phase 3/4, Prospective, Multinational, Open-label, Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease
A study to demonstrate comparable safety, efficacy, and pharmacokinetics (PK) of
alglucosidase alfa manufactured at the 160 litre (L) and 4000 L scales in participants who
had been diagnosed with infantile-onset Pompe disease. Participants were treated with
alglucosidase alfa 160 L scale product in the United States (US) and 4000 L scale product in
the regions outside the US.
alglucosidase alfa manufactured at the 160 litre (L) and 4000 L scales in participants who
had been diagnosed with infantile-onset Pompe disease. Participants were treated with
alglucosidase alfa 160 L scale product in the United States (US) and 4000 L scale product in
the regions outside the US.
Inclusion Criteria:
- The participant's parent/legal guardian was willing and able to provide signed
informed consent.
- The participant might be less than or equal to 12 months of age.
- The participant might have documented GAA enzyme deficiency from blood, skin, or
muscle tissue.
- The participant might be naïve to treatment with alglucosidase alfa.
Exclusion Criteria:
- The participant was cross-reactive immunologic material negative.
- The participant required invasive ventilator support at the time of enrollment.
- The participant had decompensated clinical heart failure.
- The participant had a major congenital abnormality, excluding cardiac hypertrophy.
- The participant had a clinically significant organ disease (excluding the signs and
symptoms of Pompe disease).
- The participant was currently receiving any investigational product.
- The participant was participating in another clinical study.
- The participant and/or the patient's parent/legal guardian was unable to adhere to
the requirements of the study.
We found this trial at
15
sites
Click here to add this to my saved trials
Click here to add this to my saved trials
Click here to add this to my saved trials
Click here to add this to my saved trials
Click here to add this to my saved trials
Click here to add this to my saved trials
Click here to add this to my saved trials
Click here to add this to my saved trials
Click here to add this to my saved trials
Click here to add this to my saved trials
Click here to add this to my saved trials
Click here to add this to my saved trials
Click here to add this to my saved trials
Click here to add this to my saved trials
Click here to add this to my saved trials