Safety and Efficacy Study of Asfotase Alfa in Severely Affected Infants With Hypophosphatasia (HPP)

Completed · Phase 1/Phase 2

Conditions studied: Hypophosphatasia (HPP)

In brief

This clinical trial studies the safety and efficacy of asfotase alfa in infants and young children with infantile onset HPP.

Key facts

Study ID

NCT00744042

Run by

Alexion Pharmaceuticals, Inc.

People needed

11

Starts

2008-09-01

Expected to finish

2010-05-01

Last updated by the study team

2019-04-01

Who can join

Age: any, up to 3. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Arkansas Children's Hospital — Little Rock, Arkansas, United States
• Alfred I. duPont Hospital for Children — Wilmington, Delaware, United States
• St. John's Hospital — Springfield, Missouri, United States
• University of Nebraska Medical Center, Munroe-Meyer Institute — Omaha, Nebraska, United States
• Vanderbilt Children's Hospital — Nashville, Tennessee, United States
• St. Vincent Hospital — Green Bay, Wisconsin, United States
• The University of Manitoba Health Sciences Centre — Winnipeg, Manitoba, Canada
• Tawam-John Hopkins Hospital — Al Ain City, Abu Dhabi Emirate, United Arab Emirates
• Sheffield Children's Hospital — Sheffield, England, United Kingdom
• Royal Belfast Hospital for Sick Children — Belfast, Northern Ireland, United Kingdom

Full record on ClinicalTrials.gov

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