Venous malformation and Klippel-Trenaunay Syndrome
Conditions
Interventions
None listed
Sponsors
ARTham Therapeutics Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
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).
Exclusion criteria
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).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change rate in the target lesion volume from baseline to Day 180 | — |
Secondary
| Measure | Time frame |
|---|---|
| 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) | — |
Countries
Japan
Contacts
Public ContactKanako Kuniyeda
ARTham Therapeutics Inc. Discovery Science
Outcome results
None listed