Status:
COMPLETED
Open-Label Study of Asfotase Alfa in Infants and Children ≤ 5 Years of Age With Hypophosphatasia (HPP)
Lead Sponsor:
Alexion Pharmaceuticals, Inc.
Conditions:
Hypophosphatasia
Eligibility:
All Genders
1-5 years
Phase:
PHASE2
PHASE3
Brief Summary
This clinical trial was conducted to study hypophosphatasia (HPP), a bone disorder caused by gene mutations or changes. These gene mutations cause low levels of an enzyme needed to harden bone. The pu...
Detailed Description
Asfotase alfa was formerly referred to as ENB-0040 Hypophosphatasia (HPP) is a life-threatening, genetic, and ultra-rare metabolic disease characterized by defective bone mineralization and impaired ...
Eligibility Criteria
Inclusion
- Inclusion Criteria:
- Patients must meet all of the following criteria for enrollment in this study:
- Parent or legal guardian(s) must provide written informed consent prior to any study procedures being performed and must be willing to comply with all study-required procedures. Where appropriate and required by local regulations, patient assent should also be provided prior to any study procedures being performed.
- Documented diagnosis of HPP as indicated by:
- Total serum alkaline phosphatase (ALP) below the lower limit of normal for age NOTE: Historical values for ALP may be used to determine patient eligibility.
- Plasma pyridoxal-5'-phosphate (PLP) above the upper limit of normal (unless patient is receiving pyridoxine for seizures) NOTE: Historical values for PLP may be used to determine patient eligibility.
- Radiographic evidence of HPP at screening, characterized by:
- Flared and frayed metaphyses, and
- Severe, generalized osteopenia, and
- Widened growth plates, and
- Areas of radiolucency or sclerosis
- Two or more of the following HPP-related findings:
- History or presence of: i) Nontraumatic post-natal fracture or ii) Delayed fracture healing
- Nephrocalcinosis or history of elevated serum calcium
- Functional craniosynostosis
- Respiratory compromise or rachitic chest deformity
- Vitamin B6-responsive seizures
- Failure to thrive
- Onset of symptoms prior to 6 months of age
- Chronological age or adjusted age for premature infants born ≤ 37 weeks gestation of ≤ 5 years
- Otherwise medically stable in the opinion of the Investigator and/or Sponsor
- 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 opinion of the Investigator and/or Sponsor
- Serum calcium or phosphate levels below the normal range
- Current evidence of treatable form of rickets
- Prior treatment with bisphosphonates
- Treatment with an investigational drug within 1 month prior to the start of asfotase alfa treatment
- Current enrollment in any other study involving an investigational new drug, device or treatment for HPP (e.g., bone marrow transplantation)
- Intolerance to the investigational product (IP) or any of its excipients
- Previous participation in the same study
- Family relative of the Investigator
Exclusion
Key Trial Info
Start Date :
July 1 2010
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 1 2016
Estimated Enrollment :
69 Patients enrolled
Trial Details
Trial ID
NCT01176266
Start Date
July 1 2010
End Date
September 1 2016
Last Update
March 13 2019
Active Locations (23)
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1
Children's Hospital & Research Center Oakland
Oakland, California, United States, 94609
2
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
3
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States, 15224
4
Lady Cilento Children's Hospital
South Brisbane, Queensland, Australia, 4101