An Open-Label, Multicenter, Multinational Study of the Safety, Efficacy and Pharmacokinetics of Asfotase alfa (human recombinant tissue nonspecific alkaline phosphatase fusion protein) in Infants and Children = 5 Years of Age with Hypophosphatasia (HPP)

Recruitment & Eligibility

Status: ot Recruiting

Sex: Not specified

Target Recruitment: 69

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.
- Documented diagnosis of HPP as indicated by:
o Total serum alkaline phosphatase (ALP) below the lower limit of normal for age.
o Plasma PLP above the upper limit of normal (unless patient is receiving pyridoxine for seizures).
o 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
o Two or more of the following HPP-related findings:
* History or presence of:
+ Nontraumatic post-natal fracture
+ Delayed fracture healing
* Nephrocalcinosis or history of elevated serum calcium
* Functional craniosynostosis
* Respiratory compromise or rachitic chest deformity
* Vitamin B6-dependent seizures
* Failure to thrive
- Onset of symptoms prior to 6 months of age
- Chronological age of = 5 years or adjusted age for premature infants born = 37 weeks gestation
- Otherwise medically stable in the opinion of the Investigator and/or Sponsor
Are the trial subjects under 18? yes
Number of subjects for this age range: 100
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, such as, but not limited to, hepatitis C virus / human immunodeficiency virus / hepatitis B virus, 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 (eg, bone marrow transplantation)
- Intolerance to the IP or any of its excipients
- Previous participation in the same study

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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