- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01203826
Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP)
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)
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Asfotase alfa was formerly referred to as ENB-0040
Hypophosphatasia (HPP) is a life-threatening, genetic, and ultra-rare metabolic disease characterized by defective bone mineralization and impaired phosphate and calcium regulation that can lead to progressive damage to multiple vital organs, including destruction and deformity of bones, profound muscle weakness, seizures, impaired renal function, and respiratory failure. There are limited data available on the natural course of this disease over time, particularly in patients with the juvenile-onset form.
Study Type
Enrollment (Actual)
Phase
- Phase 2
Contacts and Locations
Study Locations
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Manitoba
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Winnipeg, Manitoba, Canada, R3A 1S1
- Children's Hospital Health Sciences Centre
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Missouri
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Saint Louis, Missouri, United States, 63110
- Shriners Hospital for Children
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- Compliant and satisfactory completion of Enobia-sponsored clinical trial ENB-006-09
- 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:
- Clinically significant disease that precludes study participation, in the Investigator's opinion
- Treatment with an investigational drug other than asfotase alfa
- Enrollment in any study (other than ENB-006-09) involving an investigational drug, device, or treatment for HPP
- Prior treatment with bisphosphonates
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: asfotase alfa
asfotase alfa starting dose 3 mg/kg/week SC injection, increased to 6 mg/kg/week SC injection
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Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Skeletal Radiograph Evaluation Using a Qualitative Radiographic Global Impression of Change (RGI-C) Scale Compared to Baseline (Pre-treatment) in Study ENB-006-09.
Time Frame: At least 72 months of treatment with asfotase alfa
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Evaluation of radiographic change in rickets severity (as assessed by skeletal radiographs of the hands/wrists and knees) from the Baseline of Study ENB-006-09 (NCT00952484) to the End of Study (EOS) visit in Study ENB-008-10 using an ordinal RGI-C scale score. The RGI-C is a 7-point rating scale that ranges from -3 (indicative of severe worsening of HPP associated rickets) to +3 (indicative of complete or near complete healing of HPP associated rickets). The time points will be pre-treatment (Baseline from Study ENB-006-09) to the last radiographic assessment in Study ENB-008-10, which represents at least 72 months of treatment. |
At least 72 months of treatment with asfotase alfa
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Collaborators and Investigators
Sponsor
Publications and helpful links
General Publications
- Simmons JH, Rush ET, Petryk A, Zhou S, Martos-Moreno GA. Dual X-ray absorptiometry has limited utility in detecting bone pathology in children with hypophosphatasia: A pooled post hoc analysis of asfotase alfa clinical trial data. Bone. 2020 Aug;137:115413. doi: 10.1016/j.bone.2020.115413. Epub 2020 May 14.
- Whyte MP, Madson KL, Phillips D, Reeves AL, McAlister WH, Yakimoski A, Mack KE, Hamilton K, Kagan K, Fujita KP, Thompson DD, Moseley S, Odrljin T, Rockman-Greenberg C. Asfotase alfa therapy for children with hypophosphatasia. JCI Insight. 2016 Jun 16;1(9):e85971. doi: 10.1172/jci.insight.85971.
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- ENB-008-10
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Hypophosphatasia (HPP)
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AlexionEnrolling by invitationHypophosphatasia (HPP)France, Poland, United Kingdom, Germany, Spain, Saudi Arabia, United States, Canada, Russian Federation, Australia, Italy
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Alexion PharmaceuticalsCompletedHypophosphatasia (HPP)United States, Canada
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Alexion PharmaceuticalsCompletedHypophosphatasia (HPP)United States, France, United Kingdom, Canada, Netherlands, Russian Federation, Turkey, Australia
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Alexion PharmaceuticalsCompletedHypophosphatasia (HPP)United States, Canada, United Arab Emirates, United Kingdom
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Hvidovre University HospitalOdense University HospitalActive, not recruitingHypophosphatasia (HPP)Denmark
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Alexion PharmaceuticalsCompletedHypophosphatasia (HPP)United States, Taiwan, United Kingdom, Australia, Canada, Germany, Spain
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Alexion PharmaceuticalsCompletedHypophosphatasia (HPP)United States, Canada
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Alexion Pharmaceuticals, Inc.Not yet recruitingHypophosphatasiaUnited States, United Kingdom, Italy, Japan, Australia, Turkey, France, Germany, Argentina, Canada
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Alexion Pharmaceuticals, Inc.Not yet recruiting
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AlexionRecruiting
Clinical Trials on Asfotase Alfa
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Alexion PharmaceuticalsWithdrawn
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Alexion Pharmaceuticals, Inc.Not yet recruitingHypophosphatasiaUnited States, United Kingdom, Italy, Japan, Australia, Turkey, France, Germany, Argentina, Canada
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AlexionRecruiting
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Alexion PharmaceuticalsCompletedHypophosphatasiaUnited States, Germany
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Alexion Pharmaceuticals, Inc.Xcenda, LLCActive, not recruitingHypophosphatasiaUnited States
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Alexion PharmaceuticalsCompleted
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Alexion PharmaceuticalsApproved for marketingHypophosphatasiaUnited States, France
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Alexion PharmaceuticalsCompletedHypophosphatasiaUnited States, United Arab Emirates, United Kingdom
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Alexion PharmaceuticalsCompletedHypophosphatasiaUnited States, Spain, Australia, United Kingdom, Italy, France, Canada, Germany, Japan, Russian Federation, Saudi Arabia, Turkey
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Translational Research Center for Medical Innovation...Osaka University Graduate School of MedicineCompleted