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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01132690
• Date of registration: 26/05/2010
• Prospective Registration: Yes
• Primary sponsor: Pfizer
• Public title: A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
• Scientific title: A Multicenter, Double-blind, Randomized Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
• Date of first enrolment: August 2010
• Target sample size: 11
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01132690
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 4

Countries of recruitment

Israel, Paraguay, South Africa, United States

Key inclusion & exclusion criteria

Inclusion Criteria:

- Males and females 2 to <18 years old.

- Diagnosis of Gaucher disease with leukocyte acid ß-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.



Age minimum: 2 Years
Age maximum: 17 Years
Gender: All

Health Condition(s) or Problem(s) studied

Gaucher Disease

Intervention(s)

Drug: Taliglucerase alfa

Primary Outcome(s)

Hemoglobin [Time Frame: Every 3 months for 12 months]

Secondary Outcome(s)

Platelet Count [Time Frame: Baseline and 12 months]

Chitotriosidase [Time Frame: Every 3 months for 12 months]

Liver Volume [Time Frame: Baseline and Month 12]

Spleen Volume [Time Frame: Baseline and Month 12]

Chemokine (C-C Motif) Ligand 18 (CCL18) [Time Frame: Every 3 months for 12 months]

Secondary ID(s)

PB-06-005

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 11/11/2014
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01132690