Please select the eligibility criteria that correspond to the clinical trial below.
Title: A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Avalglucosidase Alfa (neoGAA, GZ402666) and Alglucosidase Alfa in Treatment naïve Patients With Late-onset Pompe Disease
Brief Summary: Primary Objective:

To determine the effect of avalglucosidase alfa treatment on respiratory muscle strength measured by percent (%) predicted forced vital capacity (FVC) in the upright position, as compared to alglucosidase alfa.

Secondary Objective:

To determine the safety and effect of avalglucosidase alfa treatment on functional endurance (6-minute walk test, inspiratory muscle strength (maximum inspiratory pressure), expiratory muscle strength (maximum expiratory pressure), lower extremity muscle strength (hand-held dynamometry), motor function (Quick Motor Function Test), and health-related quality of life (Short Form-12).


Options:
A: Inclusion Criteria:

1. Diagnosis of Pompe disease by blood acid alpha-glucosidase assay and acid alpha-glucosidase gene sequencing,
2. Age: 18+ years at enrollment,
3. Receiving ERT at standard dose (20 mg/kg every 2 weeks) for at least 52 weeks,
4. Subjects are capable of giving written consent.

Exclusion Criteria:

1. Continuous invasive ventilation (via tracheostomy or endotracheal tube)
2. Clinically relevant illness within two weeks of enrollment including fever \> 38.2 C, vomiting more than once in 24 hours, seizure, or other symptom deemed contraindicative to new therapy.
3. Chronic heart disease (Myocardial infarction, arrythmia, cardiomyopathy)
4. Tachycardia
5. History of seizure disorder
6. Hyperthyroidism
7. Pheochromocytoma
8. Pregnancy
9. History of diabetes
10. History of hypersensitivity to beta 2-agonist drugs such as albuterol, levalbuterol (Xopenex), bitolterol (Tornalate), pirbuterol (Maxair), terbutaline, salmeterol (Serevent),
11. Patients on a non-standard schedule for ERT; for example, weekly infusions as opposed to infusions every two weeks.
12. Treatment for asthma in the previous 12 months.
13. The use of the following concommitant meds is prohibited during the study:

    * diuretics (water pill);
    * digoxin (digitalis, Lanoxin);
    * beta-blockers such as atenolol (Tenormin), metoprolol (Lopressor), and propranolol (Inderal);
    * tricyclic antidepressants such as amitriptyline (Elavil, Etrafon), doxepin (Sinequan), imipramine (Janimine, Tofranil), and nortriptyline (Pamelor);
    * Monoamine oxidase inhibitors such as isocarboxazid (Marplan), phenelzine (Nardil), rasagiline (Azilect), selegiline (Eldepryl, Emsam), or tranylcypromine (Parnate); or
    * other bronchodilators such as albuterol, levalbuterol (Xopenex), bitolterol (Tornalate), pirbuterol (Maxair), terbutaline (Brethine, Bricanyl), salmeterol (Serevent), isoetherine (Bronkometer), metaproterenol (Alupent, Metaprel), or isoproterenol (Isuprel Mistometer).
B: Inclusion criteria :

* The participant has confirmed acid alpha-glucosidase (GAA) enzyme deficiency from any tissue source and/or 2 confirmed GAA gene mutations.
* The participant must provide signed, informed consent prior to performing any study related procedures. Consent of a legally authorized guardian(s) is (are) required for legally minor participant as defined by local regulation. If the participant is legally minor, signed written consent shall be obtained from parent(s)/legal guardian and assent obtained from participants, if applicable.

Exclusion criteria:

* The participant is \<3 years of age.
* The participant has known Pompe specific cardiac hypertrophy.
* The participant is wheelchair dependent.
* The participant is not able to ambulate 40 meters (approximately 130 feet) without stopping and without an assistive device.
* The participant requires invasive-ventilation (non-invasive ventilation is allowed).
* The participant is not able to successfully perform repeated forced vital capacity (FVC) measurements in upright position of greater than or equal to 30% predicted and less than or equal to 85% predicted.
* The participant (and participant's legal guardian if participant is legally minor as defined by local regulation) is (are) not able to comply with the clinical protocol.
* The participant has had previous treatment with alglucosidase alfa or any investigational therapy for Pompe disease.
* The participant has prior or current use of immune tolerance induction therapy.
* The participant, if female and of childbearing potential, has a positive pregnancy test (beta-human chorionic gonadotropin) at baseline.

The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
C: Inclusion Criteria:

* Patient must provide signed, informed consent prior to performing any study-related procedures.
* Patient must have a diagnosis of Pompe disease based on deficient endogenous GAA activity in cultured skin fibroblasts of less than or equal to 40% of the normal mean of the testing laboratory and 2 confirmed GAA gene mutations;
* Patient must be greater than or equal to 8 years of age at the time of enrollment;
* Patient must be able to ambulate 40 meters (approximately 130 feet) in 6 minutes on each test performed on two consecutive days (use of assistive devices such as a walker, cane, or crutches, is permitted);
* Patient must have an FVC of greater than or equal to 30% and \< 80% predicted in the upright position;
* Patient must have a postural drop in FVC (liters) of at least 10% from the upright to the supine position;
* Patient must have proximal muscle weakness in the lower limbs based on unilateral QMT of the knee extensors defined as \< 80% of the predicted value based on age, gender and body size
* Patient must be able to tolerate pulmonary function testing (PFT) and muscle testing in the supine position;
* Patient must have testable muscle in bilateral knee flexors and knee extensors, and testable muscle in bilateral elbow flexors and elbow extensors;
* Patient must be able to provide reproducible muscle and pulmonary function test results;
* Patient (and patient's legal guardian if patient is \< 18 years of age) must have the ability to comply with the clinical protocol;
* A female patient of childbearing potential must have a negative pregnancy test (urine) at Baseline. Note: All female patients of childbearing potential and sexually mature males must use a medically accepted method of contraception throughout the study.

Exclusion Criteria:

* Patient requires the use of invasive ventilatory support;
* Patient requires the use of noninvasive ventilatory support while awake and in an upright position;
* Patient has received enzyme replacement therapy with GAA from any source;
* Patient has used an investigational product within 30 days prior to study enrollment, or is currently enrolled in another study which involves clinical evaluations, unless prior approval is given by Genzyme;
* Patient has a major congenital anomaly, medical condition, serious intercurrent illness, or other extenuating circumstance that, in the opinion of the investigator, may significantly interfere with study compliance, including all prescribed evaluations and follow-up activities;
D: Inclusion Criteria:

* Patient must have completed protocol AGLU02704 (NCT00158600)
* Patient must provide signed, informed consent prior to performing any study-related procedures
* Patient (and patient's legal guardian if patient is under 18 years of age) must have the ability to comply with the clinical protocol
* A female patient of childbearing potential must have a negative pregnancy test at Baseline. (note: all female patients of childbearing potential and sexually mature males must use a medically accepted method of contraception throughout the study.)

Exclusion Criteria:

* The patient has a medical condition, serious intercurrent illness, or other extenuating circumstance that, in the opinion of the Investigator, would preclude treatment with alglucosidase alfa.

Answer: B