Clinical trial NCT01132690: A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
Official title: A Multicenter, Double-blind, Randomized Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Gaucher Disease
Keywords: gaucher disease
pediatric
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2010-05-28
Enrollment: 11
Sex: ALL
Minimum age (years): 2
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


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.

Primary outcomes:
measure: Hemoglobin
timeFrame: Every 3 months for 12 months
Secondary outcomes:
measure: Chitotriosidase
timeFrame: Every 3 months for 12 months
measure: Spleen Volume
timeFrame: Baseline and Month 12
measure: Platelet Count
timeFrame: Baseline and 12 months
measure: Chemokine (C-C Motif) Ligand 18 (CCL18)
timeFrame: Every 3 months for 12 months
measure: Liver Volume
timeFrame: Baseline and Month 12
Other outcomes:
Locations:
city: Jerusalem
country: Israel
facility: Shaare Zedek Medical Center
city: Barrio Sajonia Asunción
country: Paraguay
facility: Instituto Privado de Hematologia E Investigacion Clinica (I.P.H.I.C)
city: Morningside
country: South Africa
facility: Morningside Medi-Clinic
Study officials:
References:
citation: 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.