Extension open-label use of ENB-0040 for infantile hypophosphatasia

Extension Study of ENB-0040 (Human Recombinant Tissue-Nonspecific Alkaline Phosphatase Fusion Protein) in Severely Affected Infants and Young Children with Hypophosphatasia (HPP)

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2009-009369-32-GB

Enrollment

Registered

2009-04-14

Start date

2009-03-30

Completion date

Unknown

Last updated

2019-04-30

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

Conditions

Infantile hypoposphatasia MedDRA version: 19.1 Level: PT Classification code 10049933 Term: Hypophosphatasia System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Trade Name: Strensiq Product Name: Asfotase alfa Product Code: Asfotase alfa Pharmaceutical Form: Solution for injection INN or Proposed

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: · Compliant and satisfactory completion, in the opinion of the Sponsor and Investigator, of study ENB-002-08 · 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: 6 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: - The patient has a history of sensitivity to any ENB 0040 constituents - The patient has a clinically significant disease that precludes study participation, in the Investigator’s opinion - The patient has been enrolled in any study involving an investigational drug, device, or treatment for HPP (e.g., one marrow transplantation)

Design outcomes

Primary

Measure	Time frame
Main Objective: - To determine the long-term tolerability of subcutaneous (SC) Asfotase alfa - To assess the long-term efficacy of Asfotase alfa in treating rickets in infants and young children with HPP ; Secondary Objective: - To assess the long-term pharmacodynamics (PD) of SC Asfotase alfa - To assess the effect of SC Asfotase alfa on growth and development - To assess the effect of SC Asfotase alfa on mortality and other clinical signs and symptoms of HPP in infants and young children ; Primary end point(s): Skeletal radiograph using a qualitative Radiographic Global Impression of Change (RGI-C) scale. ;Timepoint(s) of evaluation of this end point: 84 months	—

Measure

Time frame

Main Objective: - To determine the long-term tolerability of subcutaneous (SC) Asfotase alfa - To assess the long-term efficacy of Asfotase alfa in treating rickets in infants and young children with HPP ; Secondary Objective: - To assess the long-term pharmacodynamics (PD) of SC Asfotase alfa - To assess the effect of SC Asfotase alfa on growth and development - To assess the effect of SC Asfotase alfa on mortality and other clinical signs and symptoms of HPP in infants and young children ; Primary end point(s): Skeletal radiograph using a qualitative Radiographic Global Impression of Change (RGI-C) scale. ;Timepoint(s) of evaluation of this end point: 84 months

—

Secondary

Measure	Time frame
Secondary end point(s): Long-term pharmacodynamics of SC Asfotase alfa. Effect of SC Asfotase alfa on growth and development, mortality, and other clinical signs and symptoms of HPP in infants and young children. ;Timepoint(s) of evaluation of this end point: 84 months	—

Countries

United Arab Emirates, United Kingdom, 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