An Open-label, Multicenter, Expanded Access Program for Asfotase Alfa (human recombinant tissue-nonspecific alkaline phosphatase fusion protein) Treatment for Patients with Infantile- or Juvenile-onset Hypophosphatasia (HPP)

Alexion Pharma International Sàrl0 sites100 target enrollmentAugust 5, 2015

Overview

No summary available.

Registry

who.int

Start Date

August 5, 2015

End Date

TBD

Last Updated

8 years ago

Study Type

Interventional clinical trial of medicinal product

Sex

All

Sponsor

•Patients must meet all of the following inclusion criteria to be eligible for participation in this program:
•1\.Parent or legal guardian must provide written informed consent prior to the performance of any program\-related procedures and must be willing to comply with program procedures. Where appropriate and required by local regulations, patient assent for participation must also be obtained.
•2\.Patient must have a documented diagnosis of HPP as indicated by a documented history of HPP\-related skeletal abnormalities and one or more of the following:
•Documented tissue\-nonspecific alkaline phosphatase (TNSALP) gene mutation(s)
•Serum alkaline phosphatase (ALP) level below the age\-adjusted normal range AND plasma pyridoxal\-5’\-phosphate (PLP) above the upper limit of normal at Screening.
•3\.Patient must have infantile\- or juvenile\-onset HPP, defined as documented onset of signs/symptoms of HPP prior to 18 years of age.
•4\.Male patient is:
•Prepubertal; OR
•Surgically sterile (defined as vasectomized for \=6 months at Baseline); OR
•Non\-surgically sterile (defined as non\-vasectomized or vasectomized for \<6 months at Baseline) and his female spouse/partner who is of childbearing potential must be using highly effective contraception consisting of two forms of birth control (at least one of which must be a barrier method) as described below starting at Baseline and continuing for 3 months after program completion.

•Patients will be excluded from participation in this program if they meet one or more of the following exclusion criteria:
•1\.Patient has geographic access to and qualifies for enrollment (as determined by the Sponsor and Investigator) in any other Alexion\-sponsored clinical trials of asfotase alfa that are open to enrollment (eg, ENB\-010\-10\).
•2\.Patient is pregnant, trying to become pregnant, or is lactating.
•3\.Patient has a documented sensitivity to any of the components of asfotase alfa.
•4\.Patient has serum calcium and/or phosphorus levels below the normal range at Screening.
•5\.Patient is currently enrolled in any other program or study involving an investigational new drug, device, or treatment for HPP (eg, bone marrow transplantation).
•6\.Patient has any other medical condition, serious intercurrent illness, or other extenuating circumstance that, in the opinion of the investigator, may significantly interfere with program compliance, including all evaluations and follow\-up activities.