Vascular MalformationPhase 2PERFORMUSNCT02509468
suPERficial Slow-flow Vascular malFORMations Treated With sirolimUS
A Phase 2 study testing Sirolimus in people with Vascular Malformation, run by University Hospital, Tours at 12 sites in France.
Trial summary
The most recent classification, adopted by International Society for the Study of Vascular Anomalies (ISSVA) in 1996, and updated in Melbourne in 2014, divides these lesions into two broad categories: vascular tumors and vascular malformations. Read more about this study
- Who is it for?
- Ages 6 to 18 years
- Conditions the study is for
- Vascular Malformation
- What is being tested?
- Sirolimus
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 63 participants
- Where
- 12 sitesFrance
- Study dates
- 30 Sep 2015to Mar 2019
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 6 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
None listed.
Who cannot join
- 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
Show all 21Show fewer
- 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
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
France12 sites
Service de dermatologie, CHU Angers
Angers, 49933
Service de dermatologie, Hôpital du Bocage, CHU Dijon
Dijon, 21079
Explorations Médecine Vasculaire Hôpital A. Michallon, CHU de Grenoble
Grenoble, 38700
Service de radiologie Hôpital Femme-Mère-Enfant, Hospices Civils de Lyon
Lyon, 69000
Service de Dermatologie, vénéréologie et cancérologie cutanée, Hôpital La Timone APHM
Marseille, 13000
Service de Dermatologie, Hôpital St Eloi, CHU Montpellier
Montpellier, 34000
Service de Dermatologie, Hôpital Hôtel-Dieu, CHU Nantes
Nantes, 44000
Service de dermatologie, CHU Nice
Nice, 06202
Service de dermatologie, APHP Necker
Paris, 75743
Service de Dermatologie, Hôpital Pontchaillou, CHU RENNES
Rennes, 35000
Service de dermatologie, Hôpital Larrey, CHU Toulouse
Toulouse, 31059
Consultations externes de Dermatologie, Hôpital Clocheville, CHU Tours
Tours, 37000
About this study
Official title: Treatment of Superficial Voluminous Complicated Slow-flow Vascular Malformations With Sirolimus: a Phase 2 Trial in Children Observational-phase Designed
The most recent classification, adopted by International Society for the Study of Vascular Anomalies (ISSVA) in 1996, and updated in Melbourne in 2014, divides these lesions into two broad categories: vascular tumors and vascular malformations. Vascular malformations (VMs) are subdivided into high-flow VM and slow-flow VM.
Slow-flow VMs consist of congenital anomalies which may involve abnormal capillaries vessels, venous vessels, lymphatic vessels or combination of several of them. They can be superficial (involving cutaneous and subcutaneous tissues) and/or may have visceral involvement. They can be limited or diffuse, and are sometimes components of genetic hypertrophic syndromes.
Read more
The diagnosis of slow-flow VMs is performed on physical examination (biopsy may be required for confirmation), and is completed with imaging (ultrasonography and magnetic resonance imaging (MRI)). Slow-flow VMs may be particularly voluminous; associated with underlying hypertrophy responsible for functional impairment; painful; associated with seepage or continuous cutaneous bleeding; complicated with visceral signs or hematologic disturbances (anemia, thrombopenia). Management requires dedicated multispecialty care. There are no guidelines for treatment, and management may include no intervention - but natural history of these VMs is progressive worsening -, compression by physical bandage, sclerotherapy, resection (when feasible),anti-inflammatory or anti-coagulation drugs.
Case reports and series have provided evidence for supporting the need for a clinical trial of sirolimus by reporting successful treatment on several children with complicated vascular anomalies. The choice of sirolimus is rational. Mammalian target of rapamycin (mTOR) is a serine/threonine kinase regulated by phosphoinositide-3-kinase involved in cell mobility, cell growth and angiogenesis. Sirolimus inhibits mTOR, which induces inhibition of angiogenesis, in particular lymphangiogenesis, which has been demonstrated in several models.
Read the detailed description
Vascular anomalies include a heterogeneous group of disorders of newborns and children. While infantile hemangioma are common (10% of infants), generally not complicated and easily managed, the majority of other vascular anomalies are rare (<2% altogether) and have no guidelines for management. The most recent classification, adopted by International Society for the Study of Vascular Anomalies (ISSVA) in 1996, divides these lesions into two broad categories: vascular tumors and vascular malformations. Vascular malformations (VMs) are subdivided into high-flow VM and slow-flow VM.
Slow-flow VMs consist of congenital anomalies which may involve abnormal capillaries vessels, venous vessels, lymphatic vessels or combination of several of them. They can be superficial (involving cutaneous and subcutaneous tissues) and/or may have visceral involvement. They can be limited or diffuse, and are sometimes components of genetic hypertrophic syndromes. They always result from defective embryologic vasculogenesis.
The diagnosis of slow-flow VMs is performed on physical examination - a biopsy may be required for confirmation -, and is completed with imaging, which includes ultrasonography and magnetic resonance imaging (MRI). Slow-flow VMs may be simple to manage or can be complicated for several reasons: they may be particularly voluminous; associated with underlying hypertrophy responsible for functional impairment; painful; associated with seepage or continuous cutaneous bleeding; complicated with visceral signs or hematologic disturbances (anemia, thrombopenia). Management requires dedicated multispecialty care. There are no guidelines for treatment, and management may include no intervention - but natural history of these VMs is progressive worsening -, compression by physical bandage, sclerotherapy, resection (when feasible), anti-inflammatory or anti-coagulation drugs.
The vast majority of literature reporting medical therapies consists of paediatric case reports, and is complicated by publication bias, inconsistent use of nomenclature and absence of clinical trials. Case reports and series have provided evidence for supporting the need for a clinical trial of sirolimus by reporting successful treatment on several children with complicated vascular anomalies. The choice of sirolimus is rational. Mammalian target of rapamycin (mTOR) is a serine/threonine kinase regulated by phosphoinositide-3-kinase involved in cell mobility, cell growth and angiogenesis. Sirolimus inhibits mTOR, which induces inhibition of angiogenesis, in particular lymphangiogenesis, which has been demonstrated in several models.
Randomized observational-phase design (Feldman et al. J Clin Epidemiol 2001;54:550-557):
- each patient will be followed during a 12-month-period
- each patient will start by an observational period and will end being treated by sirolimus
- at a random date (between month 4 and month 8), each patient will switch from the observational period to the sirolimus period Therefore, each patient will be his/her own control, as in a cross-over trial (but the difference is that the cross-over is all in one direction, from observational period to treatment period). This explains why variation in volume will be standardized by period durations.
As specified by Feldman et al, the randomized placebo-phase design is well adapted in situations where "a placebo controlled study would be perceived as being unacceptable by enrolling physicians and by patient" and "may be especially useful when highly potent therapies for rare diseases"
- Conditions
- Vascular Malformation
- Being tested
- Sirolimus
- Medical subject headings
- Vascular Malformations
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SirolimusDrug
Also called rapamune
- each patient will be followed during a 12-month-period
- each patient will start by an observational period and end being treated by sirolimus
- at a random date (between month 4 and month 8), each patient will switch from the observational period to the sirolimus period
| Study group | Role | What they receive |
|---|---|---|
| Observational | No intervention | Patients will first be included in an observational period, then, at a randomized time different from one to another, will all receive the experimental treatment (i.e. sirolimus). This design has been defined a the "randomized placebo-phase design" (Feldman et al. J Clin Epidemiol. 2001 Jun;54(6):550-7) |
| Experimental | Experimental | At a randomized date, patients will start treatment with sirolimus (beginning dose: 0.08mg/kg/day) Receives: Drug: Sirolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change of volume of the Vascular Malformation
Primary outcome will be based on the volume of the VMs on MRI. Three MRI will be performed: one at baseline (M0), one at the date of switch from the observational period to the sirolimus period (MS) and one at the end of follow-up (M12). Relative change of volume, standardized by the duration period, will define the outcome. Thus, for the observational period, the primary outcome is defined as {(VMS - V0)/V0}/(MS-M0) where V0 and VMS are the volumes assessed at baseline and month S, respectively, and (MS-M0) corresponds to the duration of the observational period. For the sirolimus period, the outcome is defined in the same way as {(V12 - VMS)/VMS}/(M12-MS), where V12 is the volume assessed at month 12 and (M12-MS) corresponds to the duration of the sirolimus period.
Interpretation of the MRI will be centralized and performed by a radiologist blinded from physical assessment and from treatment period.
Measured: at baseline, at date of switch from the observational period to the sirolimus period (between 4 and 8 month) and at 12 months
Secondary outcomes (6)
Efficacy of study treatment measured on digital photographs
Qualitative assessment of efficacy on digital photographs
Measured: inclusion, switch from the observational period to the sirolimus period (between 4 and 8 month), switch+1month, 12 month
Self assessment of efficacy of study treatment
Patient self assessment or proxy (parents) self assessments using visual analogic scale (0-10):
- Global treatment efficacy on a visual analogic scale (0-10)
- Skin complications/symptoms (seepage, bleeding, skin tension, functional impairment)
- Pain
- Quality of life by the dermatological quality of life scale (DLQI and DLQI adapted to children)
Measured: Participants will be followed during 12 months
Dermatologist's assessment of efficacy of study treatment
Dermatologist's global assessment of efficacy using a visual analogic scale (0-10)
Measured: Participants will be followed during 12 months
Efficacy of study treatment
Decrease of vascular endothelium growth factor (VEGF) and Tissue Factor (TF) plasma levels Platelet count, and fibrinogen, D-dimers, factor V levels supporting the presence and disappearance of an abnormal intravascular coagulation consumption
Measured: Participants will be followed during 12 months
Adverse events and safe adverse events will be compared
Adverse events and safe adverse events will be compared using the Mc Nemar test, if applicable. Otherwise, descriptive statistics (percentages) will be estimated.
Measured: Participants will be followed during 12 months
Organic collection of skin and blood samples
From the organic collection (including blood and skin samples), genetic analysis of several genes involved in vasculogenesis (currently TIE2 and PIK3CA) will be performed. A genotype/phenotype study will be carried out.
Measured: at 5 month or 6 month or 7 month or 8 month or 9 month after inclusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jul 2015 (estimated)
- Study start30 Sep 2015
- Primary completionMar 2019
- Study completionMar 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Vascular MalformationStudies of the same condition, open and under way.
- 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
- Randomised
- Intervention model
- Crossover
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 63 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Tours
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Annabel Maruani, MD, PhD, Principal investigator, CHRU TOURS
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02509468
- Sponsor's protocol ID
- PHRN14-AM/PERFORMUS
- Other IDs
- 2015-001096-43, 2015
- Acronym
- PERFORMUS
- Status last verified
- Sep 2019
- First submitted
- 15 Jul 2015
- Last updated
- 22 Dec 2025
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Maruani A, Tavernier E, Boccara O, Mazereeuw-Hautier J, Leducq S, Bessis D, Guibaud L, Vabres P, Carmignac V, Mallet S, Barbarot S, Chiaverini C, Droitcourt C, Bursztejn AC, Lengelle C, Woillard JB, Herbreteau D, Le Touze A, Joly A, Leaute-Labreze C, Powell J, Bourgoin H, Gissot V, Giraudeau B, Morel B. Sirolimus (Rapamycin) for Slow-Flow Malformations in Children: The Observational-Phase Randomized Clinical PERFORMUS Trial. JAMA Dermatol. 2021 Nov 1;157(11):1289-1298. doi: 10.1001/jamadermatol.2021.3459.
PubMed 34524406Maruani A, Boccara O, Bessis D, Guibaud L, Vabres P, Mazereeuw-Hautier J, Barbarot S, Chiaverini C, Blaise S, Droitcourt C, Mallet S, Martin L, Lorette G, Woillard JB, Jonville-Bera AP, Rollin J, Gruel Y, Herbreteau D, Goga D, le Touze A, Leducq S, Gissot V, Morel B, Tavernier E, Giraudeau B; Groupe de Recherche de la Societe Francaise de Dermatologie Pediatrique. Treatment of voluminous and complicated superficial slow-flow vascular malformations with sirolimus (PERFORMUS): protocol for a multicenter phase 2 trial with a randomized observational-phase design. Trials. 2018 Jun 27;19(1):340. doi: 10.1186/s13063-018-2725-1.
PubMed 29945674
Study at a glance
From the registry record, last updated by the sponsor on 22 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment of Superficial Voluminous Complicated Slow-flow Vascular Malformations With Sirolimus: a Phase 2 Trial in Children Observational-phase Designed
- ClinicalTrials.gov ID
- NCT02509468
- Acronym / sponsor ID
- PERFORMUS / PHRN14-AM/PERFORMUS
- Conditions
- Vascular Malformation
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 63 participants (actual)
- Lead sponsor
- University Hospital, ToursUniversity, hospital or other
- Study start
- 30 Sep 2015 (actual)
- Primary completion
- Mar 2019 (actual)
- Study completion
- Mar 2019 (actual)
- First posted
- 28 Jul 2015 (estimated)
- 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.
The most recent classification, adopted by International Society for the Study of Vascular Anomalies (ISSVA) in 1996, and updated in Melbourne in 2014, divides these lesions into two broad categories: vascular tumors and vascular malformations. Vascular malformations (VMs) are subdivided into high-flow VM and slow-flow VM.
Slow-flow VMs consist of congenital anomalies which may involve abnormal capillaries vessels, venous vessels, lymphatic vessels or combination of several of them. They can be superficial (involving cutaneous and subcutaneous tissues) and/or may have visceral involvement. They can be limited or diffuse, and are sometimes components of genetic hypertrophic syndromes.
The diagnosis of slow-flow VMs is performed on physical examination (biopsy may be required for confirmation), and is completed with imaging (ultrasonography and magnetic resonance imaging (MRI)). Slow-flow VMs may be particularly voluminous; associated with underlying hypertrophy responsible for functional impairment; painful; associated with seepage or continuous cutaneous bleeding; complicated with visceral signs or hematologic disturbances (anemia, thrombopenia). Management requires dedicated multispecialty care. There are no guidelines for treatment, and management may include no intervention - but natural history of these VMs is progressive worsening -, compression by physical bandage, sclerotherapy, resection (when feasible),anti-inflammatory or anti-coagulation drugs.
Case reports and series have provided evidence for supporting the need for a clinical trial of sirolimus by reporting successful treatment on several children with complicated vascular anomalies. The choice of sirolimus is rational. Mammalian target of rapamycin (mTOR) is a serine/threonine kinase regulated by phosphoinositide-3-kinase involved in cell mobility, cell growth and angiogenesis. Sirolimus inhibits mTOR, which induces inhibition of angiogenesis, in particular lymphangiogenesis, which has been demonstrated in several models.
Detailed description
Vascular anomalies include a heterogeneous group of disorders of newborns and children. While infantile hemangioma are common (10% of infants), generally not complicated and easily managed, the majority of other vascular anomalies are rare (<2% altogether) and have no guidelines for management. The most recent classification, adopted by International Society for the Study of Vascular Anomalies (ISSVA) in 1996, divides these lesions into two broad categories: vascular tumors and vascular malformations. Vascular malformations (VMs) are subdivided into high-flow VM and slow-flow VM.
Slow-flow VMs consist of congenital anomalies which may involve abnormal capillaries vessels, venous vessels, lymphatic vessels or combination of several of them. They can be superficial (involving cutaneous and subcutaneous tissues) and/or may have visceral involvement. They can be limited or diffuse, and are sometimes components of genetic hypertrophic syndromes. They always result from defective embryologic vasculogenesis.
The diagnosis of slow-flow VMs is performed on physical examination - a biopsy may be required for confirmation -, and is completed with imaging, which includes ultrasonography and magnetic resonance imaging (MRI). Slow-flow VMs may be simple to manage or can be complicated for several reasons: they may be particularly voluminous; associated with underlying hypertrophy responsible for functional impairment; painful; associated with seepage or continuous cutaneous bleeding; complicated with visceral signs or hematologic disturbances (anemia, thrombopenia). Management requires dedicated multispecialty care. There are no guidelines for treatment, and management may include no intervention - but natural history of these VMs is progressive worsening -, compression by physical bandage, sclerotherapy, resection (when feasible), anti-inflammatory or anti-coagulation drugs.
The vast majority of literature reporting medical therapies consists of paediatric case reports, and is complicated by publication bias, inconsistent use of nomenclature and absence of clinical trials. Case reports and series have provided evidence for supporting the need for a clinical trial of sirolimus by reporting successful treatment on several children with complicated vascular anomalies. The choice of sirolimus is rational. Mammalian target of rapamycin (mTOR) is a serine/threonine kinase regulated by phosphoinositide-3-kinase involved in cell mobility, cell growth and angiogenesis. Sirolimus inhibits mTOR, which induces inhibition of angiogenesis, in particular lymphangiogenesis, which has been demonstrated in several models.
Randomized observational-phase design (Feldman et al. J Clin Epidemiol 2001;54:550-557):
- each patient will be followed during a 12-month-period
- each patient will start by an observational period and will end being treated by sirolimus
- at a random date (between month 4 and month 8), each patient will switch from the observational period to the sirolimus period Therefore, each patient will be his/her own control, as in a cross-over trial (but the difference is that the cross-over is all in one direction, from observational period to treatment period). This explains why variation in volume will be standardized by period durations.
As specified by Feldman et al, the randomized placebo-phase design is well adapted in situations where "a placebo controlled study would be perceived as being unacceptable by enrolling physicians and by patient" and "may be especially useful when highly potent therapies for rare diseases"
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Observational | No intervention | –Patients will first be included in an observational period, then, at a randomized time different from one to another, will all receive the experimental treatment (i.e. sirolimus). This design has been defined a the "randomized placebo-phase design" (Feldman et al. J Clin Epidemiol. 2001 Jun;54(6):550-7) |
| Experimental | Experimental | Drug: SirolimusAt a randomized date, patients will start treatment with sirolimus (beginning dose: 0.08mg/kg/day) |
Who may qualify
- Ages
- 6 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (0 listed)
Key exclusion criteria (21 listed)
- 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
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 |
|---|---|---|
| SirolimusAlso: rapamune* each patient will be followed during a 12-month-period * each patient will start by an observational period and end being treated by sirolimus * at a random date (between month 4 and month 8), each patient will switch from the observational period to the sirolimus period | Drug | Experimental |
Primary outcome
Change of volume of the Vascular Malformation
Time frame: at baseline, at date of switch from the observational period to the sirolimus period (between 4 and 8 month) and at 12 months
Primary outcome will be based on the volume of the VMs on MRI. Three MRI will be performed: one at baseline (M0), one at the date of switch from the observational period to the sirolimus period (MS) and one at the end of follow-up (M12). Relative change of volume, standardized by the duration period, will define the outcome. Thus, for the observational period, the primary outcome is defined as {(VMS - V0)/V0}/(MS-M0) where V0 and VMS are the volumes assessed at baseline and month S, respectively, and (MS-M0) corresponds to the duration of the observational period. For the sirolimus period, the outcome is defined in the same way as {(V12 - VMS)/VMS}/(M12-MS), where V12 is the volume assessed at month 12 and (M12-MS) corresponds to the duration of the sirolimus period. Interpretation of the MRI will be centralized and performed by a radiologist blinded from physical assessment and from treatment period.
6 secondary outcomes
Efficacy of study treatment measured on digital photographs
Time frame: inclusion, switch from the observational period to the sirolimus period (between 4 and 8 month), switch+1month, 12 month
Self assessment of efficacy of study treatment
Time frame: Participants will be followed during 12 months
Dermatologist's assessment of efficacy of study treatment
Time frame: Participants will be followed during 12 months
Efficacy of study treatment
Time frame: Participants will be followed during 12 months
Adverse events and safe adverse events will be compared
Time frame: Participants will be followed during 12 months
Organic collection of skin and blood samples
Time frame: at 5 month or 6 month or 7 month or 8 month or 9 month after inclusion
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Dec 2025.
- Results publication: PubMed 34524406Maruani A, Tavernier E, Boccara O, Mazereeuw-Hautier J, Leducq S, Bessis D, Guibaud L, Vabres P, Carmignac V, Mallet S, Barbarot S, Chiaverini C, Droitcourt C, Bursztejn AC, Lengelle C, Woillard JB, Herbreteau D, Le Touze A, Joly A, Leaute-Labreze C, Powell J, Bourgoin H, Gissot V, Giraudeau B, Morel B. Sirolimus (Rapamycin) for Slow-Flow Malformations in Children: The Observational-Phase Randomized Clinical PERFORMUS Trial. JAMA Dermatol. 2021 Nov 1;157(11):1289-1298. doi: 10.1001/jamadermatol.2021.3459.
- Derived from this study: PubMed 29945674Maruani A, Boccara O, Bessis D, Guibaud L, Vabres P, Mazereeuw-Hautier J, Barbarot S, Chiaverini C, Blaise S, Droitcourt C, Mallet S, Martin L, Lorette G, Woillard JB, Jonville-Bera AP, Rollin J, Gruel Y, Herbreteau D, Goga D, le Touze A, Leducq S, Gissot V, Morel B, Tavernier E, Giraudeau B; Groupe de Recherche de la Societe Francaise de Dermatologie Pediatrique. Treatment of voluminous and complicated superficial slow-flow vascular malformations with sirolimus (PERFORMUS): protocol for a multicenter phase 2 trial with a randomized observational-phase design. Trials. 2018 Jun 27;19(1):340. doi: 10.1186/s13063-018-2725-1.
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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. This study is not recruiting at the moment.
Annabel Maruani, MD, PhD principal investigator
CHRU TOURS
12 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 Sirolimus in people with Vascular Malformation, run by University Hospital, Tours at 12 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 0 inclusion and 21 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: finished, as of the sponsor's last update on 22 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change of volume of the Vascular Malformation (time frame: at baseline, at date of switch from the observational period to the sirolimus period (between 4 and 8 month) and at 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02509468, which also lists 2 linked publications.
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 Dec 2025.