Lymphatic MalformationPhase 2RAPAMALYMPHNCT03243019
Efficacy of Rapamycin in the Treatment of Cervico-facial Lymphatic Malformations
A Phase 2 study testing rapamycin, MRI and Rapamycin dosage in people with Lymphatic Malformation and Pediatric, run by University Hospital, Lille at 2 sites in France.
Trial summary
To evaluate the efficacy of Rapamycin in extended cervicofacial lymphatic malformations in pediatric patients. Rapamycin is administered oral for a 6 month period. The success rate is determined by volume reduction superior to 1/5e of the initial volume measured by MRI, impact on QOL and reduction of bleeding in case… Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Lymphatic Malformation, Pediatric
- What is being tested?
- rapamycin, MRI and Rapamycin dosage
- Type of treatment
- Pill or oral medicine, Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 28 participants
- Where
- 2 sitesFrance
- Study dates
- 25 Jun 2018to Feb 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patient from 0 to 18 years of age, presenting with poly-cystic suprahyoid or mediastinal lymphatic.
- with chronic pain or functional respiratory or swallowing impairment with a CDS score < 8
- Curative treatment is not possible or associated with a high risk of morbidity ,mortality and functional and cosmetic impairment
- Karnofsky Score (> 10 years of age) or Lansky score (≤10 years of age) > 50%
- Biology
- Neutrophils count≥1.0 x 109/L
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- Platelets count ≥ 100 x 109/L
- Hemoglobin ≥ 8 g/dL
- Bilirubin ≤ 1,5 ULN
- Transaminases < 2,5 ULN
- Serum albumin ≥ 2 g/dL.
- LDL cholesterol <160 mg/dL
- Triglycerides < 150 mg/dL
- Negative test of pregnancy if relevant
- Social security affiliation
- At least 2 months after a previous procedure on the malformation
Who cannot join
- Non-respect of inclusion criteria
- Other immunosuppressive therapy or long-term general corticosteroid therapy without a 28-day washout period
- renal failure
- Liver failure
- Digestive disease leading to rapamycin malabsorption
- uncontrolled or severe infectious disease
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- Patients requiring treatment interfering with CYP3A4 isoenzyme (rifampicin, rifabutin, carbamazepine, phenobarbital, phenytoin) or inhibiting CYP3A4 isoenzyme's activity (ketoconazole, voriconazole, itraconazole, telithromycin, clarithromycin, Diltiazem, Verapamil, nicardipine, clotrimazole, fluconazole , troleandomycin, bromocriptine, cimetidine, danazol, protease inhibitors) -patients requiring treatment by cisapride and metoclopramide
- Concomitant administration of mTOR inhibitor
- Peanuts or soya allergy
- Impossibility to receive informed consent
- Absence of social security affiliation
- refusal to sign consent
- Ongoing pregnancy or breastfeeding
- refusal to participate
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
France2 sites, 2 recruiting
- Recruiting
Hôpital Jeanne de Flandres, CHU
Lille
pierre Fayoux, MD
- Recruiting
Hu Robert Debre Aphp - Paris
Paris
About this study
Official title: Evaluation of the Efficacy of Rapamycin in the Treatment of Cervico-facial Lymphatic Malformations of Poor Prognosis
To evaluate the efficacy of Rapamycin in extended cervicofacial lymphatic malformations in pediatric patients. Rapamycin is administered oral for a 6 month period.
The success rate is determined by volume reduction superior to 1/5e of the initial volume measured by MRI, impact on QOL and reduction of bleeding in case of mucosal involvement.
- Conditions
- Lymphatic Malformation, Pediatric
- Being tested
- rapamycin, MRI, Rapamycin dosage
- Keywords
- cervico-facial, rapamycin
- Medical subject headings
- Lymphatic Abnormalities
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rapamycinDrug
oral administration
MRIDevice
cervicofacial MRI
Rapamycin dosageBiological
Biological dosage of Rapamycin level
| Study group | Role | What they receive |
|---|---|---|
| SIROLIMUS | Experimental | Not described Receives: Drug: rapamycin; Device: MRI; Biological: Rapamycin dosage |
What is being measured
The primary outcome is the main question the study is designed to answer.
Response rate to rapamycin
Volumetric assessment by MRI. A response is considered as positive if volume decrease is superior to 1/5th of the initial volume.
Measured: At 3 months
Secondary outcomes (5)
Kinetic of rapamycin response
MRI assessment of the volume
Measured: At 3, 6 and 12 months
Efficacy of rapamycin on clinical symptoms
Clinical and fiberscopy evaluation by scoring
Measured: At 3, 6 and 12 months
Pediatric Quality of Life Inventory (PedsQL 4) Scales
Assesses health-related quality of life among children with chronic and acute diseases
Measured: Baseline, at 3, 6 and 12 months
Biological response to rapamycin
biological effect of mTOR blockage by measuring pAKT, p70S6 kinase, pMEK, and VEGF C, VEGFR3
Measured: Baseline and at 6 months
Rapamycin side effects
Side effect assessment using the NCI-CTC 3.0 scale
Measured: Monthly during 1 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Aug 2017
- Study start25 Jun 2018
- Primary completionFeb 2027 (estimated)
- Study completionFeb 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Lymphatic MalformationStudies of the same condition, open and under way.
- More trials run by University Hospital, LilleThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 28 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Hospital, Lille
- Sponsor type
- University, hospital or other
- Collaborators
- Ministry of Health, France
- Responsible party
- Sponsor
- Investigators
- Pierre Fayoux, MD, Principal investigator, University Hospital, Lille
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03243019
- Sponsor's protocol ID
- 2012_67
- Other IDs
- 2013-002800-15
- Acronym
- RAPAMALYMPH
- Status last verified
- May 2026
- First submitted
- 1 Aug 2017
- Last updated
- 22 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of the Efficacy of Rapamycin in the Treatment of Cervico-facial Lymphatic Malformations of Poor Prognosis
- ClinicalTrials.gov ID
- NCT03243019
- Acronym / sponsor ID
- RAPAMALYMPH / 2012_67
- Conditions
- Lymphatic Malformation, Pediatric
- Keywords
- cervico-facial, rapamycin
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 28 participants (estimated)
- Lead sponsor
- University Hospital, LilleUniversity, hospital or other
- Collaborators
- Ministry of Health, France
- Study start
- 25 Jun 2018 (actual)
- Primary completion
- Feb 2027 (estimated)
- Study completion
- Feb 2027 (estimated)
- First posted
- 8 Aug 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To evaluate the efficacy of Rapamycin in extended cervicofacial lymphatic malformations in pediatric patients. Rapamycin is administered oral for a 6 month period.
The success rate is determined by volume reduction superior to 1/5e of the initial volume measured by MRI, impact on QOL and reduction of bleeding in case of mucosal involvement.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| SIROLIMUS | Experimental | Drug: rapamycin, Device: MRI, Biological: Rapamycin dosage |
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (16 listed)
- Patient from 0 to 18 years of age, presenting with poly-cystic suprahyoid or mediastinal lymphatic.
- with chronic pain or functional respiratory or swallowing impairment with a CDS score < 8
- Curative treatment is not possible or associated with a high risk of morbidity ,mortality and functional and cosmetic impairment
- Karnofsky Score (> 10 years of age) or Lansky score (≤10 years of age) > 50%
- Biology
Key exclusion criteria (14 listed)
- Non-respect of inclusion criteria
- Other immunosuppressive therapy or long-term general corticosteroid therapy without a 28-day washout period
- renal failure
- Liver failure
- Digestive disease leading to rapamycin malabsorption
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| rapamycinoral administration | Drug | SIROLIMUS |
| MRIcervicofacial MRI | Device | SIROLIMUS |
| Rapamycin dosageBiological dosage of Rapamycin level | Biological | SIROLIMUS |
Primary outcome
Response rate to rapamycin
Time frame: At 3 months
Volumetric assessment by MRI. A response is considered as positive if volume decrease is superior to 1/5th of the initial volume.
5 secondary outcomes
Kinetic of rapamycin response
Time frame: At 3, 6 and 12 months
Efficacy of rapamycin on clinical symptoms
Time frame: At 3, 6 and 12 months
Pediatric Quality of Life Inventory (PedsQL 4) Scales
Time frame: Baseline, at 3, 6 and 12 months
Biological response to rapamycin
Time frame: Baseline and at 6 months
Rapamycin side effects
Time frame: Monthly during 1 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 May 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Pierre Fayoux, MD principal investigator
University Hospital, Lille
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing rapamycin, MRI and Rapamycin dosage in people with Lymphatic Malformation and Pediatric, run by University Hospital, Lille at 2 sites in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 16 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 22 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Pierre Fayoux, MD (3 20 44 50 67 ext. +33, pierre.fayoux@chru-lille.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Response rate to rapamycin (time frame: At 3 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 May 2026.