Status:
COMPLETED
Safety and Efficacy Study of Asfotase Alfa in Severely Affected Infants With Hypophosphatasia (HPP)
Lead Sponsor:
Alexion Pharmaceuticals, Inc.
Conditions:
Hypophosphatasia (HPP)
Eligibility:
All Genders
Up to 36 years
Phase:
PHASE1
PHASE2
Brief Summary
This clinical trial studies the safety and efficacy of asfotase alfa in infants and young children with infantile onset HPP.
Detailed Description
Hypophosphatasia (HPP) is a life-threatening, genetic, and ultra-rare metabolic disease characterized by defective bone mineralization and impaired phosphate and calcium regulation that can lead to pr...
Eligibility Criteria
Inclusion
- Legal guardian(s) must provide informed consent prior to any study procedures
- Documented diagnosis of severe HPP as indicated by:
- Total serum alkaline phosphatase at least 3 standard deviations (SD) below the mean for age
- Plasma pyridoxal 5'-phosphate (PLP) at least 4 times the upper limit of normal
- Radiographic evidence of HPP (hypophosphatasia), characterized by:
- Flared and frayed metaphyses
- Severe, generalized osteopenia
- Widened growth plates
- One or more HPP-related findings:
- History or presence of:
- Non-traumatic post-natal fracture
- Delayed fracture healing
- History of elevated serum calcium
- Functional craniosynostosis with decreased head circumference growth
- Nephrocalcinosis
- Respiratory compromise
- Rachitic chest deformity and/or vitamin B6 dependent seizures
- Failure to thrive
- Onset of symptoms prior to 6 months of age
- Age ≤ 36 months
- Otherwise medically stable (patient may be on ventilatory support)
- Legal guardian(s) must be willing to comply with the study
Exclusion
- History of sensitivity to any of the constituents of the study drug
- Current or prior clinically significant cardiovascular, endocrinologic, hematologic, hepatic, immunologic, metabolic, infectious, urologic, pulmonary, neurologic, dermatologic, renal condition and/or other major disease which, in the opinion of the investigator, precludes study participation
- Treatment with an investigational drug within 1 month prior to the start of study drug administration
- Current enrollment in any other study involving an investigational new drug, device or treatment for HPP (e.g., bone marrow transplantation)
- Low serum calcium, phosphate or 25(OH) vitamin D
- Current evidence of a treatable form of rickets
- Prior treatment with bisphosphonate
Key Trial Info
Start Date :
September 1 2008
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 1 2010
Estimated Enrollment :
11 Patients enrolled
Trial Details
Trial ID
NCT00744042
Start Date
September 1 2008
End Date
May 1 2010
Last Update
April 1 2019
Active Locations (10)
Enter a location and click search to find clinical trials sorted by distance.
1
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
2
Alfred I. duPont Hospital for Children
Wilmington, Delaware, United States, 19803
3
St. John's Hospital
Springfield, Missouri, United States, 65804
4
University of Nebraska Medical Center, Munroe-Meyer Institute
Omaha, Nebraska, United States, 68114