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

Extension Study of Protocol ENB-006-09 Evaluating the Long-Term Safety and Efficacy of Asfotase Alfa (Human Recombinant Tissue-Nonspecific Alkaline Phosphatase Fusion Protein) in Children with Hypophosphatasia (HPP)

Status
Unknown
Phases
Phase 2
Study type
Interventional
Source
EU CTR
Registry ID
EUCTR2017-003153-42-Outside-EU/EEA
Enrollment
12
Registered
2017-08-03
Start date
Unknown
Completion date
Unknown
Last updated
2017-08-21

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hypophosphatasia MedDRA version: 20.0 Level: PT Classification code 10049933 Term: Hypophosphatasia System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Trade Name: Strensiq Product Name: Asfotase alfa Pharmaceutical Form: Solution for injection INN or Proposed INN: ASFOTASE ALFA Other descriptive name: Human recombinant tissue non-specific alkaline p

Sponsors

Alexion Pharma GmbH
Lead Sponsor

Eligibility

Sex/Gender
All

Inclusion criteria

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

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

Design outcomes

Primary

MeasureTime frame
Main Objective: To assess the long-term tolerability of subcutaneous (SC) asfotase alfa 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 To evaluate the effect of SC asfotase alfa on reduction in plasma inorganic pyrophosphate (PPi) and plasma pyridoxal-5’-phosphate (PLP) ;Primary end point(s): - Long-term tolerability of subcutaneous (SC) asfotase alfa - 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—

Secondary

MeasureTime frame
Secondary end point(s): To evaluate the long-term pharmacokinetics (PK) of SC asfotase alfa 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—

Countries

Canada, United States

Contacts

Public ContactEuropean Clinical Trial Information

Alexion Europe SAS

clinicaltrials.eu@alexion.com+33147100606

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026