Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP)
1 other identifier
interventional
12
2 countries
2
Brief Summary
This clinical trial studies the long term safety and efficacy of asfotase alfa in children with HPP who completed Study ENB-006-09 (NCT00952484).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Apr 2010
Longer than P75 for phase_2
2 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
April 1, 2010
CompletedFirst Submitted
Initial submission to the registry
September 15, 2010
CompletedFirst Posted
Study publicly available on registry
September 16, 2010
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 1, 2016
CompletedStudy Completion
Last participant's last visit for all outcomes
June 1, 2016
CompletedResults Posted
Study results publicly available
July 26, 2017
CompletedMarch 13, 2019
March 1, 2019
6.2 years
September 15, 2010
June 27, 2017
March 11, 2019
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Skeletal Radiograph Evaluation Using a Qualitative Radiographic Global Impression of Change (RGI-C) Scale Compared to Baseline (Pre-treatment) in Study ENB-006-09.
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
Study Arms (1)
asfotase alfa
EXPERIMENTALasfotase alfa starting dose 3 mg/kg/week SC injection, increased to 6 mg/kg/week SC injection
Interventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (2)
Shriners Hospital for Children
St Louis, Missouri, 63110, United States
Children's Hospital Health Sciences Centre
Winnipeg, Manitoba, R3A 1S1, Canada
Related Publications (2)
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.
PMID: 32417537DERIVEDWhyte 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.
PMID: 27699270DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Director of Clinical Trials
- Organization
- Alexion Pharmaceuticals, Inc.
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- LTE60
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
September 15, 2010
First Posted
September 16, 2010
Study Start
April 1, 2010
Primary Completion
June 1, 2016
Study Completion
June 1, 2016
Last Updated
March 13, 2019
Results First Posted
July 26, 2017
Record last verified: 2019-03