Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-24 @ 4:16 PM
Ignite Modification Date:
2025-12-24 @ 4:16 PM
NCT ID:
NCT01176266
Eligibility Criteria:
Inclusion Criteria:
Patients must meet all of the following criteria for enrollment in this study:
1. 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.
2. Documented diagnosis of HPP as indicated by:
1. 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.
2. 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.
3. 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
4. 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
3. Onset of symptoms prior to 6 months of age
4. Chronological age or adjusted age for premature infants born ≤ 37 weeks gestation of ≤ 5 years
5. 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:
1. Clinically significant disease that precludes study participation, in the opinion of the Investigator and/or Sponsor
2. Serum calcium or phosphate levels below the normal range
3. Current evidence of treatable form of rickets
4. Prior treatment with bisphosphonates
5. Treatment with an investigational drug within 1 month prior to the start of asfotase alfa treatment
6. Current enrollment in any other study involving an investigational new drug, device or treatment for HPP (e.g., bone marrow transplantation)
7. Intolerance to the investigational product (IP) or any of its excipients
8. Previous participation in the same study
9. Family relative of the Investigator
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
1 Minute
Maximum Age:
5 Years
Study:
NCT01176266
Study Brief:
Protocol Section:
NCT01176266