UMIN-ICDS Clinical Trial
| Unique ID issued by UMIN | UMIN000062825 |
|---|---|
| Receipt number | R000071918 |
| Scientific Title | A Cross-Sectional Study on the Reasons for Treatment Interruption and Unmet Needs in PKU Patients Lost to Follow-Up |
| Date of disclosure of the study information | 2026/09/07 |
| Last modified on | 2026/09/04 14:40:23 |
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Basic information
Public title
A Survey of Reasons for Interruption of Medical Follow-up and Associated Challenges Among Patients with Phenylketonuria (PKU)
Acronym
A Survey of Reasons for Interruption of Medical Follow-up and Associated Challenges Among Patients with Phenylketonuria (PKU)
Scientific Title
A Cross-Sectional Study on the Reasons for Treatment Interruption and Unmet Needs in PKU Patients Lost to Follow-Up
Scientific Title:Acronym
A Cross-Sectional Study on the Reasons for Treatment Interruption and Unmet Needs in PKU Patients Lost to Follow-Up
Region
| Japan |
Condition
Condition
Phenylketonuria; PKU
Classification by specialty
| Endocrinology and Metabolism | Pediatrics |
Classification by malignancy
Others
Genomic information
NO
Objectives
Narrative objectives1
To identify unmet needs and challenges associated with the resumption and continuation of treatment among patients aged 15 years and older with phenylketonuria (PKU) who are not regularly attending medical institutions.
Basic objectives2
Others
Basic objectives -Others
Survey on reasons for treatment discontinuation, unmet needs, and current symptoms.
Trial characteristics_1
Trial characteristics_2
Developmental phase
Not applicable
Assessment
Primary outcomes
Results of questionnaire responses on reasons for treatment discontinuation, unmet needs, and current symptoms.
Key secondary outcomes
Base
Study type
Observational
Study design
Basic design
Randomization
Randomization unit
Blinding
Control
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment
Intervention
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
Eligibility
Age-lower limit
| 15 | years-old | <= |
Age-upper limit
| Not applicable |
Gender
Male and Female
Key inclusion criteria
1) Patients diagnosed with PKU*
2) Patients who meet either of the following criteria:
a) Patients who have not visited a medical institution for PKU management for approximately one year or longer
b) Patients who have visited a medical institution for PKU management within the past year but had not attended regular follow-up visits (approximately at least once per year) for an extended period prior to that
3) Male or female patients aged 15 years or older
4) Patients who are able to complete the survey independently
5) Patients who are able to provide voluntary informed consent or assent to participate in this study
*Based on a clinical diagnosis according to the standard of care at the time of diagnosis
Key exclusion criteria
1) Patients who are currently continuing regular outpatient visits for PKU follow-up at a frequency of approximately once per year or more
2) Patients who are currently participating in an interventional clinical trial
Target sample size
50
Research contact person
Name of lead principal investigator
| 1st name | Mika |
| Middle name | |
| Last name | Ishige |
Organization
Nihon University Hospital
Division name
Pediatrics
Zip code
101-8309
Address
1-6 Kanda-Surugadai, Chiyoda-ku, Tokyo, Japan
TEL
03-3293-1711
ishige.mika@nihon-u.ac.jp
Public contact
Name of contact person
| 1st name | Yuki |
| Middle name | |
| Last name | Murabayashi |
Organization
Mebix, Inc.
Division name
Department of Clinical Operation
Zip code
105-0001
Address
10F. Toranomon 33 Mori Building 3-8-21 Toranomon, Minato-ku, Tokyo
TEL
03-4362-4500
Homepage URL
PKU_LTFU_insight_CRA@mebix.co.jp
Sponsor or person
Institute
Nihon University Hospital
Institute
Department
Personal name
Funding Source
Organization
BioMarin Pharmaceutical Japan K.K.
Organization
Division
Category of Funding Organization
Profit organization
Nationality of Funding Organization
Other related organizations
Co-sponsor
Name of secondary funder(s)
IRB Contact (For public release)
Organization
Nihon University Hospital Certified Clinical Research Review Board
Address
1-6 Kanda-Surugadai, Chiyoda-ku, Tokyo, Japan
Tel
03-3293-1711
kotani.yasuyuki@nihon-u.ac.jp
Secondary IDs
Secondary IDs
NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW
Institutions
Institutions
日本大学病院（東京都）、広島大学病院（広島県）、久留米大学病院（福岡県）、医誠会法人医誠会 医誠会国際総合病院（大阪府）、大阪公立大学医学部附属病院（大阪府）、福井大学医学部附属病院（福井県）、徳島大学病院（徳島県）、順天堂大学（東京都）
Other administrative information
Date of disclosure of the study information
| 2026 | Year | 09 | Month | 07 | Day |
Related information
URL releasing protocol
Publication of results
Unpublished
Result
URL related to results and publications
Number of participants that the trial has enrolled
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
Progress
Recruitment status
Preinitiation
Date of protocol fixation
| 2026 | Year | 05 | Month | 11 | Day |
Date of IRB
Anticipated trial start date
| 2026 | Year | 08 | Month | 01 | Day |
Last follow-up date
| 2027 | Year | 01 | Month | 31 | Day |
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
Other
Other related information
Study design:
A multicenter, cross-sectional, non-interventional quantitative study using a questionnaire survey.
Potential eligible patients will be screened, and an information sheet, informed consent form, and patient information leaflet will be sent to them from each participating study site.
Study participants will complete the questionnaire either via a web-based platform or on paper.
Management information
Registered date
| 2026 | Year | 09 | Month | 04 | Day |
Last modified on
| 2026 | Year | 09 | Month | 04 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000071918
