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
NCT Number: NCT05458492
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.
Trial opening soon.
Get Notified18 year–74 year
All sexes
Interventional
Phase 2
Healthy volunteers accepted: No
Only the study team can determine whether someone qualifies for participation.
Inclusion criteria
Exclusion criteria
Sirolimus, 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
Time frame: at week 16
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 16
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 16
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 16
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 16
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 16
Sarcoidosis activity for all organs will be evaluted using Extra-Pulmonary Physician Organ Severity Tool (ePOST) (from 0 (normal) to 6(severe impairment))
Time frame: at 12 months
Sarcoidosis activity for all organs will be evaluted using Extra-Pulmonary Physician Organ Severity Tool (ePOST) (from 0 (normal) to 6(severe impairment))
Time frame: at 16 weeks
Sarcoidosis activity for all organs will be evaluted using Score Sarcoidosis disease activity index (SDAI score) : it goes from 0 to 167 (the higher the score, the greater the disease activity).
Time frame: at 12 months
Sarcoidosis activity for all organs will be evaluted using Score Sarcoidosis disease activity index (SDAI score) : it goes from 0 to 167 (the higher the score, the greater the disease activity).
Time frame: at week 16
Pulmonary Sarcoidosis activity will be assessed using Abbreviated CT score to quantify disease activity in pulmonary sarcoidosis (aCTAS score) to assess pulmonary involvement. It goes from 0 to 4 (the higher the score, the greater the pulmonary disease activity).
Time frame: at 12 months
Pulmonary Sarcoidosis activity will be assessed using Abbreviated CT score to quantify disease activity in pulmonary sarcoidosis (aCTAS score) to assess pulmonary involvement. It goes from 0 to 4 (the higher the score, the greater the pulmonary disease activity).
Time frame: at week 16
Pulmonary sarcoidosis functionnal evaluation will be assessed using Functional respiratory explorations by vital capacity
Time frame: at 12 months
Pulmonary sarcoidosis functionnal evaluation will be assessed using Functional respiratory explorations by vital capacity
Time frame: at week 16
Pulmonary sarcoidosis functionnal evaluation will be assessed by walking perimeter (in meters)
Time frame: at 12 months
Pulmonary sarcoidosis functionnal evaluation will be assessed by walking perimeter (in meters)
Time frame: up to 16 weeks
Time frame: at 16 weeks
Immunohistochemical assessment will be assessed using optical microscopy
Time frame: at 16 weeks
It will be assessed using microarray (Affymetrix)
Time frame: at 16 weeks
It will be assessed using microarray (Affymetrix)
Time frame: at 16 weeks
Contact information is provided by the study sponsor or research team.
Jean-David Bouaziz, Pr
CONTACT
Matthieu Resche-Rigon
CONTACT
Assistance Publique - Hôpitaux de Paris
Other
Cutaneous Sarcoidosis With Moderate to Severe Involvement of the Face : Multicenter Open-label Study of Oral Sirolimus Efficacy and Tolerance
Acronym: SIRIUS
OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.
View the official ClinicalTrials.gov record (opens in a new tab)This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.
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