NeoGAA Extension Study



Status:Enrolling by invitation
Conditions:Diabetes
Therapuetic Areas:Endocrinology
Healthy:No
Age Range:Any
Updated:1/18/2019
Start Date:February 27, 2014
End Date:December 16, 2021

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An Open-Label, Multicenter, Multinational Extension Study Of The Long-Term Safety And Pharmacokinetics Of Repeated Biweekly Infusions Of NeoGAA In Patients With Pompe Disease

Primary Objective:

Long-term safety and pharmacokinetics (PK) of neoGAA

Secondary Objective:

Long-term effect of neo-GAA on pharmacodynamic and exploratory efficacy variables

The duration of the study will be 6 years from the date the first patient enters the study.
Each patient will continue with the study until the patient withdraws, the Investigator
withdraws the patient, or the Sponsor terminates the study.

Inclusion criteria:

Patients with Pompe disease who previously completed a neoGAA 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.
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