Phase 4 N=95 Randomized Treatment

Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease

Gaucher Disease, Type 1 · Cerebroside Lipidosis Syndrome · Glucocerebrosidase Deficiency Disease · Glucosylceramide Beta-Glucosidase Deficiency Disease · Gaucher Disease, Non-Neuronopathic Form

Bottom Line

View on ClinicalTrials.gov: NCT00364858 ↗

Enrolled (actual)

Serious AEs

11.6%

Results posted

Sep 2009

Primary outcome: Primary: Number of Participants With Clinical Success at Month 24/Discontinuation — 21; 36; 0.808; 0.632 patients

Study Design & Population

Study type: Interventional
Phase: Phase 4
Interventions: Cerezyme (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Genzyme, a Sanofi Company
Primary completion: —

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants With Clinical Success at Month 24/Discontinuation	21; 36; 0.808; 0.632	—
SECONDARY Mean Composite Scores of the SF-36 Health Survey at Baseline	49.8; 46.9; 52.9; 53.0	—
SECONDARY Mean Composite Scores of the SF-36 Health Survey at Month 24/Discontinuation.	49.1; 46.2; 53.8; 52.6	—
SECONDARY Mean Change From Baseline in Composite Scores of the SF-36 Health Survey at Month 24/Discontinuation	-1.5; -0.5; 0.6; -0.5	—

Summary

This is a multicenter, randomized trial to compare the safety and efficacy of two dosing frequencies of Cerezyme® in patients with Gaucher disease who are currently being treated with Cerezyme®. Approximately 90 patients will be randomized in a 2:1 (q4 : q2) ratio to one of two treatment arms at up to 26 study centers worldwide. Patients will continue to receive the same total 4-week dose that they were receiving prior to study enrollment, however, they will be randomized to receive either their total 4-week dose in two infusions, one infusion every 2 weeks or their total 4-week dose in one infusion every 4 weeks. The randomization scheme will ensure a 2:1 balance between the every 4-week versus every 2-week infusion groups, respectively.

Eligibility Criteria

Inclusion Criteria

The patient must provide written informed consent prior to undergoing any study-related procedures.
The patient has a confirmed diagnosis of Gaucher disease with a documented deficiency of glucocerebrosidase by enzyme assay
The patient has been genotyped or will have genotyping performed within 3 months of study enrollment.
The patient has been treated with Cerezyme for at least 2 years prior to study enrollment.
The patient has been on a stable dose of between 20-60 U/kg every 2 weeks for at least 6 months prior to study enrollment.
The patient is at least 18 years old.
The patient has a hemoglobin value of ≥ 11.0 g/dL for women and ≥ 12.0 g/dL for men and a platelet count of ≥ 100,000 mm^3.
The patient's liver volume is ≤ 1.8 x normal confirmed by MRI or CT within 6 months of randomization.
The patient's spleen volume is ≤ 10 x normal confirmed by MRI or CT within 6 months of randomization.
The patient has a serum creatinine 3 days) within 12 months of randomization.
Patient has received an investigational drug within 30 days of the start of their participation in this trial. Patients may not receive any other investigational product throughout the course of the study.
The patient has a clinically significant disease (with the exception of symptoms relating to Gaucher disease), including clinically significant cardiovascular, hepatic, immunologic, pulmonary, neurologic, or renal disease, or other medical condition, serious intercurrent illness, or extenuating circumstances that, in the opinion of the Investigator, would preclude participation in the trial or potentially decrease survival
Patient has a medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the patient's compliance with the requirements of the study.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00364858). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.