UMIN-CTR Clinical Trial

Basic information
Public title	A Prospective Natural History Study of Patients with Intractable Venous Malformation and Klippel-Trenaunay Syndrome
Acronym	A Prospective Natural History Study of Patients with Intractable Venous Malformation and Klippel-Trenaunay Syndrome
Scientific Title	A Prospective Natural History Study of Patients with Intractable Venous Malformation and Klippel-Trenaunay Syndrome
Scientific Title:Acronym	A Prospective Natural History Study of Patients with Intractable Venous Malformation and Klippel-Trenaunay Syndrome
Region	Japan

Condition
Condition	Venous malformation and Klippel-Trenaunay Syndrome
Classification by specialty	Medicine in general Surgery in general Pediatrics Dermatology Orthopedics Radiology Plastic surgery
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	To follow the natural history of patients either with venous malformation (VM) or Klippel-Trenaunay Syndrome (KTS) for 6 months.
Basic objectives2	Others
Basic objectives -Others	To obtain a benchmark to design a clinical trials to assess safety and efficacy of novel therapeutic candidates in patients with VM and KTS.
Trial characteristics_1	Others
Trial characteristics_2	Others
Developmental phase	Not applicable

Assessment
Primary outcomes	Change rate in the target lesion volume from baseline to Day 180
Key secondary outcomes	Changes in the following endpoints from baseline to Day 180. -Severity (Appearance, function and others according to Hemangioma and Vascular Malformation Severity Classification created by Mimura group of Japanese Ministry of Health, Labor and Welfare) -Pain score (visual analog scale [VAS]) -Quality of Life (PedsQL, SF-36) -Performance Status (Lansky play-performance scale or Karnofsky Performance Status) -History of infection -Circumference of the lesion (if available) -Photograph of lesion site (if available) -Biomarkers (D-dimer, platelet count, fibrinogen)

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	Not applicable
Age-upper limit	Not applicable
Gender	Male and Female
Key inclusion criteria	1)Male or female patients at any age 2)Patients diagnosed with intractable VM or KTS following the diagnostic criteria (Japanese clinical practice guidelines for vascular anomalies 2017, Japan Intractable Disease Information Center, and Information Center for Specific Pediatric Chronic Diseases, Japan) 3)Presence of symptom(s) (e.g., pain, fever, infection, bleeding, disfigurement, etc.) 4)Patients having refractory disease (e.g., poor response to standard of care [e.g., analgesics, oral medications, compression stockings, sclerotherapy, and surgical resection]) 5)Patients who have taken or plan to take Magnetic resonance imaging (MRI) scan in their daily practice 6) Patients who have provided written consent or assent (for children, consent by their legal guardians is acceptable). Signed informed consent is obtained either from patient (aged 20 and older), patient and parent/guardian (aged from 16 to 19), or parent/guardian (aged younger than 16).
Key exclusion criteria	1)Patients for whom MRI scan is difficult or impossible to perform and whose lesions are difficult to be evaluated by MRI due to artifacts (e.g., patients who have electronic devices in their body such as cardiac pacemakers, implantable cardioverter defibrillators (ICDs), ventricular shunts, orthodontic appliances, cochlear implants neurostimulators or spinal cord stimulators, who need sedation for MRI scan but have risk to use it, and who are uncomfortable in closed or dark areas, etc.) 2)Patients whose lesion volume is difficult to measure by MRI (e.g., lesions for which the long and short diameters of the maximum cross-sectional area to be evaluated are unclear). 3)Patients who received invasive treatment including sclerotherapy or resection surgery for the target lesions within 12 weeks at obtaining consent, regardless of the classification of treatment, clinical research, or clinical trial 4)Patients who participated in any clinical trials (excluding observational studies) within 12 weeks at obtaining consent. 5)Patients who are judged by the investigator not to be eligible for the study (e.g., problems with cognitive function, comprehension, or compliance with hospital visits).
Target sample size	100

Research contact person
Name of lead principal investigator	1st name Hiroshi Middle name Last name Nagabukuro
Organization	ARTham Therapeutics Inc.
Division name	-
Zip code	2310023
Address	24-8 Yamashita-cho, Naka-ku, Yokohama, Kanagawa, Japan
TEL	0452258858
Email	hiroshi.nagabukuro@arthamther.com

Public contact
Name of contact person	1st name Kanako Middle name Last name Kuniyeda
Organization	ARTham Therapeutics Inc.
Division name	Discovery Science
Zip code	2310023
Address	24-8 Yamashita-cho, Naka-ku, Yokohama, Kanagawa, Japan
TEL	0452258858
Homepage URL
Email	kanako.kuniyeda@arthamther.com

Sponsor
Institute	ARTham Therapeutics Inc.
Institute
Department

Funding Source
Organization	Japan Agency for Medical Research and Development
Organization
Division
Category of Funding Organization	Japanese Governmental office
Nationality of Funding Organization

Other related organizations
Co-sponsor
Name of secondary funder(s)

IRB Contact (For public release)
Organization	Osaka National Hospital
Address	2-chome-1-14 Hoenzaka, Chuo-ku, Osaka, Osaka
Tel	0669421331
Email	408-chiken@mail.hosp.go.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	国立成育医療研究センター、岐阜大学医学部附属病院、神戸大学医学部附属病院、大分大学医学部附属病院、順天堂大学医学部附属浦安病院、東北大学病院、慶應義塾大学病院、東海大学医学部付属病院、北海道大学病院、大阪大学医学部附属病院、和歌山県立医科大学

Other administrative information
Date of disclosure of the study information	2022 Year 04 Month 01 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	34
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	Completed
Date of protocol fixation	2019 Year 01 Month 30 Day
Date of IRB	2019 Year 01 Month 30 Day
Anticipated trial start date	2019 Year 06 Month 19 Day
Last follow-up date	2021 Year 04 Month 08 Day
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information	To follow the natural history of patients either with venous malformation (VM) or Klippel-Trenaunay Syndrome (KTS) for 6 months.

Management information
Registered date	2022 Year 03 Month 30 Day
Last modified on	2022 Year 03 Month 30 Day

Link to view the page
URL(English)	https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000053980