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

NCT ID: NCT01132690

Last Updated: 2018-10-05

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE4
• Total Enrollment: 11 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2010-08-31
• Study Completion Date: 2012-07-31

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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

30 units/kg

Group Type: EXPERIMENTAL

Taliglucerase alfa

Intervention Type: DRUG

Taliglucerase alfa for infusion every two weeks for 12 months

60 units/kg

Group Type: EXPERIMENTAL

Taliglucerase alfa

Intervention Type: DRUG

Taliglucerase alfa for infusion every two weeks for 12 months

Interventions

Taliglucerase alfa

Taliglucerase alfa for infusion every two weeks for 12 months

Intervention Type: DRUG

Other Intervention Names

prGCD, plant cell expressed glucocerebrosidase

Eligibility Criteria

Inclusion Criteria

• 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).

Exclusion Criteria

• 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.

• Minimum Eligible Age: 2 Years
• Maximum Eligible Age: 17 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Pfizer

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Shaare Zedek Medical Center

Jerusalem, , Israel

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

Barrio Sajonia Asunción, , Paraguay

Morningside Medi-Clinic

Morningside, , South Africa

Countries

Israel; Paraguay; South Africa

References

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.

Reference Type: DERIVED

PMID: 25453586 (View on PubMed)

Other Identifiers

PB-06-005

Identifier Type: -

Identifier Source: org_study_id