TOPical Sirolimus in linGUal Microcystic Lymphatic Malformation -TOPGUN

• Register: ClinicalTrials.gov
• Last refreshed on: 11 December 2023
• Main ID: NCT04128722
• Date of registration: 24/09/2019
• Prospective Registration: Yes
• Primary sponsor: University Hospital, Tours
• Public title: TOPical Sirolimus in linGUal Microcystic Lymphatic Malformation -TOPGUN TOPGUN
• Scientific title: TOPical Sirolimus in linGUal Microcystic Lymphatic Malformation -TOPGUN
• Date of first enrolment: February 14, 2020
• Target sample size: 12
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT04128722
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Single (Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

France

Contacts

• Name: Annabel MARUANI, MD-PhD

• Name: Annabel MARUANI, MD-PhD

• Name: Annabel MARUANI, MD-PhD
• Affiliation: University Hospital of TOURS;INSERM 1246 SPHERE

Key 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



Age minimum: 5 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Lingual Microcystic Lymphatic Malformations

Intervention(s)

Drug: Sirolimus Oral Liquid Product 1mg/mL

Primary Outcome(s)

Change in Physical Global Assessment (PGA) after topical application of Sirolimus for 12 weeks [Time Frame: 12 weeks]

Secondary Outcome(s)

Evaluation of biological safety [Time Frame: after 8,16 and up to 24 weeks]

Assessment by the patient regarding severity of oozing, bleeding, sialorrhea, eating impairment, taste modification, aesthetic impairment, pain and global discomfort, [Time Frame: at weeks 0, 4, 8, 12, 16, 20 and 24.]

Global Quality of life assessment [Time Frame: at baseline, at time of switch to the intervention (either week 0, week 4, week 8 or week 12) and week 24.]

Assessment of tolerance of topical sirolimus: [Time Frame: from the switch to intervention up to the end of the study, i.e a maximum of 24 weeks.]

Investigator-assessed PGA [Time Frame: at weeks 0, 4, 8, 12, 16, 20 and 24]

Time to obtain optimal results [Time Frame: up to 24 weeks]

Assessment of sirolimus blood passage [Time Frame: after 4 weeks of treatment, then 8 weeks, then every 8 weeks until week 24]

General side effects [Time Frame: rom the switch to intervention up to the end of the study, i.e a maximum of 24 weeks.]

Global evolution assessed by the patient [Time Frame: at weeks 4, 8, 12, 16, 20 and 24.]

Measurements of the lesion [Time Frame: at baseline, at time of switch to the intervention (either week 0, week 4, week 8 or week 12) and week 24.]

Secondary ID(s)

2019-001530-33

DR190041-TOPGUN

Source(s) of Monetary Support

Please refer to primary and secondary sponsors