Retrospective, Non-interventional Natural History of Patients With Juvenile-onset Hypophosphatasia (HPP)
NCT ID: NCT02104219
Last Updated: 2019-04-01
Study Results
Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.
View full resultsBasic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
COMPLETED
32 participants
OBSERVATIONAL
2014-03-31
2014-09-30
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Safety and Efficacy Study of Asfotase Alfa in Severely Affected Infants With Hypophosphatasia (HPP)
NCT00744042
Pharmacokinetic and Dose Response Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia (HPP)
NCT02797821
Evaluate and Monitor Physical Performance of Adults Treated With Asfotase Alfa for Hypophosphatasia
NCT03418389
Extension Study of Protocol ENB-002-08 - Study of Asfotase Alfa in Infants and Young Children With Hypophosphatasia (HPP)
NCT01205152
Natural History Study of ENPP1 Deficiency and the Early-onset Form of ABCC6 Deficiency
NCT05050669
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
OTHER
RETROSPECTIVE
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* Patients with Juvenile-onset HPP, defined as documented onset of first signs/symptoms at ≥ 6 months to ˂18 years
* Documented diagnosis of HPP as indicated by skeletal manifestations and low alkaline phosphatase or genotyping
Exclusion Criteria
* Received other treatment and/or intervention to treat HPP up to 15 years old
* Other clinically significant disease
5 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Alexion Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Atlanta, Georgia, United States
Indianapolis, Indiana, United States
Boston, Massachusetts, United States
Springfield, Missouri, United States
St Louis, Missouri, United States
Durham, North Carolina, United States
Cincinnati, Ohio, United States
Houston, Texas, United States
Seattle, Washington, United States
Sydney, , Australia
Winnipeg, Manitoba, Canada
Le Kremlin-Bicêtre, , France
Paris, , France
Toulouse, , France
Rotterdam, , Netherlands
Moscow, , Russia
Istanbul, , Turkey (Türkiye)
Manchester, , United Kingdom
Sheffield, , United Kingdom
Countries
Review the countries where the study has at least one active or historical site.
Related Links
Access external resources that provide additional context or updates about the study.
Hypophosphatasia Website
Hypophosphatasia Website for Health Care Professionals
HPP support group
US Hypophosphatasia Group (Soft Bones)
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
ALX-HPP-502
Identifier Type: -
Identifier Source: org_study_id
NCT02104206
Identifier Type: -
Identifier Source: nct_alias
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.