Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP)

Hypophosphatasia (HPP) Clinical Trial

Official title:

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01203826
• Other study ID #: ENB-008-10
• Status: Completed
• Phase: Phase 2
• Start date: April 2010
• Est. completion date: June 2016

Study information

• Verified date: March 2019
• Source: Alexion Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 12
• Est. completion date: June 2016
• Est. primary completion date: June 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: 5 Years to 12 Years

Eligibility

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

Related Conditions & MeSH terms

• Hypophosphatasia
• Hypophosphatasia (HPP)

Intervention

Biological:
• Asfotase Alfa

Locations

Country	Name	City	State
Canada	Children's Hospital Health Sciences Centre	Winnipeg	Manitoba
United States	Shriners Hospital for Children	Saint Louis	Missouri

Sponsors (1)

Lead Sponsor	Collaborator
Alexion Pharmaceuticals

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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

External Links

•   HPP support group
•   Hypophosphatasia Website
•   Hypophosphatasia Website for Healthcare Providers
•   Hypophosphatasie Europe
•   US Hypophosphatasia Group (Soft Bones)