Efficacy of sirolimus in the treatment of microcystic lymphatic malformation of the tongue.
N Ghariani Fetoui, L Boussofara, R Gammoudi...
https://pubmed.ncbi.nlm.nih.gov/30980684Actively Recruiting
Phase 2
Phase 3
Age: 1Year - 18Years
All Genders
ID06673290
Different Doses of Sirolimus for the Treatment of Cystic Lymphatic Malformations
Led by West China Hospital · Updated on 2024-12-31
150
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
This research aims to compare how well different concentration levels of sirolimus work and how safe they are for treating cystic lymphatic malformation, a condition caused by abnormal growth and structure of lymphatic vessels. This disease can cause fluid buildup, swelling, pain, and organ problems in areas like the skin, soft tissues, and internal organs. The study explores whether adjusting the sirolimus dose to maintain lower blood levels might improve patient outcomes and reduce side effects such as oral mucositis. Participants will be randomly assigned to two groups: one group will receive sirolimus adjusted to keep a blood concentration between 5-8 ng/ml, and the other group will have a higher target range of 10-15 ng/ml. Both groups will receive treatment for one year. The study is single-blind and phase 2/3, meaning patients will not know which dose they receive, and the research will evaluate both doses over this period. During the study, patients will be monitored for treatment response, quality of life, and any side effects. Researchers will measure how many patients show an objective response at 6 and 12 months, track lesion changes, and assess disease-related aftereffects. Safety and adverse events will also be recorded throughout the 12-month treatment. The trial includes children aged 1 to 18 years and involves regular clinical assessments and laboratory tests to evaluate the impact and safety of different sirolimus doses.
CONDITIONS
Brief Title
Different Doses of Sirolimus for the Treatment of Cystic Lymphatic Malformations
Who Can Participate
Age: 1Year - 18Years
All Genders
Eligibility Criteria
You may qualify if you...
- Presenting a lymphatic malformation (LM) confirmed by biopsy, MRI, or clinical features
- Male or female participants
- Between 0 and 18 years of age
- LM diagnosis confirmed by local investigators and vascular anomaly experts at West China Hospital
You will not qualify if you...
- Allergy or contraindication to sirolimus or rapamycin analogs
- Use of chemotherapy, embolization, corticosteroids, propranolol, sclerotherapy, or investigational agents within 1 week before enrollment
- Major surgery within 2 weeks before enrollment
- Previous treatment with sirolimus or other mTOR inhibitors
- Active uncontrolled infection requiring intravenous antibiotics
- Severe or uncontrolled medical illnesses (e.g., diabetes, hypertension, malnutrition, liver or kidney disease, gastrointestinal ulcers)
- Impaired gastrointestinal function affecting drug absorption
- Inadequate liver function defined by specific bilirubin and enzyme levels
- Inadequate renal function by age-specific creatinine thresholds
- Low bone marrow function with absolute neutrophil count below 1 x 10^9/L
- History of malignancy within 5 years
- HIV infection or known immunodeficiency
- Need for corticosteroids, vincristine, interferon-alpha, sirolimus, or tacrolimus for other indications
- Inability to follow study procedures or give informed consent
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
1
2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Treatment
Duration - 1 year
Participants receive low or high doses of sirolimus, with doses adjusted to maintain specific plasma trough concentrations, for the treatment of cystic lymphatic malformations.
Regular visits for dose adjustment and monitoring throughout treatment
Trial Site Locations
Total: 1 location
1
West China Hospital of Sichuan University
Chengdu, Sichuan, China, 610041
Actively Recruiting
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How is the study designed?
Study Type
INTERVENTIONAL
Masking
SINGLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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Ren Cai, Xi Yang, Hao Gu...
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Yaron Gu, Deshan F Sebaratnam
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Satoru Yonekura, Takaya Komori, Yoshihiro Ishida...
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https://pubmed.ncbi.nlm.nih.gov/34524406