BridgeMD
RAPAMALYMPH protocol
EU CTIS ID: 2024-517769-17-00
What this study is testing
To determine the response rate to treatment with Rapamycin, by evaluating the reduction in volume of cervico-facial lymphatic malformations 3 months after obtaining the therapeutic plateau. A response will be considered positive if the reduction in volume is greater than 1/5th of the initial lesion volume.
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Minor patients aged between 1 and 18 years, regardless of sex, with a cervico-facial lymphatic malformation of micro-cystic or mixed form with a unilateral or bilateral supra-hyoid location (class II, III and IV of the De Serres classification), or any form with mediastinal extension.
- Presenting a chronic algic symptomatology, or functional symptomatology with respiratory discomfort or swallowing or phonatory disorders with a CDS score strictly below 8
- For which curative treatment is not possible or presents a major functional or vital risk. Lesions corresponding to these definitions include extensions - exceeding half of the mobile hemi-tongue or half of the hemi-base of the tongue -infiltrating more than a third of the floor of the mouth -more than half of the jugal mucosa one-third of the hemipharynx -the larynx -the trachea -both recurrent spaces -the mediastinum -the infra-temporal fossa -the pterygoid muscles -the mandibular or maxillary bone intrastructures -conjunctivo-palpebral -intra-conical orbital structures
- Karnofsky score (>10 years) or Lansky score (≤10 years) >50%
- Biological functions with the following standards: -Neutrophil count ≥1.0 x 109/L -Platelet count ≥ 100 x 109/L -Haemoglobin ≥ 8 g/dL -Total bilirubin ≤ 1.5 ULN -Transaminases < 2.5 ULN -Serum albumin ≥ 2 g/dL. -LDL cholesterol <160 mg/dL -Triglycerides < 150 mg/dL -Glomerular filtration rate (GFR) calculated using the Schwartz formula (for creatinine expressed in mg/l): GFR (ml/min/1.73 m2) = [k x height (cm)] / [creatinine (mg/l) x 8.84] > 70 ml/min/1.73 m2 With : k = 29 for premature newborns k = 40 for full-term newborns and children under 1 year of age k = 49 for children aged 2 to 12 years k = 49 for girls aged 13 to 21 k = 62 for boys aged 13 to 21
- Informed consent signed by both parents or the person(s) with parental authority
You likely can't join if
- Non-compliance with inclusion criteria
- Known allergy to peanuts or soya
- Inability to receive enlightened information
- Not covered by the social security system
- Refusal to sign the consent form
- For patients of childbearing potential, contraception other than oral contraceptives should be offered during treatment. Although the results of a single-dose interaction study with an oral contraceptive suggest the absence of a pharmacokinetic interaction, the possibility of pharmacokinetic changes that may affect the efficacy of oral contraception during prolonged treatment with Rapamune® cannot be ruled out.
See the full eligibility criteria
Who can join
- Minor patients aged between 1 and 18 years, regardless of sex, with a cervico-facial lymphatic malformation of micro-cystic or mixed form with a unilateral or bilateral supra-hyoid location (class II, III and IV of the De Serres classification), or any form with mediastinal extension.
- Presenting a chronic algic symptomatology, or functional symptomatology with respiratory discomfort or swallowing or phonatory disorders with a CDS score strictly below 8
- For which curative treatment is not possible or presents a major functional or vital risk. Lesions corresponding to these definitions include extensions - exceeding half of the mobile hemi-tongue or half of the hemi-base of the tongue -infiltrating more than a third of the floor of the mouth -more than half of the jugal mucosa one-third of the hemipharynx -the larynx -the trachea -both recurrent spaces -the mediastinum -the infra-temporal fossa -the pterygoid muscles -the mandibular or maxillary bone intrastructures -conjunctivo-palpebral -intra-conical orbital structures
- Karnofsky score (>10 years) or Lansky score (≤10 years) >50%
- Biological functions with the following standards: -Neutrophil count ≥1.0 x 109/L -Platelet count ≥ 100 x 109/L -Haemoglobin ≥ 8 g/dL -Total bilirubin ≤ 1.5 ULN -Transaminases < 2.5 ULN -Serum albumin ≥ 2 g/dL. -LDL cholesterol <160 mg/dL -Triglycerides < 150 mg/dL -Glomerular filtration rate (GFR) calculated using the Schwartz formula (for creatinine expressed in mg/l): GFR (ml/min/1.73 m2) = [k x height (cm)] / [creatinine (mg/l) x 8.84] > 70 ml/min/1.73 m2 With : k = 29 for premature newborns k = 40 for full-term newborns and children under 1 year of age k = 49 for children aged 2 to 12 years k = 49 for girls aged 13 to 21 k = 62 for boys aged 13 to 21
- Informed consent signed by both parents or the person(s) with parental authority
- Negative pregnancy test if indicated
- Person covered by a Social Security system
- For patients who have undergone surgery or sclerotherapy: inclusion possible if they meet the above criteria and after a minimum of 2 months after the procedure. Surgical procedures that do not modify the target area, such as tracheostomy or gastrostomy, do not modify the inclusion delay
What rules you out
- Non-compliance with inclusion criteria
- Known allergy to peanuts or soya
- Inability to receive enlightened information
- Not covered by the social security system
- Refusal to sign the consent form
- For patients of childbearing potential, contraception other than oral contraceptives should be offered during treatment. Although the results of a single-dose interaction study with an oral contraceptive suggest the absence of a pharmacokinetic interaction, the possibility of pharmacokinetic changes that may affect the efficacy of oral contraception during prolonged treatment with Rapamune® cannot be ruled out.
- Pregnancy or breastfeeding
- Patients, parents or legal guardians incapable of giving consent or benefiting from a legal protection regime (guardianship)
- Refusal of the child to participate
- Other immunosuppressive treatment or long-term general corticosteroid therapy
- Chronic renal failure
- Chronic liver failure
- Digestive disorders that may interfere with the absorption of Rapamycin: ulcerative gastritis, repeated vomiting, malabsorption syndrome, etc.
- Severe uncontrolled infection
- Patients requiring treatment that may induce CYP3A4 activity (rifampicin, rifabutin, carbamazepine, phenobarbital, phenytoin) or inhibit CYP3A4 activity (ketoconazole, voriconazole, itraconazole, telithromycin, clarithromycin, diltiazem, verapamil, nicardipine ; clotrimazole, fluconazole , troleandomycin , bromocriptine, cimetidine, danazol , protease inhibitors) due to changes in Rapamycin metabolism and consequent changes in Rapamycin plasma levels.
- Patients already receiving treatment with an inhibitor of the mTOR pathway
- Patients taking digestive motor stimulants such as cisapride and metoclopramide if they cannot be stopped since Pharmacokinetic interactions may be observed.
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years, 18-64 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.