Extension Study of Protocol ENB-002-08 - Study of Asfotase Alfa in Infants and Young Children With Hypophosphatasia (HPP)

Alexion Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: Hypophosphatasia

Treatments: Biological: asfotase alfa

Study type: Interventional

Funder types: Industry

Identifiers

NCT01205152

ENB-003-08

FD-R-003745-03 (Other Grant/Funding Number)

Details and patient eligibility

About

This clinical trial studied the long term safety and efficacy of asfotase alfa in infants and young children with infantile onset HPP who completed study ENB-002-08 (NCT00744042).

Partial funding for this study was provided by the Office of Orphan Product Development (OOPD).

Full 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.

Enrollment: 10 patients

Sex: All

Ages: 24 weeks to 42 months old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria

• Patient completed participation in ENB-002-08 (NCT00744042)
• 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

• History of sensitivity to any of the constituents of the study drug
• Clinically significant disease that precludes study participation
• Enrollment in any study (other than ENB-002-08) involving an investigational drug, device, or treatment for HPP (e.g., bone marrow transplantation)

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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10 participants in 1 patient group

asfotase alfa

Experimental group

Description:

An initial single intravenous (IV) infusion of 2 mg/kg asfotase alfa, followed by subcutaneous (SC) injections of 1 mg/kg asfotase alfa 3 times per week

Treatment:

Biological: asfotase alfa