A Retrospective Study of the Natural History of Patients With Severe Perinatal and Infantile Hypophosphatasia (HPP)

Study Identifier:
ENB-011-10
ClinicalTrials.gov Identifier:
NCT01419028
EudraCT Identifier:
N/A
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Study Complete

Study Details

Medical Condition
  • Unmapped
Study Drug
    Date
    Aug 2012 - Jun 2013
    Phase 1
    Phase 2
    Phase 3
    Phase 4
    N/A
    Patient Requirements
    Sex: Female & Male
    Age: N/A
    Requirements Information
    Inclusion and Exclusion Criteria
    Inclusion Criteria
    • Parent(s) or legal guardian(s) must provide written informed consent prior to data abstraction, unless all of the following apply:
    • The patient is deceased; AND
    • The responsible IRB/IEC/REB does not require informed consent per a review of their documented local policies for collecting retrospective data on patients who are deceased; AND
    • Written confirmation is received from the responsible IRB/IEC/REB confirming that the abstracted data can be analyzed and used to support regulatory filings by the Sponsor
    • Patient must have a documented diagnosis of HPP as indicated by 1 or more of the following:
    • Documented ALPL gene mutation(s)
    • Serum alkaline phosphatase (ALP) below the age-adjusted normal range and either plasma pyridoxal 5'-phosphate (PLP) or urinary phosphoethanolamine (PEA) above the upper limit of normal
    • Serum ALP below the age-adjusted normal range and HPP-related radiographic abnormalities on X-ray
    • Patient must have onset of signs of HPP prior to 6 months of age and have documentation of 1 or more of the following characteristics of perinatal and infantile HPP:
    • Respiratory compromise (up to and including respiratory failure) requiring institution of respiratory support measure(s), requiring medication(s) for management of symptom(s), and/or associated with other respiratory complications (e.g., pneumonia(s), respiratory tract infection(s))
    • Pyridoxine (vitamin B6)-responsive seizures
    • Rachitic chest deformity
    Exclusion Criteria
    • Patients will be excluded from study participation if they have 1 or more of the following exclusion criteria:
    • Patient received treatment with asfotase alfa at any time prior to data abstraction
    • Patient has clinically significant other disease
    • Both living and deceased patients will be considered for study participation
    Sex
    Female & Male
    Age
    N/A

    Study Details

    Medical Condition
    • Unmapped
    Study Drug
      Date
      Aug 2012 - Jun 2013
      Phase 1
      Phase 2
      Phase 3
      Phase 4
      N/A
      Patient Requirements
      Sex: Female & Male
      Age: N/A years
      Requirements Information
      Inclusion and Exclusion Criteria
      Inclusion Criteria
      • Parent(s) or legal guardian(s) must provide written informed consent prior to data abstraction, unless all of the following apply:
      • The patient is deceased; AND
      • The responsible IRB/IEC/REB does not require informed consent per a review of their documented local policies for collecting retrospective data on patients who are deceased; AND
      • Written confirmation is received from the responsible IRB/IEC/REB confirming that the abstracted data can be analyzed and used to support regulatory filings by the Sponsor
      • Patient must have a documented diagnosis of HPP as indicated by 1 or more of the following:
      • Documented ALPL gene mutation(s)
      • Serum alkaline phosphatase (ALP) below the age-adjusted normal range and either plasma pyridoxal 5'-phosphate (PLP) or urinary phosphoethanolamine (PEA) above the upper limit of normal
      • Serum ALP below the age-adjusted normal range and HPP-related radiographic abnormalities on X-ray
      • Patient must have onset of signs of HPP prior to 6 months of age and have documentation of 1 or more of the following characteristics of perinatal and infantile HPP:
      • Respiratory compromise (up to and including respiratory failure) requiring institution of respiratory support measure(s), requiring medication(s) for management of symptom(s), and/or associated with other respiratory complications (e.g., pneumonia(s), respiratory tract infection(s))
      • Pyridoxine (vitamin B6)-responsive seizures
      • Rachitic chest deformity
      Exclusion Criteria
      • Patients will be excluded from study participation if they have 1 or more of the following exclusion criteria:
      • Patient received treatment with asfotase alfa at any time prior to data abstraction
      • Patient has clinically significant other disease
      • Both living and deceased patients will be considered for study participation

      Protocol Summary

      This study aims to characterize the natural history of patients with severe perinatal or infantile onset HPP.

      Trial Locations

      Location
      Status
      Location
      Cedars-Sinai Medical Center
      Los Angeles, California, United States
      Status
      N/A
      Location
      Indiana University school of medicine
      Indianapolis, Indiana, United States, 46202
      Status
      N/A
      Location
      Shriners Hospital for Children
      St Louis, Missouri, United States
      Status
      N/A
      Location
      Oregon Health & Science University
      Portland, Oregon, United States
      Status
      N/A
      Location
      Cook Children's Health Care System
      Fort Worth, Texas, United States
      Status
      N/A
      Location
      Royal Children's Hospital
      Parkville, Australia
      Status
      N/A
      Go to page