Retrospective, Non-interventional Natural History of Patients With Juvenile-onset Hypophosphatasia (HPP)

This study is currently recruiting participants. (see Contacts and Locations)
Verified April 2014 by Alexion Pharmaceuticals
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT02104206
First received: March 25, 2014
Last updated: April 2, 2014
Last verified: April 2014
  Purpose

The purpose of this study is to characterize the natural history of HPP in patients with Juvenile-onset HPP.


Condition
Hypophosphatasia (HPP)

Study Type: Observational
Study Design: Time Perspective: Retrospective
Official Title: A Retrospective, Non-interventional, Epidemiologic Study of the Natural History of Patients With Juvenile-onset Hypophosphatasia (HPP)

Resource links provided by NLM:


Further study details as provided by Alexion Pharmaceuticals:

Primary Outcome Measures:
  • Characterize the natural history of skeletal manifestations and growth in patients with HPP [ Time Frame: 10 years ] [ Designated as safety issue: No ]
    Pairs of x-rays will be reviewed for severity and change over time. Growth information will be collected and change in height z-score will be analyzed


Secondary Outcome Measures:
  • Characterize the natural history of growth in patients with HPP [ Time Frame: 10 years ] [ Designated as safety issue: No ]
    Growth information will be collected and change in weight z-scores will be analyzed


Estimated Enrollment: 40
Study Start Date: March 2014
Estimated Primary Completion Date: July 2014 (Final data collection date for primary outcome measure)
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 progressive damage to multiple vital organs, including destruction and deformity of bones, profound muscle weakness, seizures, impaired renal function, and respiratory failure. There are no approved disease-modifying treatments for patients with this disease. There is also limited data available on the natural course of this disease over time, particularly in patients with the juvenile-onset form.

  Eligibility

Ages Eligible for Study:   5 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Juvenile-onset HPP (≥ 6 months and ˂18 years)

Criteria

Inclusion Criteria:

  • Documented informed consent unless patient is deceased
  • 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 treatment with asfotase alfa in the ENB-006-09 study and/or currently enrolled in the ENB-008-10 study
  • Received other treatment and/or intervention to treat HPP up to 15 years old
  • Other clinically significant disease
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02104206

Contacts
Contact: Alexion (Sponsor) clinicaltrials@alxn.com

Locations
United States, Georgia
Recruiting
Atlanta, Georgia, United States
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United States, Indiana
Recruiting
Indianapolis, Indiana, United States
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United States, Massachusetts
Recruiting
Boston, Massachusetts, United States
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United States, Missouri
Recruiting
Springfield, Missouri, United States
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Recruiting
St. Louis, Missouri, United States
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United States, North Carolina
Recruiting
Durham, North Carolina, United States
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United States, Ohio
Recruiting
Cincinnati, Ohio, United States
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United States, Texas
Recruiting
Houston, Texas, United States
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United States, Washington
Recruiting
Seattle, Washington, United States
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Australia
Recruiting
Sydney, Australia
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Canada, Manitoba
Recruiting
Winnipeg, Manitoba, Canada
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France
Recruiting
Le Kremlin-Bicêtre, France
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Recruiting
Paris, France
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Recruiting
Toulouse, France
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Netherlands
Recruiting
Rotterdam, Netherlands
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Russian Federation
Recruiting
Moscow, Russian Federation
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Turkey
Recruiting
Istanbul, Turkey
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United Kingdom
Recruiting
Manchester, United Kingdom
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Recruiting
Sheffield, United Kingdom
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Sponsors and Collaborators
Alexion Pharmaceuticals
  More Information

Additional Information:
No publications provided

Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02104206     History of Changes
Other Study ID Numbers: ALX-HPP-502
Study First Received: March 25, 2014
Last Updated: April 2, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Alexion Pharmaceuticals:
Hypophosphatasia
HPP
bone disease
Soft Bones
low Alkaline Phosphatase
genetic metabolic disorder
alkaline phosphatase
tissue non-specific alkaline phosphatase
rickets
osteomalacia

Additional relevant MeSH terms:
Hypophosphatasia
Metal Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases

ClinicalTrials.gov processed this record on July 29, 2014