Sirolimus in Cutaneous Sarcoidosis
Trial statusNot yet recruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18-74
SponsorAssistance Publique - Hôpitaux de Paris
About this trial
Sarcoidosis is a multisystemic disease of unknown etiology characterized by the presence of epithelioid granulomas without caseous necrosis in the organs involved. Sarcoidosis cutaneous lesions can be severe. There is no recommendation for the treatment of cutaneous sarcoidosis. A recent study highlights the potential efficacy of mTOR inhibitors in the treatment of sarcoidosis granulomas. The hypothesis is that sirolimus could be effective for sarcoidosis treatment, especially for cutaneous lesions.
The main objective of this study is to evaluate sirolimus efficacy on cutaneous sarcoidosis of the face.
The main evaluation criteria is the percentage of patients with a significant clinical response (relative decrease in "facial SASI" ≥ 25%) at week 16 of treatment.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Age ≥ 18 years old <75 years old (men and women)
Cutaneous sarcoidosis of the face (diagnosed according to the following criteria : compatible clinical appearance showing erythemato-purple, brownish or yellowish macules or papules or nodules and compatible histological appearance with a facial or extra facial skin biopsy confirming the diagnosis of sarcoidosis showing epithelioid and giganto-cellular granuloma without caseous necrosis) moderate to severe defined by: "Facial SASI" Score (Sarcoidosis Area and Severity Index) ≥ 2 and PGA (Physician's Global Assessment,0 to 10 scale) ≥ 5
Health insurance plan coverage
Patients who never had a systemic treatment or who had at least one classical systemic treatment failure for sarcoidosis treatment
Disqualifiers
Severe hepatic failure (Cytolysis (ALAT)> 3N and / or Cholestase (PAL)> 3N)
Allergy or intolerance to sirolimus or at one of its excipients
Allergy to peanut or soybeans
Patient with a pulmonary or hepatic graft
Trial design
Design model
Single group
Treatments tested in this trial
Sirolimus
DrugSirolimus, tablets 2 mg/day with dose adjustment of 1 to 3 mg/day for residual concentrations between 4 and 10 ng/mL 1 dose daily for 16 weeks
Treatment groups
10 Participants
are divided into 1 treatment groupGroup A: Sirolimus ArmExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Percentage of patients with a significant clinical response
Significant clinical response will be defined as a relative decrease in "facial SASI" ≥ 25% compared to baseline. Facial SASI score evaluates 4 features for each of 4 facial quadrants and the nose: erythema, induration, and desquamation, each ranging from 0 (none) to 4 (very severe), and an area score ranging from 0 (0%) to 6 (90-100%). The maximal range of modified Facial SASI scores is 0 to 72.
Time frame
at week 16Secondary outcomes
1
Percentage of patients with a good clinical response
A good clinical response will be defined as a relative decrease of "facial SASI" ≥ 50%. Facial SASI score evaluates 4 features for each of 4 facial quadrants and the nose: erythema, induration, and desquamation, each ranging from 0 (none) to 4 (very severe), and an area score ranging from 0 (0%) to 6 (90-100%). The maximal range of modified Facial SASI scores is 0 to 72.
Time frame
at week 162
Percentage of patients with complete clinical response
Complete clinical response will be defined as "facial SASI" = 0. Facial SASI score evaluates 4 features for each of 4 facial quadrants and the nose: erythema, induration, and desquamation, each ranging from 0 (none) to 4 (very severe), and an area score ranging from 0 (0%) to 6 (90-100%). The maximal range of modified Facial SASI scores is 0 to 72.
Time frame
at week 163
Percentage of patients with an improvement of their quality of dermatological life
An improvement will be defined as a decrease of Dermatology Life Quality Index (DLQI) questionnaire\> 25%. DLQI goes from 0 to 30. A lower score indicated a good quality of Life, and a higher score a worse quality of life.
Time frame
at week 164
Comparison of face facial photographs with good brightness compared to baseline
Qualitative clinical aspects of skin sarcoidosis lesions will be compared between baseline and Week 16, as an illustrative proof in addition to quantitative skin score assessment (Facial SASI)
Time frame
at week 16Locations
Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov
This trial is not recruiting at the moment. You can still explore other options: