Extension Study of Protocol ENB-006-09 -Asfotase Alfa Treatment in Children With Hypophosphatasia
| Tracking Information | |||||||||
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| First Received Date ICMJE | September 15, 2010 | ||||||||
| Last Updated Date | January 24, 2013 | ||||||||
| Start Date ICMJE | April 2010 | ||||||||
| Estimated Primary Completion Date | December 2014 (final data collection date for primary outcome measure) | ||||||||
| Current Primary Outcome Measures ICMJE |
Skeletal radiograph using a qualitative Radiographic Global Impression of Change (RGI-C)scale compared to baseline of treatment in ENB-006-09. [ Time Frame: 60 months ] [ Designated as safety issue: No ] The time points will be pre-treatment (Baseline from the 006 study) to Month 60 of the 008 study which represents 66 months of treatment. |
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| Original Primary Outcome Measures ICMJE |
Skeletal radiograph using a qualitative Radiographic Global Inpression of Change (RGI-C)scale compared to historical controls [ Time Frame: 12 months ] [ Designated as safety issue: No ] | ||||||||
| Change History | Complete list of historical versions of study NCT01203826 on ClinicalTrials.gov Archive Site | ||||||||
| Current Secondary Outcome Measures ICMJE | Not Provided | ||||||||
| Original Secondary Outcome Measures ICMJE |
PK using serum peak and trough levels and PD of Plasma PPi, PLP and serum PTH as biomarkers for HPP [ Time Frame: 12 months ] [ Designated as safety issue: Yes ] | ||||||||
| Current Other Outcome Measures ICMJE | Not Provided | ||||||||
| Original Other Outcome Measures ICMJE | Not Provided | ||||||||
| Descriptive Information | |||||||||
| Brief Title ICMJE | Extension Study of Protocol ENB-006-09 -Asfotase Alfa Treatment in Children With Hypophosphatasia | ||||||||
| Official Title ICMJE | Extension Study of Protocol ENB-006-09 Evaluating the Long-term Safety and Efficacy of Asfotase Alfa (Human Recombinant Tissue Nonspecific Alkaline Phosphatase Fusion Protein) in Children With Hypophosphatasia (HPP) | ||||||||
| Brief Summary | This clinical trial is being conducted to study long term safety and efficacy outcomes in children with hypophosphatasia (HPP) being treated with an investigational study drug called ENB-0040 (Asfotase Alfa). |
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| Detailed Description | Asfotase Alfa was formerly referred to as ENB-0040 Hypophosphatasia (HPP) is a rare inherited form of rickets and osteomalacia caused by inactivating mutations in the gene encoding the tissue-nonspecific isoenzyme of alkaline phosphatase (TNSALP). The prevalence of the disease is thought to be about 1:100,000 although it is markedly higher in a small Canadian Mennonite population (Fraser 1957, Chodirker 1990). Inheritance can be autosomal recessive or dominant, and penetrance is variable resulting in a wide range of clinical expressivity. HPP differs from other forms of rickets and osteomalacia in that serum levels of calcium and phosphorus are generally normal or even elevated (Whyte 2002). Low circulating levels of alkaline phosphatase with elevated serum or urine levels of the TNSALP substrates inorganic pyrophosphate (PPi), pyridoxal 5'-phosphate (PLP) and phosphoethanolamine (PEA) are the biochemical hallmarks of this inborn error of metabolism. Disease severity in HPP is inversely related to the age at symptom presentation. The most severe cases occur in utero and almost invariably result in death, generally due to pulmonary compromise. Infants who present in the first six months of life have about 50% mortality. Children and adults have less severe disease but can have frequent fractures, bone pain, bowing of the long bones and muscle weakness, and morbidity is generally cumulative. Some patients cannot ambulate independently and end up wheelchair-bound. |
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| Study Type ICMJE | Interventional | ||||||||
| Study Phase | Phase 2 | ||||||||
| Study Design ICMJE | Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
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| Condition ICMJE | Hypophosphatasia | ||||||||
| Intervention ICMJE | Biological: Asfotase Alfa
2 mg/kg SC injection 3 times per week for 60 months or 1mg/kg sc injection 6 times per week for 60 months.
Other Name: human recombinant tissue non-specific alkaline phosphatase fusion protein |
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| Study Arm (s) | Experimental: Asfotase Alfa
Asfotase Alfa 6 mg/kg/week SC injection for 60 months
Intervention: Biological: Asfotase Alfa |
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| Publications * | Not Provided | ||||||||
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* 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 | ||||||||
| Enrollment ICMJE | 12 | ||||||||
| Estimated Completion Date | December 2014 | ||||||||
| Estimated Primary Completion Date | December 2014 (final data collection date for primary outcome measure) | ||||||||
| Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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| Gender | Both | ||||||||
| Ages | 5 Years to 13 Years | ||||||||
| Accepts Healthy Volunteers | No | ||||||||
| Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||||||
| Location Countries ICMJE | United States, Canada | ||||||||
| Administrative Information | |||||||||
| NCT Number ICMJE | NCT01203826 | ||||||||
| Other Study ID Numbers ICMJE | ENB-008-10 | ||||||||
| Has Data Monitoring Committee | Yes | ||||||||
| Responsible Party | Alexion International Sàrl | ||||||||
| Study Sponsor ICMJE | Alexion International Sàrl | ||||||||
| Collaborators ICMJE | Not Provided | ||||||||
| Investigators ICMJE |
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| Information Provided By | Alexion International Sàrl | ||||||||
| Verification Date | July 2012 | ||||||||
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ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |
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