A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
NCT01132690
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Adult Trials: 18+ Years
- 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).
- 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.
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| Descriptive Information | |||
|---|---|---|---|
| Brief Title ICMJE | A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease | ||
| Official Title ICMJE | A Multicenter, Double-blind, Randomized Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease | ||
| 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. | ||
| Detailed Description | Not Provided | ||
| Study Type ICMJE | Interventional | ||
| Study Phase ICMJE | Phase 4 | ||
| Study Design ICMJE | Allocation: Randomized Intervention Model: Parallel Assignment Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) Primary Purpose: Treatment | ||
| Condition ICMJE | Gaucher Disease | ||
| Intervention ICMJE | Drug: Taliglucerase alfa
Taliglucerase alfa for infusion every two weeks for 12 months Other Name: prGCD, plant cell expressed glucocerebrosidase | ||
| Study Arms ICMJE |
| ||
| Publications * | 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. | ||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. | |||
| Recruitment Information | |||
| Recruitment Status ICMJE | Completed | ||
| Actual Enrollment ICMJE | 11 | ||
| Original Estimated Enrollment ICMJE | 10 | ||
| Actual Study Completion Date ICMJE | July 2012 | ||
| Actual Primary Completion Date | May 2012 (Final data collection date for primary outcome measure) | ||
| Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
| ||
| Sex/Gender ICMJE |
| ||
| Ages ICMJE | 2 Years to 17 Years (Child) | ||
| Accepts Healthy Volunteers ICMJE | No | ||
| Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||
| Listed Location Countries ICMJE | Israel, Paraguay, South Africa | ||
| Removed Location Countries | United States | ||
| Administrative Information | |||
| NCT Number ICMJE | NCT01132690 | ||
| Other Study ID Numbers ICMJE | PB-06-005 | ||
| Has Data Monitoring Committee | Yes | ||
| U.S. FDA-regulated Product | Not Provided | ||
| IPD Sharing Statement ICMJE | Not Provided | ||
| Responsible Party | Pfizer | ||
| Study Sponsor ICMJE | Pfizer | ||
| Collaborators ICMJE | Not Provided | ||
| Investigators ICMJE | Not Provided | ||
| PRS Account | Pfizer | ||
| Verification Date | September 2018 | ||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP | |||