Trial · NCT04128722
TOPical Sirolimus in linGUal Microcystic Lymphatic Malformation -TOPGUN
NCT04128722, Phase 2, Completed, University Hospital, Tours
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. TOPical Sirolimus in linGUal Microcystic Lymphatic Malformation -TOPGUN. Trials, record NCT04128722. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04128722
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- TOPical Sirolimus in linGUal Microcystic Lymphatic Malformation -TOPGUN
- NCT number
- NCT04128722
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 12 participants, actual
- Lead sponsor
- University Hospital, Tours
- Conditions
- Lingual Microcystic Lymphatic Malformations
- Interventions
- Sirolimus Oral Liquid Product 1mg/mL
- First posted
- 2019-10-16
- Last updated
- 2025-11-24
- Other registries' IDs
- eudract:2019-001530-33
Source ClinicalTrials.gov
Registered outcomes11
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change in Physical Global Assessment (PGA) after topical application of Sirolimus for 12 weeks | 12 weeks | The primary outcome will consist in the evaluation of global severity of the LMLM using PGA (Physical Global Assessment) 0 to 5 score, by three independent blinded experts, on monthly standardized photographs. A 1-point improvement versus baseline in PGA scale would already have a clinical relevance. Our primary analysis will focus on change in PGA after topical application of Sirolimus for 12 weeks |
| Secondary | Investigator-assessed PGA | at weeks 0, 4, 8, 12, 16, 20 and 24 | Investigator-assessed PGA (Physical Global Assessment) |
| Secondary | Assessment by the patient regarding severity of oozing, bleeding, sialorrhea, eating impairment, taste modification, aesthetic impairment, pain and global discomfort, | at weeks 0, 4, 8, 12, 16, 20 and 24. | Assessment by the patient regarding severity of oozing, bleeding, sialorrhea, eating impairment, taste modification, aesthetic impairment, pain and global discomfort, each using a numeric scale from 0 to 10 (0: clear, 10: very severe), at weeks 0, 4, 8, 12, 16, 20 and 24 |
| Secondary | Global evolution assessed by the patient | at weeks 4, 8, 12, 16, 20 and 24. | Global evolution assessed by the patient from -10 to 10 (-10 = severe worsening, 0 = no change, 10 = complete recovery), at weeks 4, 8, 12, 16, 20 and 24. |
| Secondary | Global Quality of life assessment | at baseline, at time of switch to the intervention (either week 0, week 4, week 8 or week 12) and week 24. | (DLQI or children's DLQI for minors aged 5 to 16), at baseline, time of switch to treatment and week 24. |
| Secondary | Measurements of the lesion | at baseline, at time of switch to the intervention (either week 0, week 4, week 8 or week 12) and week 24. | by the investigator, at baseline, time of switch to treatment and week 24. |
| Secondary | Time to obtain optimal results | up to 24 weeks | i.e. time from switch to treatment to time reaching the minimal PGA score |
| Secondary | Assessment of tolerance of topical sirolimus: | from the switch to intervention up to the end of the study, i.e a maximum of 24 weeks. | record of local side effects at each visit after the patient has crossed over to the intervention, up to 24 weeks |
| Secondary | General side effects | rom the switch to intervention up to the end of the study, i.e a maximum of 24 weeks. | Follow-up of general side effects |
| Secondary | Assessment of sirolimus blood passage | after 4 weeks of treatment, then 8 weeks, then every 8 weeks until week 24 | by measuring residual sirolimus blood concentration: after 4 weeks of treatment, then 8 weeks, then every 8 weeks until week 24 |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 35804404 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04128722
GET https://conductscience.org/api/trials/v1/NCT04128722/registered