ong-Term Safety and Efficacy of Asfotase Alfa in Children with Hypophosphatasia (HPP)

Phase 1

Conditions: Therapeutic area: Body processes [G] - Genetic Phenomena [G05]
Hypophosphatasia
MedDRA version: 20.0Level: PTClassification code 10049933Term: HypophosphatasiaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders

Registration Number: EUCTR2017-003153-42-Outside-EU/EEA

Lead Sponsor: Alexion Pharma GmbH

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: A

Sex: All

Target Recruitment: 12

Inclusion Criteria

Patients must meet all of the following criteria for enrollment in this study:
? Compliant and satisfactory completion of Alexion-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
Are the trial subjects under 18? yes
Number of subjects for this age range: 12
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Patients will be excluded from enrollment in this study if they meet any of the following exclusion criteria:
? Clinically significant disease that precludes study participation, in the Investigator’s opinion
? Treatment with an investigational drug other than asfotase alfa
? Prior treatment with bisphosphonates
? Enrollment in any study (other than ENB-006-09) involving an investigational drug, device, or treatment
for HPP

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To assess the long-term tolerability of subcutaneous (SC) asfotase alfa<br>To assess the proportion of asfotase alfa-treated patients showing radiographic change in the skeletal manifestations of HPP ;Secondary Objective: To evaluate the long-term pharmacokinetics (PK) of SC asfotase alfa<br>To evaluate the effect of SC asfotase alfa on reduction in plasma inorganic pyrophosphate (PPi) and plasma pyridoxal-5’-phosphate (PLP)<br>;Primary end point(s): - Long-term tolerability of subcutaneous (SC) asfotase alfa<br>- Proportion of asfotase alfa-treated patients showing radiographic change in the skeletal manifestations of HPP ;Timepoint(s) of evaluation of this end point: Throughout the study<br>

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): To evaluate the long-term pharmacokinetics (PK) of SC asfotase alfa<br>To evaluate the effect of SC asfotase alfa on reduction in plasma inorganic pyrophosphate (PPi) and plasma pyridoxal-5’-phosphate (PLP);Timepoint(s) of evaluation of this end point: Throughout the study<br>