Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial

NCT00705939 | Completed Phase 3 Results DMC

• 1 other identifier: PB-06-003
• Study Type: interventional
• Target: 45
• Locations: 8 countries
• Sites: 11

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.

Conditions

Gaucher Disease

Keywords

Gaucher Disease; Enzyme replacement therapy

Outcome Measures

Primary Outcomes (1)

• Spleen Volume

  Spleen volume measured by MRI

  Spleen Volume at Baseline and Months 12, 24, and 36

Secondary Outcomes (3)

• Liver Volume

  Liver volume at Baseline and Months 12, 24 and 36

• Hemoglobin

  Hemoglobin at Baseline and Months 12, 24 and 36

• Platelet Count

  Platelet count at Baseline and Months 12, 24 and 36

Other Outcomes (2)

• Spleen Volume Multiples of Normal (MN)

  Baseline and Months 12, 24, and 36

• Liver Volume Multiples of Normal (MN)

  Baseline and Months 12, 24 and 36

Study Arms (3)

Naive 30 Units/kg

EXPERIMENTAL

Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168)

Drug: Taliglucerase alfa

Naive 60 Units/kg

EXPERIMENTAL

Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168)

Drug: Taliglucerase alfa

Switchover

EXPERIMENTAL

Continue taliglucerase alfa treatment from PB-06-002 (NCT00712348)

Drug: Taliglucerase alfa

Interventions

Taliglucerase alfa DRUG

Intravenous infusion every 2 weeks

Also known as: Plant Cell Expressed Recombinant Human Glucocerebrosidase, prGCD

Naive 30 Units/kg; Naive 60 Units/kg; Switchover

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• Pfizer: lead

Study Sites (11)

Department of Human Genetics, Emory University School of Medicine

Decatur, Georgia, 30033, United States

Location

Neurogenetics, NYU at Rivergate

New York, New York, 10016, United States

Location

Bone Marrow Transplant Service, The Royal Melbourne Hospital

Parkville, Victoria, Australia

Location

Mount Sinai Hospital

Toronto, Ontario, M5G 1X5, Canada

Location

Pontificia Universidad Catolica de Chile

Santiago, Chile

Location

Rambam Medical Center

Haifa, 31096, Israel

Location

Shaare Zedek Medical Center

Jerusalem, Israel

Location

Morningside Medi-Clinic

Morningside, 2196, South Africa

Location

Hospital Universitario Miguel Servet

Zaragoza, 50009, Spain

Location

Lysosomal Disorders Service, Addenbrookes Hospital NHS Trust

Cambridge, United Kingdom

Location

Royal Free Hospital

London, NW3 2QG, United Kingdom

Location

Related Publications (2)

• 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.

  PMID: 27174694 DERIVED

• 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.

  PMID: 27102949 DERIVED

MeSH Terms

Conditions

Gaucher Disease

Interventions

taliglucerase alfa

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Results Point of Contact

• Title: Vice President Product Development
• Organization: Protalix Ltd.

einata@protalix.com

+(972) 4-9889488

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Study Record Dates

• First Submitted: June 25, 2008
• First Posted: June 27, 2008
• Study Start: June 1, 2008
• Primary Completion: May 1, 2012
• Study Completion: August 1, 2013
• Last Updated: October 4, 2018
• Results First Posted: July 15, 2014

Record last verified: 2018-09

Locations

ZA (1); CL (1); CA (1); US (2); ES (1); GB (2); IL (2); AU (1)