Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Treatment-naive Patients With Type 1 Gaucher Disease

Protocol:

Study Design: This will be a single-center, open-label trial to assess the safety and efficacy of rapid administration of velaglucerase alfa (VPRIV) to treatment-naive patients with type I Gaucher disease. The first six infusions will be administered in the hospital: the first three infusions within 60, 30 and 20 minutes each, with the beginning of a 10 minutes administration from infusion #4. Following the three uneventful administration of the 10 minutes in the hospital, the bi-weekly ERT will continue as home therapy, as outlined in the protocol. The duration of the study will be 12 months, with an extension pending positive results.

Number of Patients: 15. The first ten patients will be adults, and then children will be allowed to enroll in the trial.

Inclusion criteria

• Males and females, 18 years or older for the first ten adult patients; 6 years or older for the last five patients.

• Confirmed enzymatic diagnosis of Gaucher disease with a defined genotype and elevated biomarker LysoGb1, performed at CentoGene using DBS methodology.
• Indications for ERT will be guided by fulfilling the MOH criteria.
• Female patients of child-bearing potential who agree to use a medically acceptable method of contraception.
• Patients who have not received ERT or SRT in the past or Patients who have not received ERT or SRT in the past 12 months and have a negative anti-glucocerebrosidase antibody

Exclusion criteria

• Currently taking another experimental drug for any condition

• Presence of neurologic signs and symptoms characteristic of Type 2 or Type 3 Gaucher disease
• Pregnant or nursing
• Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the patient's compliance with the study.