Overview

This trial is active, not recruiting.

Condition hypophosphatasia (hpp)
Treatment asfotase alfa
Phase phase 2
Sponsor Alexion Pharma International Sarl
Start date April 2010
End date December 2015
Trial size 12 participants
Trial identifier NCT01203826, ENB-008-10

Summary

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

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
asfotase alfa 6 mg/kg/week SC injection
asfotase alfa human recombinant tissue non-specific alkaline phosphatase fusion protein

Primary Outcomes

Measure
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

Eligibility Criteria

Male or female participants from 5 years up to 13 years old.

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 involving an investigational drug, device, or treatment for HPP

Additional Information

Official title Extension Study of Protocol ENB-006-09 Evaluating the Long-term Safety and Efficacy of ENB-0040 (Human Recombinant Tissue Nonspecific Alkaline Phosphatase Fusion Protein) in Children With Hypophosphatasia (HPP)
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 no approved disease-modifying treatments for patients with this disease. There is also limited data available on the natural course of this disease over time, particularly in patients with the juvenile-onset form.
Trial information was received from ClinicalTrials.gov and was last updated in May 2015.
Information provided to ClinicalTrials.gov by Alexion Pharma International Sarl.