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An Observational, Longitudinal, Prospective, Long-Term Registry Of Patients With Hypophosphatasia (HPP)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02306720
Recruitment Status : Enrolling by invitation
First Posted : December 3, 2014
Last Update Posted : August 17, 2017
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Brief Summary:
To collect and evaluate long-term safety and effectiveness data in HPP patients who have/ are receiving treatment with asfotase alfa.

Condition or disease
Hypophosphatasia (HPP)

Detailed Description:
To collect and evaluate long-term safety and effectiveness data in HPP patients who have/ are receiving treatment with asfotase alfa.

Study Type : Observational [Patient Registry]
Estimated Enrollment : 500 participants
Observational Model: Case-Control
Time Perspective: Prospective
Target Follow-Up Duration: 5 Years
Official Title: An Observational, Longitudinal, Prospective, Long-Term Registry Of Patients With Hypophosphatasia (HPP)
Actual Study Start Date : December 2014
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : December 2019

Resource links provided by the National Library of Medicine





Primary Outcome Measures :
  1. Patient-reported Outcomes [ Time Frame: 1 year ]

    Age-appropriate PRO data will be collected using instruments to asses below:

    Pain


  2. Patient-reported Outcomes [ Time Frame: 1 year ]

    Age-appropriate PRO data will be collected using instruments to asses below:

    Motor Capacity


  3. Patient-reported Outcomes [ Time Frame: 1 year ]

    Age-appropriate PRO data will be collected using instruments to asses below:

    Functional Status/Disability, including Activities of Daily Living (ADL)


  4. Patient-reported Outcomes [ Time Frame: 1 year ]

    Age-appropriate PRO data will be collected using instruments to asses below:

    Quality of Life (QoL)




Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
Patients with Hypophosphatasia (HPP)
Criteria

Inclusion Criteria:

Male and female patients, of any age, with a confirmed diagnosis of HPP. Patient or legal representative is able to read and/or understand the informed consent and study questionnaires in the local language.

Signed informed consent and medical records release by the patient or legal representative. Patient or patient's parent/legal representative must be willing and able to give written informed consent, and the patient must be willing to give written informed assent, if appropriate and required by local regulations.

Exclusion Criteria:

Currently participating in an Alexion-sponsored clinical trial or previously received asfotase alfa. Enrollment in the Registry will not exclude a patient from enrolling in a future clinical trial.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02306720


  Hide Study Locations
Locations
United States, Arkansas
Little Rock, Arkansas, United States, 72202
United States, Colorado
Centennial, Colorado, United States, 80112
Lakewood, Colorado, United States, 80227
United States, Connecticut
Hartford, Connecticut, United States, 06106
United States, Florida
Tampa, Florida, United States, 33606
United States, Georgia
Decatur, Georgia, United States, 30033
United States, Illinois
Chicago, Illinois, United States, 60611
United States, Massachusetts
Boston, Massachusetts, United States, 02111
United States, Minnesota
Rochester, Minnesota, United States, 55905
United States, New York
Rochester, New York, United States, 14642
Syracuse, New York, United States, 13214
United States, North Carolina
Durham, North Carolina, United States, 27710
United States, Ohio
Cincinnati, Ohio, United States, 45229
Columbus, Ohio, United States, 43205
United States, Pennsylvania
Pittsburgh, Pennsylvania, United States, 15224
United States, Rhode Island
Providence, Rhode Island, United States, 02903
United States, Tennessee
Nashville, Tennessee, United States, 37212
United States, Utah
Salt Lake City, Utah, United States, 84112
Australia, New South Wales
Westmead, New South Wales, Australia
Australia, Victoria
Parkville, Victoria, Australia
Canada, Manitoba
Winnipeg, Manitoba, Canada
France
Toulouse, Haute Garonne, France
Paris cedex 14, Paris, France
Le Kremlin-Bicêtre, Île-de-France, France
Germany
Würzburg, Bayern, Germany
Essen, Nordrhein-Westfalen, Germany
Dresden, Sachsen, Germany
Ireland
Dublin, Ireland
Italy
Firenze, Italy
Genova, Italy
Milano, Italy
Pisa, Italy
Rome, Italy
San Giovanni Rotondo, Italy
Verona, Italy
Portugal
Lisboa, Portugal
Russian Federation
Kurgan, Russian Federation
Moscow, Russian Federation
Saint Petersburg, Russian Federation
Saudi Arabia
Riyadh, Saudi Arabia
Spain
Granollers, Barcelona, Spain
Barcelona, Spain
Madrid, Spain
Móstoles, Spain
Palma, Spain
Pamplona, Spain
United Kingdom
Bristol, Avon, United Kingdom
Birmingham, United Kingdom
Leicester, United Kingdom
London, United Kingdom
Manchester, United Kingdom
Newcastle upon Tyne, United Kingdom
Norwich, United Kingdom
Sheffield, United Kingdom
Stanmore, United Kingdom
Sponsors and Collaborators
Alexion Pharmaceuticals

Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02306720     History of Changes
Other Study ID Numbers: ALX-HPP-501
First Posted: December 3, 2014    Key Record Dates
Last Update Posted: August 17, 2017
Last Verified: August 2017

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