Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Registry for Patients With X-linked Hypophosphatemia (XLHRegistry)

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03193476
Recruitment Status : Recruiting
First Posted : June 20, 2017
Last Update Posted : April 22, 2019
Sponsor:
Information provided by (Responsible Party):
Kyowa Kirin Pharmaceutical Development Ltd

Tracking Information
First Submitted Date June 13, 2017
First Posted Date June 20, 2017
Last Update Posted Date April 22, 2019
Actual Study Start Date September 1, 2017
Estimated Primary Completion Date February 1, 2029   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: April 18, 2019)
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 [ Time Frame: 10 years ]
Original Primary Outcome Measures
 (submitted: June 17, 2017)
Change to concomitant medications [ Time Frame: 10 years ]
Change History Complete list of historical versions of study NCT03193476 on ClinicalTrials.gov Archive Site
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 for Patients With X-linked Hypophosphatemia
Official Title An International, Multicentre, Prospective, Non-interventional Observational Registry for Patients With X-linked Hypophosphatemia (XLH)
Brief Summary This is an international, multicentre, prospective, non-interventional, observational registry of patients with X-linked hypophosphatemia (XLH). The main objective of the registry is to collect data to characterise the treatment, progression and long-term outcomes of XLH in both adult and paediatric settings.
Detailed Description

Methodology

This is an international, multicentre, prospective, non-interventional, observational registry of patients with X-linked hypophosphatemia (XLH). The objectives of the registry is to collect natural history data of XLH to characterise the treatment, progression and long-term outcomes of XLH in both adult and paediatric patients.

The registry will include patients with XLH independent of treatment regimen (unless participating in an interventional clinical trial) at the time of identification. Those patients participating in an interventional clinical trial will be approached to take part in the registry when their involvement in the clinical trial has ended.

A subset of the registry data will be used to fulfil a Post-Authorisation Safety Study (PASS) as requested by the European Medicines Agency's (EMA's) Committee for Medical Products for Human Use (CHMP). Study centres which agree to participate in the PASS will be asked to solicit adverse events on enrolled patients. Not all centres are expected to participate in the PASS.

The PASS has been designated by the EMA as a non-interventional observational study. All data collected will arise from the usual clinical management of these patients. Any investigations performed for patients in the PASS (such as blood tests, ECGs, renal ultrasound scans or echocardiograms) will be at the discretion of the physicians managing the patients according to the patients' medical needs. Such investigations are not mandatory under the PASS protocol

All eligible patients at the participating clinics will be asked to participate in the registry.

  • Informed consent will be obtained from adult patients.
  • Parental informed consent for the inclusion of a child will be obtained the child's legally designated representative in line with national guidance.
  • Assent will also be sought from children of applicable age in line with national guidance. In all cases the health professional responsible for enrolling the patient into the registry will assess the appropriateness of gaining assent from an individual at their discretion.

After the patient or legally designated representative have signed the informed consent, the patient data will be recorded in the registry, including baseline, retrospective and prospective data. Data will be collected using a web-based Electronic Data Capture (EDC) system. A patient identification number will be automatically generated by the system upon enrolment.

No pre-determined follow-up requirements will apply. However, physicians will be prompted to update patient data in the registry in relation to the patients' visits to the physician. Physicians will be reminded to update the registry at 12 months after the previous visit information was entered for a patient.

For those patients who attend clinic at more frequent intervals as part of their standard care, data for these visits will be entered at the 12-month intervals with an option to add multiple dates.

This is a prospective observational registry and no additional interventions other than standard clinical practice are required by the protocol.

Study Type Observational [Patient Registry]
Study Design Observational Model: Other
Time Perspective: Other
Target Follow-Up Duration 10 Years
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Patients with XLH independent of treatment regimen
Condition X-linked Hypophosphatemia
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: June 17, 2017)
1200
Original Estimated Enrollment Same as current
Estimated Study Completion Date July 1, 2029
Estimated Primary Completion Date February 1, 2029   (Final data collection date for primary outcome measure)
Eligibility Criteria

A patient must meet the following criteria at the enrolment visit (baseline) to be eligible for inclusion into the registry

Inclusion Criteria:

  1. Patients of all ages from baseline
  2. In the opinion of the treating physician the patient has a clinical presentation, radiological, biochemical or genetic investigation results that support diagnosis of XLH
  3. Patient is not currently participating in an interventional clinical trial

A patient who meets any of the following criteria at the enrolment visit (baseline) will be excluded from the registry

Exclusion Criteria:

  1. Patient or their legally designated representative does not have the cognitive capacity to provide informed consent.
  2. Patient is currently participating in an interventional clinical trial. Patients will be approached for inclusion into the registry once their involvement in the trial ends (including the completion of all trial follow up assessments).
  3. Participation in a Compassionate Use Program, Pre-commercial Program (i.e. Named Patient Sales, Nominative ATU) or Investigator Initiated Study does not preclude a patient from participation in this registry
Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts
Contact: Clinical Trial Cordinator 0189666183 KKI-XLH-Registry@iqvia.com
Listed Location Countries United Kingdom
Removed Location Countries  
 
Administrative Information
NCT Number NCT03193476
Other Study ID Numbers XLH Registry Version 3.0
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement Not Provided
Responsible Party Kyowa Kirin Pharmaceutical Development Ltd
Study Sponsor Kyowa Kirin Pharmaceutical Development Ltd
Collaborators Not Provided
Investigators Not Provided
PRS Account Kyowa Kirin Pharmaceutical Development Ltd
Verification Date April 2019