2024-511937-37-01招募中2 期
A single arm single center exploratory observational phase II study, aimed to detect long term changes in volume of port-wine macrocheilia and color of port-wine stain in patients undergoing surgery and laser therapy during sirolimus treatment. Quality of life and efficacy and safety of sirolimus will be assessed.
Oslo University Hospital HF1 个研究点 分布在 1 个国家目标入组 20 人开始时间: 2026年10月1日最近更新:
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 20
- 试验地点
- 1
研究概览
简要总结
The primary objective is to demonstrate that treatment with sirolimus after surgery will achieve long-term volume reduction of PWM 5 years after study start.
入排标准
- 年龄范围
- 0 years 至 64 years（18-64 Years, 0-17 Years）
- 接受健康志愿者
- 否
入选标准
- •Patients of both gender with PWS in V2 and V3 area, with upper- or/and lower lip hypertrophy (PWM)
- •Patients with progression of PWS or no change in PWS during the last two months before inclusion
- •Age >5 and 25
- •Positive GNAQ R 183 gene failure test
- •Performance status ASA I or II
- •Patients with adequate bone marrow function: Hemoglobin > 10.0 g/dl, neutrophiles >1500/mm3 and platelets > 100.000/mm
- •Patients with following laboratory values: Total serum bilirubin ≤ 1.5 upper limit of normal (ULN). Serum alanine aminotransferase (ALAT) and aspartate aminotransferase ASAT) ≤ 3 x ULN. Serum creatinine 1.5 x ULN. If the serum creatinine is ≥ 1.5 x ULN, then a 24-hour Creatinine Clearance must be conducted and the result must be ≥ 60 mL/min.
- •Negative urine pregnancy test in female patients with childbearing potential.
- •Written and verbal informed consent received from patient or patients legally authorized representative.
排除标准
- •Suspected lack of compliance
- •Impairment of Gastro-Intestinal (GI) function or GI disease that may significantly alter the absorption of sirolimus (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea ≥ Grade 2, malabsorption syndromes, or small bowel resection).
- •Known hypersensitivity to drugs or metabolites from similar classes as the study medicine (i.e., everolimus and temsirolimus).
- •The oral solution contains soy and must not be provided to subjects with hypersensitivity to peanuts or soy
- •Patient with other concurrent severe and /or uncontrolled medical condition that may react negatively on the study drug.
- •Patient or patients legally authorized representative who do not understand or read Norwegian.
- •Patients with prior treatment with PI3K and/or mTOR inhibitors
- •Patients with other medication not compatible with sirolimus.
- •Patients with recent history of primary malignancy ≤ 5 years, including history of non-melanoma skin cancer, but with exception of carcinoma in situ of cervix.
- •Immunocompromised patients, including known seropositivity for HIV.
- •Severe proteinuria (> 3g proteinuria/24h).
- •Pregnancy or sexually active female patients and female partners of male patients without use of adequate contraceptive measures while on study and for up to 12 weeks after ending sirolimus treatment.
- •Breast feeding.
- •Impaired cardiac function or clinically significant cardiac diseases, including unstable angina pectoris, ventricular arrhythmia, valvular disease with documented compromise in cardiac function, documented myocardial infarction within the last 6 months, documented cardiomyopathy, family history of congenital long or short QT.
研究者
Lore Diab
Scientific
Oslo University Hospital HF
研究点 (1)
Loading locations...
相似试验
招募中
2 期
Study of Silevertinib With Temozolomide for the Treatment of Newly Diagnosed GBM With Unmethylated MGMT and EGFRvIIIGBMGliomaNCT07326566Black Diamond Therapeutics, Inc.162
尚未招募
2 期
A STUDY ON THE EFFECT OF BLOOD LETTING AND AYURVEDIC HERBAL MEDICINE IN TREATING FROZEN SHOULDERCTRI/2026/01/101046DR AKSHAYKUMAR30
招募中
不适用
A Study Comparing the Long term Results of Two Heart Stents Sirolimus and Everolimus.CTRI/2025/10/096100Anil Neerukonda Hospital150
尚未招募
不适用
Prospective Clinical evaluation of Drug Eluting Coronary StentsCTRI/2025/12/098464Multimedics LLP200
尚未招募
Unknown
Post-Marketing Study of the Sironity Drug-Coated Balloon in Patients With Coronary Artery DiseaseCTRI/2026/04/107710Sahajanand Medical Technologies Limited164