Phase 2
N=11
Safety and Efficacy Study of Asfotase Alfa in Severely Affected Infants With Hypophosphatasia (HPP)
Hypophosphatasia (HPP)
Bottom Line
View on ClinicalTrials.gov: NCT00744042 ↗Enrolled (actual)
11
Serious AEs
63.6%
Results posted
Sep 2011
Primary outcome: Primary: Change in Rickets Severity From Baseline to Week 24, Based on Assessment of Skeletal Radiographs Using Radiologic Global Impression of Change (RGI-C) — 2.00 Units on a scale — p=0.0039
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- asfotase alfa (Biological)
- Age
- Pediatric
- Sex
- All
- Sponsor
- Alexion Pharmaceuticals, Inc.
- Primary completion
- May 2010
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in Rickets Severity From Baseline to Week 24, Based on Assessment of Skeletal Radiographs Using Radiologic Global Impression of Change (RGI-C) |
2.00 | 0.0039 sig |
| SECONDARY Maximum Serum Concentration of Asfotase Alfa (Cmax) |
2230; 376; 897 | — |
| SECONDARY Time at Maximum Serum Concentration of Asfotase Alfa (Tmax) |
4.3; 29.7; 12.0 | — |
| SECONDARY Area Under Serum Concentration-time Curve to Last Measurable Concentration of Asfotase Alfa (AUCt) |
79800; 14700; 33700 | — |
Summary
This clinical trial studies the safety and efficacy of asfotase alfa in infants and young children with infantile onset HPP.
Eligibility Criteria
Inclusion Criteria
- 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 Criteria
- 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
Data sourced from ClinicalTrials.gov (NCT00744042). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.