Avalglucosidase Alfa Extension Study (NEO-EXT)
Glycogen Storage Disease Type II Pompe Disease
About this trial
This is an interventional treatment trial for Glycogen Storage Disease Type II Pompe Disease
Eligibility Criteria
Inclusion criteria:
Patients with Pompe disease who previously completed a an avalglucosidase study.
The patient and/or their parent/legal guardian is willing and able to provide signed informed consent, and the patient, if <18 years of age, is willing to provide assent if deemed able to do so.
The patient (and patient's legal guardian if patient is <18 years of age) must have the ability to comply with the clinical protocol.
The patient, if female and of childbearing potential, must have a negative pregnancy test [urine beta-human chorionic gonadotropin] at baseline.
Exclusion criteria:
The patient is concurrently participating in another clinical study using investigational treatment.
The patient, in the opinion of the Investigator, is unable to adhere to the requirements of the study.
The patient has clinically significant organic disease (with the exception of symptoms relating to Pompe disease), including clinically significant cardiovascular, hepatic, pulmonary, neurologic, or renal disease, or other medical condition, serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, precludes participation in the study or potentially decreases survival.
The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Sites / Locations
- Investigational Site Number 840006
- Investigational Site Number 840010
- Investigational Site Number 840001
- Investigational Site Number 840008
- Investigational Site Number 840002
- Investigational Site Number 840011
- Investigational Site Number 840009
- Investigational Site Number 840003
- Investigational Site Number 056001
- Investigational Site Number 208001
- Investigational Site Number 250003
- Investigational Site Number 250002
- Investigational Site Number 276003
- Investigational Site Number 276001
- Investigational Site Number 276002
- Investigational Site Number 528001
- Investigational Site Number 826003
Arms of the Study
Arm 1
Experimental
Avalglucosidase alfa
administered intravenously every 2 weeks