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Registry of Patients With Hypophosphatasia

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 : April 22, 2022
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 April 22, 2022
Actual Study Start Date December 31, 2014
Estimated Primary Completion Date December 31, 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: April 15, 2022)
  • Natural History Information [ Time Frame: 1 Year ]
    To collect information on the natural history of HPP from patients of all ages, including pediatric patients and adults with HPP, regardless of age at onset.
  • Burden of Disease/Patient-reported Outcomes [ Time Frame: 1 year ]
    Age-appropriate PRO data will be collected using instruments to asses below: Pain, Motor Capacity, Functional Status/Disability, including Activities of Daily Living (ADL), Quality of Life (QoL)
  • Characterize the epidemiology of the HPP population. [ Time Frame: 1 year ]
    To characterize the epidemiology of the HPP population. Inclusion of all classifications of HPP is planned: pediatric-onset (perinatal-, infantile-, and juvenile-onset), adult-onset, benign perinatal, and odontohypophosphatasia.
  • Long-Term Safety and Effectiveness of Asfotase Alfa [ Time Frame: 1 year ]
    To collect and evaluate long-term safety and effectiveness data in HPP patients who have/are receiving treatment with asfotase alfa.
Original 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)
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 Registry of Patients With Hypophosphatasia
Official Title An Observational, Longitudinal, Prospective, Long-Term Registry Of Patients With Hypophosphatasia (HPP)
Brief Summary In this prospective, observational, long term registry patients of all ages with a diagnosis of hypophosphatasia (HPP) are followed at participating sites in multiple countries.
Detailed Description

The HPP Registry is an observational, prospective, long-term registry designed to collect data on HPP epidemiology, disease history, clinical course, symptoms and burden of disease from patients of all ages who have a diagnosis of HPP.

Evaluation of safety and effectiveness data in patients with HPP 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 *

*   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: April 15, 2022)
900
Original Estimated Enrollment
 (submitted: December 1, 2014)
500
Estimated Study Completion Date August 28, 2030
Estimated Primary Completion Date December 31, 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. Patient must have documented alkaline phosphatase (ALP) activity below the lower limit of normal for age and sex, or a documented ALPL gene mutation.

Exclusion Criteria:

Currently participating in an Alexion-sponsored clinical trial . 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,   Italy,   Poland,   Russian Federation,   Saudi Arabia,   Spain,   United Kingdom,   United States
Removed Location Countries Ireland,   Portugal
 
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
Current Responsible Party Alexion Pharmaceuticals
Original Responsible Party Same as current
Current Study Sponsor Alexion Pharmaceuticals
Original Study Sponsor Same as current
Collaborators Not Provided
Investigators Not Provided
PRS Account Alexion Pharmaceuticals
Verification Date April 2022