A Prospective Natural History Study of Patients with Intractable Venous Malformation and Klippel-Trenaunay Syndrome

Not Applicable

• Conditions: Venous malformation and Klippel-Trenaunay Syndrome

• Registration Number: JPRN-UMIN000047330
• Lead Sponsor: ARTham Therapeutics Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Completion: 2021-04-08
• Study Start: 2022-04-01
• Last Update Posted: 17 October 2023

Recruitment & Eligibility

• Status: Complete: follow-up complete
• Sex: All
• Target Recruitment: 34

Inclusion Criteria

Not provided

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).

Study & Design

• Study Type: Observational
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Change rate in the target lesion volume from baseline to Day 180

Secondary Outcome Measures

Name	Time	Method
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)