Clinical trial NCT00705939: Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial
Official title: A Multicenter, Double-Blind, Extension Trial of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients With Gaucher Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Gaucher Disease
Keywords: Gaucher Disease
Enzyme replacement therapy
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2008-06-27
Enrollment: 45
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD) leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the accumulation of substrate glucocerebroside (GlcCer) in the cells of the monocyte-macrophage system.

This is an extension trial to Study NCT00376168 and NCT00712348.

Detailed description: This will be a multi-center, double-blind, parallel group, extension trial to assess the safety and efficacy of prGCD in patients completing NCT00376168. Patients will receive IV infusion of prGCD every two weeks at the selected medical center. The duration of the extension study will be fifteen months. There will be two treatment groups: 30 units/kg every 2 weeks or 60 units/kg every 2 weeks.

Eligibility criteria: Inclusion Criteria:

* Successful completion of Protocol PB-06-001
* The patient signs informed consent

Exclusion Criteria:

* Currently taking another experimental drug for any condition
* Presence of severe neurological signs and symptoms, defined as complete ocular paralysis, overt myoclonus or history of seizures, characteristic of neuronopathic 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 requirements of the study

Primary outcomes:
measure: Spleen Volume
timeFrame: Spleen Volume at Baseline and Months 12, 24, and 36
Secondary outcomes:
measure: Liver Volume
timeFrame: Liver volume at Baseline and Months 12, 24 and 36
measure: Hemoglobin
timeFrame: Hemoglobin at Baseline and Months 12, 24 and 36
measure: Platelet Count
timeFrame: Platelet count at Baseline and Months 12, 24 and 36
Other outcomes:
measure: Spleen Volume Multiples of Normal (MN)
timeFrame: Baseline and Months 12, 24, and 36
measure: Liver Volume Multiples of Normal (MN)
timeFrame: Baseline and Months 12, 24 and 36
Locations:
city: Decatur
country: United States
facility: Department of Human Genetics, Emory University School of Medicine
state: Georgia
city: New York
country: United States
facility: Neurogenetics, NYU at Rivergate
state: New York
city: Parkville
country: Australia
facility: Bone Marrow Transplant Service, The Royal Melbourne Hospital
state: Victoria
city: Toronto
country: Canada
facility: Mount Sinai Hospital
state: Ontario
city: Santiago
country: Chile
facility: Pontificia Universidad Catolica de Chile
city: Haifa
country: Israel
facility: Rambam Medical Center
city: Jerusalem
country: Israel
facility: Shaare Zedek Medical Center
city: Morningside
country: South Africa
facility: Morningside Medi-Clinic
city: Zaragoza
country: Spain
facility: Hospital Universitario Miguel Servet
city: Cambridge
country: United Kingdom
facility: Lysosomal Disorders Service, Addenbrookes Hospital NHS Trust
city: London
country: United Kingdom
facility: Royal Free Hospital
Study officials:
References:
citation: Zimran A, Duran G, Mehta A, Giraldo P, Rosenbaum H, Giona F, Amato DJ, Petakov M, Munoz ET, Solorio-Meza SE, Cooper PA, Varughese S, Chertkoff R, Brill-Almon E. Long-term efficacy and safety results of taliglucerase alfa up to 36 months in adult treatment-naive patients with Gaucher disease. Am J Hematol. 2016 Jul;91(7):656-60. doi: 10.1002/ajh.24369. Epub 2016 Apr 24.
citation: Pastores GM, Shankar SP, Petakov M, Giraldo P, Rosenbaum H, Amato DJ, Szer J, Chertkoff R, Brill-Almon E, Zimran A. Enzyme replacement therapy with taliglucerase alfa: 36-month safety and efficacy results in adult patients with Gaucher disease previously treated with imiglucerase. Am J Hematol. 2016 Jul;91(7):661-5. doi: 10.1002/ajh.24399. Epub 2016 May 18.