SIRIUS Sarcoidosis with moderate to severe facial skin involvement: an open multicenter study of the efficacy and safety of oral sirolimus

Phase 1

Conditions: Adult patients (men and women) (= 18 years of age) with cutaneous sarcoidosis involving the face
Therapeutic area: Diseases [C] - Immune System Diseases [C20]

Registration Number: CTIS2024-514609-76-00

Lead Sponsor: Assistance Publique Hopitaux De Paris

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 10

Inclusion Criteria

Age = 18 years < 75 years (men and wome, -Cutaneous sarcoidosis with facial involvement (diagnosed as follows: compatible clinical appearance showing brownish or yellowish erythematoviolet macules or papules or nodules and compatible histological appearance on either facial or extrafacial skin biopsy confirming the diagnosis of sarcoidosis showing epithelioid and gigantocellular granulomas without caseous necrosis) moderate to severe defined by: SASI (sarcoidosis area and severity index) facial score = 2 AND PGA (Physician's Global Assessment, scale 1 to 10) of skin = 5, Membership of a French health insurance scheme, - Patients who have failed at least one line of conventional systemic treatment, or who are naïve to systemic treatment., - For women of childbearing age (unless confirmed post-menopausal or sterile), pregnancy test with negative ßHCG. Effective contraception by contraceptive pill or intrauterine device must be used during treatment with Sirolimus and for 12 weeks after discontinuation of sirolimus., - Patients with written consent

Exclusion Criteria

-Severe hepatic impairment (cytolysis (ALT) > 3N and/or cholestasis (PAL) > 3N), -Treatment with cyclins for less than 1 month, -Treatment with topical corticosteroids or tacrolimus for less than 1 week, -At least one organ affected by sarcoidosis requiring systemic treatment other than sirolimus (oral corticosteroid or systemic per os or parenteral immunosuppressive therapy), Cholesterolemia > 3 g/L or triglyceridemia > 4 g/L, -Concomitant administration of powerful CYP3A4 inhibitors or inducers such as rifampicin, ketoconazole, voriconazole, telithromcyine, diltiazem, verapamil, erythromycin, clarythromycin, ciclosporin., Pregnancy or breast-feeding, Active infection, including tuberculosis, Uncontrolled hypertension (SBP > 150 mmHg and/or DBP > 100 mmHg), - Patients under guardianship or trusteeship, persons deprived of their liberty, under court protection, under psychiatric care, under restraint, admitted to a health or social establishment for purposes other than those of research, -cancer patient (except basal cell carcinoma of the skin or cancer in situ of the uterine cervix), -Allergy or intolerance to sirolimus or any of its excipients, -Risk of poor patient compliance., -Use of grapefruit or grapefruit juice during treatment, - patients with fructose or galactose intolerance, glucose-galactose malabsorption, sucrase-isomaltase or Lapp lactase deficiency., Peanut or soy allergy, - Lung or liver transplant patients, -Treatment with general corticosteroids or immunosuppressive agents (methotrexate, azathioprine, mycophenolate mofetil, cyclophosphamide, cyclosporine) in the month preceding inclusion., Treatment with hydroxychloroquine in the month prior to inclusion, -Treatment with intralesional corticoids for less than 3 months, -Treatment with biotherapies (anti-TNFa, anti-IL12/23, anti-IL17A) in the 3 months prior to inclusion, -Treatment with thalidomide or other imides for less than 3 months