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Could a transplant drug shrink Children's birthmarks?

NCT ID NCT02509468

Disease control Sponsor: University Hospital, Tours Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tested the drug sirolimus (rapamycin) in 63 children aged 6 to 18 with large, complicated slow-flow vascular malformations (abnormal blood or lymph vessels). Each child had an observation period followed by 4 to 8 months of sirolimus treatment. The main goal was to see if the malformation shrank on MRI scans. The trial is completed, and results may help decide if sirolimus is a useful treatment for these rare conditions.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
sirolimus (also known as rapamycin)
What this could lead to
If it works, this could point toward a drug treatment to shrink or control problematic vascular malformations in children, reducing pain and complications.
What could go wrong
This is a small, early-phase trial with only 63 children and no placebo group. The results may not apply to all types of malformations, and sirolimus can have side effects like mouth sores and infections.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

63 people

The number who actually took part.

Started

Sep 2015

Finished

Mar 2019

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

6 to 18 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

* Patients aged from 6 years to 18 years * With a slow-flow vascular malformation confirmed by MRI, included or not into a genetic disorder, among the following: * microcystic lymphatic malformation * mixed micro- and macrocystic malformation * venous malformation * combined lymphatic and venous malformation * Malformation voluminous and complicated (pain, functional impairment, bleeding, seepage) * Extended to the underlying subcutaneous tissue, to the fascias, the muscles and/or the underlying bone * MRI of the VM performed within 8 months * Vaccination schedule updated * Informed, written consent of the subject's parents or the 18 years old subject * Cooperative parent or subject, aware of the necessity and duration of controls so that perfect adhesion to the protocol could be expected * Subjects or subject's parents covered by or having the rights to social security. Exclusion criteria: * Slow-flow VMs which are only macrocystic lymphatic malformations * Visceral life-threatening involvement * Patients who received prior per os treatment with an mTOR inhibitor * Immunosuppression (immunosuppressive disease or immunosuppressive treatment) * Known chronic infectious disease * History of cancer in the 2 previous years * Brest feeding or pregnant women, or women on childbearing age without effective contraception, up to 12 weeks after treatment discontinuation * Known allergy to mTOR inhibitor * Concomitant treatment that inhibits or activates CYP3A4, and P-gp glycoprotein, cytotoxic drugs, antilymphocyte immunoglobulines and metoclopramide * Intolerance to fructose, intolerance or malabsorption to glucose, galactose, metabolic insufficiency in sucraseisomaltase, metabolic defect in lactase * Known allergy to peanuts or soyabean * Liver insufficiency (elevated transaminases \> 2.5 N) * Anemia with Hb \< 9 g/dl * Leukopenia \< 1000/mm3 * Thrombocytopenia \< 80 000/mm3 * Hypercholesterolemia (LDL-cholesterol ≥ 2g/l) * Patients with risk of opportunistic infections * Contraindication of MRI * Known allergy to lidocaïne * Live attenuated vaccine up to 3 months after sirolimus discontinuation * Subject already participating to a therapeutic study

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Conditions

The condition(s) this trial relates to.

microcystic lymphatic malformation mixed cystic lymphatic malformation vascular malformation venous hemangioma

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Vascular malformation

Contacts and locations

Locations

  • Consultations externes de Dermatologie, Hôpital Clocheville, CHU Tours

    Tours, 37000, France

  • Explorations Médecine Vasculaire Hôpital A. Michallon, CHU de Grenoble

    Grenoble, 38700, France

  • Service de Dermatologie, Hôpital Hôtel-Dieu, CHU Nantes

    Nantes, 44000, France

  • Service de Dermatologie, Hôpital Pontchaillou, CHU RENNES

    Rennes, 35000, France

  • Service de Dermatologie, Hôpital St Eloi, CHU Montpellier

    Montpellier, 34000, France

  • Service de Dermatologie, vénéréologie et cancérologie cutanée, Hôpital La Timone APHM

    Marseille, 13000, France

  • Service de dermatologie, APHP Necker

    Paris, 75743, France

  • Service de dermatologie, CHU Angers

    Angers, 49933, France

  • Service de dermatologie, CHU Nice

    Nice, 06202, France

  • Service de dermatologie, Hôpital Larrey, CHU Toulouse

    Toulouse, 31059, France

  • Service de dermatologie, Hôpital du Bocage, CHU Dijon

    Dijon, 21079, France

  • Service de radiologie Hôpital Femme-Mère-Enfant, Hospices Civils de Lyon

    Lyon, 69000, France

More trials for these conditions

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