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Trial record 10 of 24 for:    Recruiting, Not yet recruiting, Available Studies | "Rickets"

Registry for Patients With X-linked Hypophosphatemia (XLHRegistry)

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ClinicalTrials.gov Identifier: NCT03193476
Recruitment Status : Not yet recruiting
First Posted : June 20, 2017
Last Update Posted : August 10, 2017
Sponsor:
Information provided by (Responsible Party):
Kyowa Kirin Pharmaceutical Development Ltd

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.

Condition or disease
X-linked Hypophosphatemia

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.

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]
Estimated Enrollment : 1200 participants
Observational Model: Other
Time Perspective: Other
Target Follow-Up Duration: 5 Years
Official Title: An International, Multicentre, Prospective, Non-interventional Observational Registry for Patients With X-linked Hypophosphatemia (XLH)
Estimated Study Start Date : September 1, 2017
Estimated Primary Completion Date : September 1, 2022
Estimated Study Completion Date : December 1, 2022





Primary Outcome Measures :
  1. Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 [ Time Frame: 5 years ]


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:   Non-Probability Sample
Study Population
patients with XLH independent of treatment regimen
Criteria

Inclusion Criteria:

  1. Patients aged from ≥0 years of age at 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

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).

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): NCT03193476


Contacts
Contact: Julia Tonge 00 44 7557 156516 julia@medialis.co.uk

Sponsors and Collaborators
Kyowa Kirin Pharmaceutical Development Ltd

Responsible Party: Kyowa Kirin Pharmaceutical Development Ltd
ClinicalTrials.gov Identifier: NCT03193476     History of Changes
Other Study ID Numbers: XLH Registry Version 1.0
First Posted: June 20, 2017    Key Record Dates
Last Update Posted: August 10, 2017
Last Verified: August 2017

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Familial Hypophosphatemic Rickets
Rickets, Hypophosphatemic
Rickets
Hypophosphatemia
Phosphorus Metabolism Disorders
Metabolic Diseases
Bone Diseases, Metabolic
Bone Diseases
Musculoskeletal Diseases
Hypophosphatemia, Familial
Renal Tubular Transport, Inborn Errors
Kidney Diseases
Urologic Diseases
Metal Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Calcium Metabolism Disorders
Vitamin D Deficiency
Avitaminosis
Deficiency Diseases
Malnutrition
Nutrition Disorders