Asfotase Alfa in infants and children (≤ 5 Years of Age) with HPP
Research type
Research Study
Full title
An Open-Label, Multicenter, Multinational Study of the Safety, Efficacy and Pharmacokinetics of Asfotase alfa (human recombinant tissue nonspecific alkaline phosphatase fusion protein) in Infants and Children = 5 Years of Age with Hypophosphatasia (HPP)
IRAS ID
122538
Contact name
Wolfgang Högler
Contact email
Sponsor organisation
Alexion Pharmaceuticals Inc.
Eudract number
2010-019850-42
ISRCTN Number
n/a
Research summary
Hypophosphatasia (HPP) is a disorder of bone metabolism caused by a change of genetic material (gene mutations). These gene mutations result in the fact that a protein component (Tissue non-specific alkaline phosphatase (TNSALP) enzyme) that is naturally present in the body and necessary for the hardening of the bones is produced in insufficient amounts.
This study will investigate a new study drug, asfotase alfa, a synthetically produced, altered form of the natural protein component that is necessary for bone hardening.
Asfotase alfa was previously investigated in a 6-month, open-label phase 2 study (study ENB-002-08) of infants and young children less than 3 years of age presenting with severe HPP. During the study, patients received 1 intravenous (into a vein) asfotase alfa infusion followed by subcutaneous injections (under the skin) 3 times a week with dose adjustments allowed for lack of efficacy. Eleven patients were enrolled in the study and ten patients completed the treatment period and rolled over into the extension study ENB-003-08 (still ongoing).
Data from clinical studies with asfotase alfa already conducted in infants and children with HPP have shown that the treatment has been generally well tolerated. In infants, the most common side effects reported to date are fever, vomiting, reduced calcium values, and redness at the injection site, irritability, and bone pain.
For this new study ENB-10-10, 3 research sites will participate in UK and will assess the pharmacokinetics, long-term safety and effects of subcutaneous (under the skin) injections of asfotase alfa when given to infants and children, who are less than or equal to 5 years of age, with HPP.
Eligible patients will be treated with asfotase alfa at a dose of 6 mg/kg/week for a maximum of 48 months. Patients will be monitored and assessed thorough the study. In all, there will be approximately 11 examination visits at the hospital conducted in the first 2 years of this study. After the first 2 years, there will be approximately 2 visits per year until the study is stopped.
REC name
London - Harrow Research Ethics Committee
REC reference
13/LO/0503
Date of REC Opinion
5 Sep 2013
REC opinion
Further Information Favourable Opinion