Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease
Condition: Gaucher Disease, Type 1
MeSH terms: Gaucher Disease
Intervention: velaglucerase alfa; imiglucerase
Intervention description: IV infusion, 60 U/kg every other week for 9 months; IV infusion, 60 U/kg every other week for 9 months
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 34.0
Sex: All
Minimum age: 2 Years
Healthy volunteers: No
Keywords: Enzyme Replacement Therapy; Gaucher disease; glucocerebrosidase; beta-glucocerebrosidase; Acid beta-glucocerebrosidase; glucosylceramidase; D-glucosyl-N-acylsphingosine glucohydrolase; gene activation; human

Summary:

      Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme
      glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside
      accumulates within macrophages leading to cellular engorgement, organomegaly, and organ
      system dysfunction. The purpose of this non-inferiority study is to evaluate the efficacy and
      safety of GA-GCB (velaglucerase alfa) administered every other week in comparison to
      imiglucerase in treatment naive patients with type 1 Gaucher disease.
    

Eligibility criteria:

        Inclusion Criteria

        Includes:

          -  The patient has a documented diagnosis and clinical manifestation of type 1 Gaucher
             disease

          -  The patient is at least 2 years of age.

          -  The patient has not received treatment for Gaucher disease (investigational products,
             miglustat, or imiglucerase) within 12 months prior to study entry, as documented in
             the patient's medical history.

          -  Female patients of child-bearing potential must agree to use a medically acceptable
             method of contraception at all times during the study and must have negative results
             to a pregnancy test performed at the time of enrollment and as required throughout
             their participation in the study. Male patients must use a medically acceptable method
             of birth control throughout their participation in the study and must report their
             partner's pregnancy.

          -  The patient, the patient's parent(s) or legal guardian(s) has provided written
             informed consent that has been approved by the Institutional Review Board/Independent
             Ethics Committee (IRB/IEC).

          -  The patient must be sufficiently cooperative to participate in this clinical study as
             judged by the Investigator.

        Exclusion Criteria

        Includes:

          -  The patient has type 2 or 3 Gaucher disease or is suspected of having type 3 Gaucher
             disease.

          -  The patient has received treatment with any non-Gaucher disease-related
             investigational drug or device within the 30 days prior to study entry; such use
             during the study is not permitted.

          -  The patient is known to be positive for human immunodeficiency virus (HIV).

          -  The patient is known to be positive for hepatitis B and/or C.

          -  The patient, patient's parent(s), or patient's legal guardian(s) is/are unable to
             understand the nature, scope, and possible consequences of the study.

          -  The patient has a significant comorbidity(ies) that might affect study data or
             confound the study results (e.g., malignancies, primary biliary cirrhosis, autoimmune
             liver disease, etc.).

          -  The patient is unable to comply with the protocol, e.g., has a clinically relevant
             medical condition making implementation of the protocol difficult, has an
             uncooperative attitude, is unable to return for safety evaluations, or is otherwise
             unlikely to complete the study, as determined by the Investigator.