Topical sirolimus treatment for lingual microcystic lymphatic malformations
TOPical Sirolimus in linGUal Microcystic Lymphatic Malformation -TOPGUN
This study is testing if a topical sirolimus treatment can help people with lingual microcystic lymphatic malformations feel better and improve their symptoms.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 12 (estimated) |
| Ages | 5 Years and up |
| Sex | All |
| Sponsor | University Hospital, Tours Academic / other |
| Locations | 3 sites (Tours, Indre Et Loire and 2 other locations) |
| Trial ID | NCT04128722 on ClinicalTrials.gov |
What this trial studies
This clinical trial evaluates the effectiveness of a topical sirolimus solution in treating lingual microcystic lymphatic malformations (LMLMs) that do not require systemic treatment. The study employs a randomized, open-label, multicenter pilot design, where participants will receive either the sirolimus treatment or usual care over a 24-week period. Participants will be monitored for changes in their condition, with assessments made at various intervals throughout the study. The aim is to determine if topical sirolimus can alleviate symptoms associated with LMLMs, such as pain and functional impairments.
Who should consider this trial
Good fit: Ideal candidates for this study are individuals aged 5 years and older with lingual microcystic lymphatic malformations that do not require systemic treatment.
Not a fit: Patients with lymphatic malformations requiring ongoing systemic therapy or those who have received mTOR inhibitors or steroids recently may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve the quality of life for patients suffering from LMLMs by reducing symptoms and preventing complications.
How similar studies have performed: While topical sirolimus has shown effectiveness in treating other conditions, this specific application for LMLMs is novel and has not been extensively tested in prior studies.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Participants ≥ 5 years of age * Lingual microcystic lymphatic malformation that does not require systemic treatment, assessed by clinical examination and head-and-neck MRI imaging prior to study enrolment, with or without underlying syndromic malformation (CLAPO for instance) * Participants covered by or having the rights to social security * Written informed consent obtained from participant and participant's legal representative if participant is under 18 * Ability for participant to comply with the requirements of the study Exclusion Criteria: * Patients with a lymphatic malformation requiring a continued background therapy (involving deep organs) * Secondary lymphatic malformations (lymphangiectasia post-radiotherapy, etc) * Previous treatment with systemic or topical mTOR (mammilian target of rapamycin) inhibitors within 12 months before inclusion (half-life of oral sirolimus is 60 days in adults). * Previous treatment with oral or topical steroids within 10 days before inclusion (half-life of corticosteroids is 12-36 hours) * Immunosuppression (immunosuppressive disease or immunosuppressive treatment) * Ongoing neoplasia * Active chronic infectious disease (Hepatitis-B virus, Hepatitis-C virus, HIV, etc) * Local necrosis * Local fungal, viral (herpes simplex virus, varicella zoster virus, etc) or bacterial infection on the site of the LMLM (based on clinical examination) * Known allergy to one of the components of the sirolimus solution * Soy bean or Peanut allergy * Pregnant or breastfeeding women * Women of child-bearing potential (including teenagers) not using a reliable contraceptive method until the end of the study and three month after the end of the study or sirolimus discontinuation. * Already involved in another therapeutic trial
Where this trial is running
Tours, Indre Et Loire and 2 other locations
- Univsersity of TOURS _ Service de Dermatologie — Tours, Indre Et Loire, France (Recruiting)
- REGIONAL Hospital of ORLEANS -Service de Dermatologie — Orléans, Loiret, France (Withdrawn)
- Hospital NECKER -AP-HP - Dermatology — Paris, France (Active_not_recruiting)
Study contacts
- Study coordinator: Annabel MARUANI, MD-PhD
- Email: annabel.maruani@univ-tours.fr
- Phone: +33 247479076
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.