UMIN-CTR Clinical Trial

Public title

Study of longitudinal observation for patient with X-linked hypophosphatemic rickets/osteomalacia in collaboration with Asian partners

Acronym

SUNFLOWER Study

Scientific Title

Study of longitudinal observation for patient with X-linked hypophosphatemic rickets/osteomalacia in collaboration with Asian partners

Scientific Title:Acronym

SUNFLOWER Study

Region

Japan

Asia(except Japan)

Condition

X-linked hypophosphatemic rickets/osteomalacia

Classification by specialty

Endocrinology and Metabolism

Classification by malignancy

Others

Genomic information

NO

Narrative objectives1

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

Basic objectives2

Others

Basic objectives -Others

-

Trial characteristics_1

Trial characteristics_2

Developmental phase

Primary outcomes

Patients aged,<18years:Height,Rickets Severity Score,QOL,and motor function Patients aged >=18years:QOL and motor function

Key secondary outcomes

Patients aged <18 years:Growth velocity, lower limb deformity,biomarkers for phosphorus metabolism and bone metabolism,and fracture
Patients aged >=18 years:Biomarkers for phosphorus metabolism and bone metabolism,fracture,spinal ligament ossification,nephrocalcinosis,and renal function

Study type

Observational

Basic design

Randomization

Randomization unit

Blinding

Control

Stratification

Dynamic allocation

Institution consideration

Blocking

Concealment

No. of arms

Purpose of intervention

Type of intervention

Interventions/Control_1

Interventions/Control_2

Interventions/Control_3

Interventions/Control_4

Interventions/Control_5

Interventions/Control_6

Interventions/Control_7

Interventions/Control_8

Interventions/Control_9

Interventions/Control_10

Age-lower limit

Not applicable

Age-upper limit

Not applicable

Gender

Male and Female

Key inclusion criteria

Patients with a diagnosis of XLH who fulfil all of the following criteria:
1. 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
2. Typical clinical findings of rickets/osteomalacia in the past or present
3. Written informed consent obtained from patients aged >=18 years or from parents or legally acceptable representatives of patients aged <18 years

Key exclusion criteria

1. Participation in any clinical study (trial) sponsored by Kyowa Kirin at the time of informed consent
2. Any patient whose participation in the study is considered inappropriate by the investigator or the subinvestigator

Target sample size

240

Name of lead principal investigator

1st name	Keiichi
Middle name
Last name	Ozono

Organization

ISEIKAI International General Hospital

Division name

Center for Promoting Treatment of Intractable Diseases

Zip code

530-0052

Address

4-14 Minamiogimachi, Kita-ku osaka-shi, Osaka 530-0052, JAPAN

TEL

0570-099166

Email

keioz@ped.med.osaka-u.ac.jp

Name of contact person

1st name	Etsuou
Middle name
Last name	Imai

Organization

SRD Co., Ltd.

Division name

Clinical Research Department

Zip code

104-0032

Address

3-4-8 Hatchobori, Chuo-ku, Tokyo 104-0032, Japan

TEL

03-5543-0302

Homepage URL

Email

khk-xlh@cro-srd.co.jp

Institute

Kyowa Kirin Co., Ltd.

Institute

Department

Personal name

Organization

Kyowa Kirin Co., Ltd.

Organization

Division

Category of Funding Organization

Profit organization

Nationality of Funding Organization

Co-sponsor

Name of secondary funder(s)

Organization

Ethical Review Board of Osaka University Hospital

Address

2-2 Yamadaoka, Suita, Osaka 565-0871, Japan

Tel

06-6210-8296

Email

rinri@hp-crc.med.osaka-u.ac.jp

Secondary IDs

NO

Study ID_1

Org. issuing International ID_1

Study ID_2

Org. issuing International ID_2

IND to MHLW

Institutions

Date of disclosure of the study information

2018

Year

04

Month

20

Day

URL releasing protocol

https://bmjopen.bmj.com/content/10/6/e036367

Publication of results

Partially published

URL related to results and publications

https://academic.oup.com/jbmrplus/article/8/8/ziae079/7690732

Number of participants that the trial has enrolled

226

Results

Results date posted

Results Delayed

Results Delay Reason

Date of the first journal publication of results

Baseline Characteristics

Participant flow

Adverse events

Outcome measures

Plan to share IPD

IPD sharing Plan description

Recruitment status

No longer recruiting

Date of protocol fixation

2018

Year

03

Month

31

Day

Date of IRB

2018

Year

03

Month

06

Day

Anticipated trial start date

2018

Year

05

Month

01

Day

Last follow-up date

2028

Year

04

Month

30

Day

Date of closure to data entry

Date trial data considered complete

Date analysis concluded

Other related information

Study design:
Prospective cohort study

Study period:
April 2018 to December 2028

Methodology:
Patients who have given written informed consent to participation in the study will be registered in the EDC. Data of routine clinical examination and observation will be entered and if patients cooperate, study-specific assessments (blood sampling, questionnaire, motor function test, and anthropometric measurement) will be performed. In case of changes of residence or hospital during the observation period, additional consent will be optionally obtained from patients to allow follow-up by the Follow-up Center.

Registered date

2018

Year

03

Month

06

Day

Last modified on

2024

Year

10

Month

10

Day

Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000036050