Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease
This study is ongoing, but not recruiting participants.
Sponsor:
BioMarin Pharmaceutical
Information provided by (Responsible Party):
BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:
NCT01230801
First received: October 27, 2010
Last updated: September 20, 2012
Last verified: September 2012
Tracking Information | |||||
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First Received Date ICMJE | October 27, 2010 | ||||
Last Updated Date | September 20, 2012 | ||||
Start Date ICMJE | December 2010 | ||||
Estimated Primary Completion Date | December 2012 (final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures ICMJE |
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Original Primary Outcome Measures ICMJE |
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Change History | Complete list of historical versions of study NCT01230801 on ClinicalTrials.gov Archive Site | ||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
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Current Other Outcome Measures ICMJE | |||||
Original Other Outcome Measures ICMJE | |||||
Descriptive Information | |||||
Brief Title ICMJE | Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease | ||||
Official Title ICMJE | A Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant Human GAA) in Patients With Late-onset Pompe Disease | ||||
Brief Summary | A Phase 1/2, open-label, multicenter, multiple dose escalation study of BMN 701 administered by intravenous infusion every 2 weeks over a 24-week treatment period to patients with late-onset Pompe disease. |
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Detailed Description | |||||
Study Type ICMJE | Interventional | ||||
Study Phase | Phase 1 Phase 2 |
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Study Design ICMJE | Endpoint Classification: Safety Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
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Condition ICMJE | Pompe Disease | ||||
Intervention ICMJE | Biological: BMN 701
GILT-tagged recombinant human GAA |
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Study Arm (s) | Experimental: BMN 701
IV infusion
Intervention: Biological: BMN 701 |
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Publications * | |||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||
Recruitment Status ICMJE | Active, not recruiting | ||||
Estimated Enrollment ICMJE | 30 | ||||
Estimated Completion Date | December 2012 | ||||
Estimated Primary Completion Date | December 2012 (final data collection date for primary outcome measure) | ||||
Eligibility Criteria ICMJE | Inclusion criteria:
Exclusion criteria:
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Gender | Both | ||||
Ages | 13 Years and older | ||||
Accepts Healthy Volunteers | No | ||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||
Location Countries ICMJE | United States, Australia, France, Germany, United Kingdom | ||||
Administrative Information | |||||
NCT Number ICMJE | NCT01230801 | ||||
Other Study ID Numbers ICMJE | POM-001, 2010-023561-22 | ||||
Has Data Monitoring Committee | No | ||||
Responsible Party | BioMarin Pharmaceutical | ||||
Study Sponsor ICMJE | BioMarin Pharmaceutical | ||||
Collaborators ICMJE | |||||
Investigators ICMJE |
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Information Provided By | BioMarin Pharmaceutical | ||||
Verification Date | September 2012 | ||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |