Study of Longitudinal Observation for Patient With X-linked Hypophosphatemic Rickets/Osteomalacia in Collaboration With Asian Partners

X-Linked Hypophosphatemia Clinical Trial

— SUNFLOWER  

Official title:

An International, Multicenter, Prospective, Longitudinal Observational Study for Patient With X-linked Hypophosphatemic Rickets/Osteomalacia

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03745521
• Other study ID #: SUNFLOWER
• Secondary ID: UMIN000031605
• Status: Active, not recruiting
• Start date: May 1, 2018
• Est. completion date: December 31, 2028

Study information

• Verified date: August 2023
• Source: Kyowa Kirin Co., Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

Through observation of patients with X-linked hypophosphatemic rickets/osteomalacia (XLH) for up to 10 years, the study intends to collect data that allow achievement of the following objectives:

1. To determine medical characteristics of the disease and the disease process

2. To determine physical and psychological burden on patients as well as economic burden

3. To assess the efficacy and safety of the treatment of the disease

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 226
• Est. completion date: December 31, 2028
• Est. primary completion date: December 31, 2028
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• Patients must meet at least one of the following:

1. Documented PHEX gene mutation

2. Documented PHEX gene mutation in at least one family member with X-linked genetic relationship

3. Documented FGF23 >30 pg/mL

• Typical clinical findings of rickets/osteomalacia

• Written informed consent obtained from patients aged >=18 years or from parents or legally acceptable representatives of patients aged <18 years

Exclusion Criteria:

• Participation in any clinical study (trial) at the time of informed consent

• Any patient whose participation in the study is considered inappropriate by the investigator or the subinvestigator

Related Conditions & MeSH terms

• Familial Hypophosphatemic Rickets
• Hypophosphatemia
• Osteomalacia
• Rickets
• X-Linked Hypophosphatemia

Intervention

Other:
• no intervention
no intervention

Locations

Country	Name	City	State
Japan	Osaka University Hospital	Osaka

Sponsors (1)

Lead Sponsor	Collaborator
Kyowa Kirin Co., Ltd.

Country where clinical trial is conducted

Japan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Height	Height in centimeters	After enrollment, patients will be observed annually or every 2 years for up to 10 years.
Primary	The 6-Minutes Walking Test	The 6-Minutes Walking Test performed according to International Guidelines, will be measured as distance in meters.	After enrollment, patients will be observed annually for up to 10 years.
Primary	Timed Up and Go Test(TUGT)	The time required for each patient to do the TUGT will be recorded: Stand up from sitting in a chair, walk 3 meters, turn around, walk back, and sit down.	After enrollment, patients will be observed every 2 years for up to 10 years.
Secondary	Fracture	Incidence of fracture of all parts	10 years
Secondary	Radiography	Radiography of the sites listed below will be performed. AP views of both knees, PA vies of both wrists, and both long legs. Sites with symptoms such as pain, sites of suspected fracture, and spine (cervical, thoracic and lumber).	After enrollment, patients will be observed annually or every 2 years for up to 10 years.
Secondary	Nephrocalcinosis	The renal ultrasound will be performed and the presence and/or progression of nephrocalcinosis will be measured.	After enrollment, patients will be observed annually or every 2 years for up to 10 years.