CI

At a glance

ClinicalIndex Comparison Record
N/ACompleted· 48 enrolled
Drug / intervention
Not specified
Likely dose
Not stated in record
Key inclusion· 3
  • ✓Documented diagnosis of HPP by ALPL gene mutation(s), or low serum ALP with elevated PLP/PEA, or low serum ALP with HPP-related radiographic abnormalities
  • ✓Onset of HPP signs prior to 6 months of age
  • ✓At least one characteristic of severe perinatal/infantile HPP: respiratory compromise requiring support/medication, pyridoxine-responsive seizures, or rachitic chest deformity
Key exclusion· 2
  • ✕Prior treatment with asfotase alfa at any time
  • ✕Clinically significant other disease

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT01419028
NCT01419028N/ACompleted
CompareCT.gov

A Retrospective, Non-interventional Epidemiologic Study of the Natural History of Patients With Severe Perinatal and Infantile Hypophosphatasia (HPP)

Alexion Pharmaceuticals, Inc.·observational·Posted Aug 17, 2011·Updated Apr 1, 2019

In Brief

An observational study for Hypophosphatasia (HPP). Completed, enrolled 48 participants across 12 sites in 7 countries.

Detailed Summary

This study aims to characterize the natural history of patients with severe perinatal or infantile onset HPP.

Study Details

Study Typeobservational
Allocation--
Masking--
Primary Purpose--
ConditionsHypophosphatasia (HPP)
CountriesAustralia, Canada, Germany, Spain, Taiwan, United Kingdom, United States
Collaborators--

Timeline

N/ACompletedFinished
201220132014201520162017201820192020202120222023202420252026
First PostedAug 17, 2011
Enrollment StartAug 1, 2012
Primary CompletionJun 1, 2013
Study CompletionFeb 1, 2014
TodayJul 2, 2026
Enrollment to primary: 10 monthsPosted 14.9 years ago