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

Kyowa Kirin

Status: Active, not recruiting

Conditions: X-Linked Hypophosphatemia

Treatments: Other: no intervention

Study type: Observational

Funder types: Industry

Identifiers

NCT03745521

UMIN000031605 (Registry Identifier)

SUNFLOWER

Details and patient eligibility

About

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

Enrollment: 226 patients

Sex: All

Volunteers: No Healthy Volunteers

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

Trial design

226 participants in 1 patient group

X-linked Hypophosphatemia (XLH)

Description:

Hypophosphatemic Rickets/osteomalacia

Treatment:

Other: no intervention