suPERficial Slow-flow Vascular malFORMations Treated With sirolimUS

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02509468
• Date of registration: 15/07/2015
• Prospective Registration: Yes
• Primary sponsor: University Hospital, Tours
• Public title: suPERficial Slow-flow Vascular malFORMations Treated With sirolimUS PERFORMUS
• Scientific title: Treatment of Superficial Voluminous Complicated Slow-flow Vascular Malformations With Sirolimus: a Phase 2 Trial in Children Observational-phase Designed
• Date of first enrolment: September 30, 2015
• Target sample size: 63
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02509468
• 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
• Affiliation: CHRU Tours

Key inclusion & exclusion criteria

- 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



Age minimum: 6 Years
Age maximum: 18 Years
Gender: All

Health Condition(s) or Problem(s) studied

Vascular Malformation

Intervention(s)

Drug: Sirolimus

Primary Outcome(s)

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]

Secondary Outcome(s)

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]

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]

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]

Secondary ID(s)

2015-001096-43

PHRN14-AM/PERFORMUS

Source(s) of Monetary Support

Please refer to primary and secondary sponsors