Please select the eligibility criteria that correspond to the clinical trial below.
Title: A Multicenter, Double-blind, Randomized Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
Brief Summary: This is a multi-center, double-blind trial to assess the safety and efficacy of taliglucerase alfa in untreated subjects (2 to \<18 years old) with Gaucher disease randomly assigned to treatment with one of two doses, 30 or 60 units/kg. Subjects will receive an intravenous (IV) infusion of taliglucerase alfa every two weeks. The total duration of treatment will be 12 months. At the end of the 12-month treatment period eligible subjects will be offered enrollment in an open-label extension study if taliglucerase alfa is not commercially available.


Options:
A: Inclusion Criteria:

* Males and females, 18 years or older
* Confirmed enzymatic diagnosis of Gaucher disease
* Splenomegaly defined as greater than eight times the expected volume (measured volume divided by estimated volume (0.2% of body weight)\] as determined by MRI volumetric analysis
* Female patients of child-bearing potential who agree to use a medically acceptable method of contraception
* Thrombocytopenia (defined as platelet counts below the lower limit of normal) and/or anemia (defined by hemoglobin level at least 1 g/dL below normal range according to sex and age).
* Patients who have not received ERT in the past or patients whoc have not received ERT in the past 12 months and have a negative anti-glucocerebrosidase antibody test.
* Patients who have not received substrate reduction therapy (SRT) in the past 12 months.
* Ability to provide a written informed consent.

Exclusion Criteria:

* Currently taking another experimental drug for any condition
* Pregnant or nursing
* Presence of HIV and/or, HBsAg and/or hepatitis C infections
* Presence of severe neurological signs and symptoms, defined as complete ocular paralysis, overt myoclonus or history of seizures, characteristic of neuronopathic Gaucher disease.
* Previous anaphylactoid reaction to Cerezyme® or Ceredase®.
* History of allergy to carrots.
B: Inclusion Criteria:

* Males and females 2 to \<18 years old.
* Diagnosis of Gaucher disease with leukocyte acid β-glucosidase activity ≤30% of the mean of the reference range for healthy subjects.
* Subjects who have not received enzyme replacement therapy (ERT) in the past or who have not received ERT in the past 12 months and have a negative anti-glucocerebrosidase antibody assay.
* Subjects who have not received substrate reduction therapy (SRT) in the past 12 months.
* Subjects whose clinical condition, in the opinion of the investigator, requires treatment with enzyme replacement therapy (ERT).

Exclusion Criteria:

* Currently taking another investigational drug for any condition.
* Presence of neurological signs and symptoms characteristic of Gaucher disease with complex neuronopathic features other than longstanding oculomotor gaze palsy.
* Presence of unresolved anemia due to iron, folic acid, or vitamin B12 deficiency
* Previous hypersensitivity reaction to Cerezyme® (imiglucerase) or Ceredase® (alglucerase).
* History of allergy to carrots.
* Presence of HIV, HBsAg or hepatitis C infections.
* Subject's parent(s) or legal guardian(s) are unable to understand the nature, scope and possible consequences of the study.
* Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the subject's compliance with the requirements of the study.
C: Inclusion Criteria:

* The participant (and/or their parent/legal guardian) was willing and able to provide signed informed consent prior to any study-related procedures to be performed
* The participant was at least 18 years old at the time of randomization
* The participant had a confirmed diagnosis of Gaucher disease type 1
* The participant had received treatment with ERT for at least 3 years. Within the 9 months prior to randomization, the participant had received a total monthly dose of 30 to 130 Units/kilogram for at least 6 months
* The participant had reached Gaucher disease therapeutic goals prior to randomization
* Female participants of childbearing potential must have had a documented negative pregnancy test prior to dosing. In addition, all female participants of childbearing potential must use a medically accepted form of contraception throughout the study

Exclusion Criteria:

* The participant had a partial or total splenectomy within 3 years prior to randomization
* The participant had received substrate reduction therapies for Gaucher disease within 6 months prior to randomization
* The participant had Gaucher disease type 2 or 3 or was suspected of having Gaucher disease type 3
* The participant had any clinically significant disease, other than Gaucher disease, including cardiovascular, renal, hepatic, gastrointestinal (GI), pulmonary, neurologic, endocrine, metabolic (e.g. hypokalemia, hypomagnesemia), or psychiatric disease, other medical conditions, or serious intercurrent illnesses that may confound the study results or, in the opinion of the Investigator, may preclude participation in the study
* The participant had tested positive for the human immunodeficiency virus (HIV) antibody, Hepatitis C antibody, or Hepatitis B surface antigen
* The participant had received an investigational product within 30 days prior to randomization
* The participant was pregnant or lactating
D: Inclusion Criteria:

* Successful completion of Protocol PB-06-002 or PB-06-005
* The subject, parent(s) or legal guardian(s) signs an informed consent and/or assent

Exclusion Criteria:

* Currently taking another investigational drug for any condition.
* Presence of neurological signs and symptoms characteristic of Gaucher disease with complex neuronopathic features other than longstanding oculomotor gaze palsy.
* Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the subject's compliance with the requirements of the study.

Answer: B