Avalglucosidase Alfa Extension Study (NEO-EXT)
Status and phase
Conditions
Treatments
Study type
Funder types
Identifiers
Details and patient eligibility
About
Primary Objective:
Long-term safety and pharmacokinetics (PK) of avalglucosidase alfa
Secondary Objective:
Long-term effect of avalglucosidase alfa on pharmacodynamic variables
Full description
The planned duration of the study for each participant was initially 6 years. Each participant continued with the study until the participant withdrew, the Investigator withdrew the participant, or the Sponsor terminated the study. An additional follow-up phase began after the participant has completed the 6-year study period, and lasted until avalglucosidase alfa was approved in the participant's country, except in the United Kingdom (UK), Germany and Denmark, where the duration of the additional follow-up phase was up to the approval in the country or limited to a maximum of 2 years, whichever occurred first (ie, for participants in the UK, Germany and Denmark, the total study duration per participant was 8 years at the maximum including the initial 6-year period and the additional 2-year follow-up).
Enrollment
Sex
Volunteers
Inclusion criteria
Participants with Pompe disease who previously completed an avalglucosidase study.
The participant and/or their parent/legal guardian is willing and able to provide signed informed consent, and the participant, if <18 years of age, was willing to provide assent if deemed able to do so.
The participant (and participant's legal guardian if participant is <18 years of age) must have had the ability to comply with the clinical protocol.
The participant, if female and of childbearing potential, had to have a negative pregnancy test [urine beta-human chorionic gonadotropin] at baseline.
Exclusion criteria
The participant was concurrently participating in another clinical study using investigational treatment.
The participant, in the opinion of the Investigator, was unable to adhere to the requirements of the study.
The participant had 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, precluded participation in the study or potentially decreases survival.
The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
Trial design
Primary purpose
Allocation
Interventional model
Masking
19 participants in 1 patient group
Trial contacts and locations
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Data sourced from clinicaltrials.gov
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