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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT01205152 |
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Date of registration:
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17/09/2010 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Extension Study of Protocol ENB-002-08 - Study of Asfotase Alfa in Infants and Young Children With Hypophosphatasia (HPP)
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Scientific title:
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Extension Study of ENB-0040 (Human Recombinant Tissue-Nonspecific Alkaline Phosphatase Fusion Protein) in Severely Affected Infants and Young Children With Hypophosphatasia (HPP) |
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Date of first enrolment:
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April 2009 |
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Target sample size:
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10 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT01205152 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 2
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Countries of recruitment
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United Arab Emirates
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United Kingdom
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United States
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Key inclusion & exclusion criteria
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Inclusion Criteria
- Patient completed participation in ENB-002-08 (NCT00744042)
- Written informed consent by parent or other legal guardian prior to any study
procedures being performed
- Parent or other legal guardian willing to comply with study requirements
Exclusion Criteria
- History of sensitivity to any of the constituents of the study drug
- Clinically significant disease that precludes study participation
- Enrollment in any study (other than ENB-002-08) involving an investigational drug,
device, or treatment for HPP (e.g., bone marrow transplantation)
Age minimum:
24 Weeks
Age maximum:
42 Months
Gender:
All
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Health Condition(s) or Problem(s) studied
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Hypophosphatasia
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Intervention(s)
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Biological: asfotase alfa
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Primary Outcome(s)
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Long-term Efficacy of Asfotase Alfa in Treating Rickets in Infants and Young Children With Hypophosphatasia (HPP).
[Time Frame: Up to 90 Months]
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Long-term Tolerability of Subcutaneous (SC) Asfotase Alfa
[Time Frame: 84 months]
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Secondary Outcome(s)
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Effect of SC Asfotase Alfa on Growth: Height/Length Z-scores
[Time Frame: Up to 90 Months]
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Effect of SC Asfotase Alfa on Respiratory Function
[Time Frame: Up to 90 Months]
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Long-term Pharmacodynamics (PD) of SC Asfotase Alfa: Pyridoxal-5-phosphate (PLP) Levels
[Time Frame: Up to 90 Months]
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Long-term Pharmacodynamics (PD) of SC Asfotase Alfa: Plasma Inorganic Pyrophosphate (PPi) Levels
[Time Frame: Up to 90 Months]
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Effect of SC Asfotase Alfa on Growth: Weight Z-scores
[Time Frame: Up to 90 Months]
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Secondary ID(s)
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FD-R-003745-03
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ENB-003-08
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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