A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease

NCT01132690 | Completed Phase 4 Results DMC

• 1 other identifier: PB-06-005
• Study Type: interventional
• Target: 11
• Locations: 3 countries
• Sites: 3

Brief Summary

This is a multi-center, double-blind trial to assess the safety and efficacy of taliglucerase alfa in untreated subjects (2 to \<18 years old) with Gaucher disease randomly assigned to treatment with one of two doses, 30 or 60 units/kg. Subjects will receive an intravenous (IV) infusion of taliglucerase alfa every two weeks. The total duration of treatment will be 12 months. At the end of the 12-month treatment period eligible subjects will be offered enrollment in an open-label extension study if taliglucerase alfa is not commercially available.

Conditions

Gaucher Disease

Keywords

gaucher disease; pediatric

Outcome Measures

Primary Outcomes (1)

• Hemoglobin

  median and interquartile range for change from baseline in haemoglobin

  Every 3 months for 12 months

Secondary Outcomes (5)

• Chitotriosidase

  Every 3 months for 12 months

• Spleen Volume

  Baseline and Month 12

• Platelet Count

  Baseline and 12 months

• Chemokine (C-C Motif) Ligand 18 (CCL18)

  Every 3 months for 12 months

• Liver Volume

  Baseline and Month 12

Study Arms (2)

30 units/kg

EXPERIMENTAL

Drug: Taliglucerase alfa

60 units/kg

EXPERIMENTAL

Drug: Taliglucerase alfa

Interventions

Taliglucerase alfa DRUG

Taliglucerase alfa for infusion every two weeks for 12 months

Also known as: prGCD, plant cell expressed glucocerebrosidase

30 units/kg; 60 units/kg

Eligibility Criteria

• Age: 2 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Males and females 2 to \<18 years old.
• Diagnosis of Gaucher disease with leukocyte acid β-glucosidase activity ≤30% of the mean of the reference range for healthy subjects.
• Subjects who have not received enzyme replacement therapy (ERT) in the past or who have not received ERT in the past 12 months and have a negative anti-glucocerebrosidase antibody assay.
• Subjects who have not received substrate reduction therapy (SRT) in the past 12 months.
• Subjects whose clinical condition, in the opinion of the investigator, requires treatment with enzyme replacement therapy (ERT).

You may not qualify if:

• Currently taking another investigational drug for any condition.
• Presence of neurological signs and symptoms characteristic of Gaucher disease with complex neuronopathic features other than longstanding oculomotor gaze palsy.
• Presence of unresolved anemia due to iron, folic acid, or vitamin B12 deficiency
• Previous hypersensitivity reaction to Cerezyme® (imiglucerase) or Ceredase® (alglucerase).
• History of allergy to carrots.
• Presence of HIV, HBsAg or hepatitis C infections.
• Subject's parent(s) or legal guardian(s) are unable to understand the nature, scope and possible consequences of the study.
• Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the subject's compliance with the requirements of the study.

Sponsors & Collaborators

• Pfizer: lead

Study Sites (3)

Shaare Zedek Medical Center

Jerusalem, Israel

Location

Instituto Privado de Hematologia E Investigacion Clinica (I.P.H.I.C)

Barrio Sajonia Asunción, Paraguay

Location

Morningside Medi-Clinic

Morningside, 2196, South Africa

Location

Related Publications (1)

• Zimran A, Gonzalez-Rodriguez DE, Abrahamov A, Elstein D, Paz A, Brill-Almon E, Chertkoff R. Safety and efficacy of two dose levels of taliglucerase alfa in pediatric patients with Gaucher disease. Blood Cells Mol Dis. 2015 Jan;54(1):9-16. doi: 10.1016/j.bcmd.2014.10.002. Epub 2014 Nov 7.

  PMID: 25453586 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: Senior VP Development
• Organization: Protalix Biotherapeutics

einat@protalix.com

+972-4-902-8100

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Study Record Dates

• First Submitted: May 26, 2010
• First Posted: May 28, 2010
• Study Start: August 1, 2010
• Primary Completion: May 1, 2012
• Study Completion: July 1, 2012
• Last Updated: October 5, 2018
• Results First Posted: November 11, 2014

Record last verified: 2018-09

Locations

PA (1); IL (1); ZA (1)