A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease
Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Genzyme, a Sanofi Company
Age
0–1
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
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.
You may not be if
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.
Clareo Health | A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease
