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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 : September 17, 2019
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Tracking Information
First Submitted Date November 26, 2014
First Posted Date December 3, 2014
Last Update Posted Date September 17, 2019
Actual Study Start Date December 2014
Estimated Primary Completion Date December 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: December 1, 2014)
  • Patient-reported Outcomes [ Time Frame: 1 year ]
    Age-appropriate PRO data will be collected using instruments to asses below: Pain
  • Patient-reported Outcomes [ Time Frame: 1 year ]
    Age-appropriate PRO data will be collected using instruments to asses below: Motor Capacity
  • 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)
  • Patient-reported Outcomes [ Time Frame: 1 year ]
    Age-appropriate PRO data will be collected using instruments to asses below: Quality of Life (QoL)
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title An Observational, Longitudinal, Prospective, Long-Term Registry Of Patients With Hypophosphatasia (HPP)
Official Title An Observational, Longitudinal, Prospective, Long-Term Registry Of Patients With Hypophosphatasia (HPP)
Brief Summary To collect and evaluate long-term safety and effectiveness data in HPP patients who have/ are receiving treatment with asfotase alfa.
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]
Study Design Observational Model: Case-Control
Time Perspective: Prospective
Target Follow-Up Duration 5 Years
Biospecimen Not Provided
Sampling Method Probability Sample
Study Population Patients with Hypophosphatasia (HPP)
Condition Hypophosphatasia (HPP)
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications * Högler W, Langman C, Gomes da Silva H, Fang S, Linglart A, Ozono K, Petryk A, Rockman-Greenberg C, Seefried L, Kishnani PS. Diagnostic delay is common among patients with hypophosphatasia: initial findings from a longitudinal, prospective, global registry. BMC Musculoskelet Disord. 2019 Feb 14;20(1):80. doi: 10.1186/s12891-019-2420-8.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Enrolling by invitation
Estimated Enrollment
 (submitted: December 1, 2014)
500
Original Estimated Enrollment Same as current
Estimated Study Completion Date December 2024
Estimated Primary Completion Date December 2024   (Final data collection date for primary outcome measure)
Eligibility 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.

Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries Australia,   Canada,   France,   Germany,   Ireland,   Italy,   Portugal,   Russian Federation,   Saudi Arabia,   Spain,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT02306720
Other Study ID Numbers ALX-HPP-501
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Alexion Pharmaceuticals
Study Sponsor Alexion Pharmaceuticals
Collaborators Not Provided
Investigators Not Provided
PRS Account Alexion Pharmaceuticals
Verification Date September 2019