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

Phase 2CompletedNCT01203826

Alexion Pharmaceuticals, Inc.12 enrolled

Overview

This clinical trial studies the long term safety and efficacy of asfotase alfa in children with HPP who completed Study ENB-006-09 (NCT00952484).

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

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Hypophosphatasia (HPP)

Interventions

Asfotase AlfaBIOLOGICAL

Eligibility

Sex: ALLMin age: 5 YearsMax age: 12 Years

Medical Language ↔ Plain English

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

Locations (2)

Shriners Hospital for Children

St Louis, Missouri, United States

Children's Hospital Health Sciences Centre

Winnipeg, Manitoba, Canada

Outcomes

Primary Outcomes

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.

Time frame: At least 72 months of treatment with asfotase alfa

Data from ClinicalTrials.gov

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