PREDNISONE (prednisone) for Sarcoidosis | Pulmonary sarcoidosis

Inclusion criteria

• {"criterion_text":"- Aged ≥18 and ≤80 years\n- Pulmonary sarcoidosis meeting ATS 2020 AJRCCM diagnostic criteria ( (i) compatible presentation, (ii) non-necrotizing granuloma in one or more tissues, exclusion of alternative granulomatous diseases)\n- Patient presenting : - Lung activity on imaging (e.g., micronodules, nodules, ground glass, consolidations, septal lines) - And FVC ≤ 80% pred and/or ≥10% absolute decline in FVC from the best FVC or FVC ≤ 90% pred with significant physical performance deterioration in the last year - And one or more respiratory symptom(s) among the following: cough, dyspnea, chest pain\n- Effective contraception for women of childbearing potential until 8 months post-treatment\n- Informed and signed consent\n- Social security coverage"}

Exclusion criteria

• {"criterion_text":"- Severe sarcoidosis requiring immediate and urgent results and/or high-dose corticosteroids (severe/active neurological (except facial nerve involvement), cardiac, ophthalmic (except local treatment-responsive form), laryngeal, renal involvement, severe hypercalcemia, severe hepatic)\n- Glucose-6-Phosphate Dehydrogenase deficiency (acute hemodialysis risk)\n- HIV positivity, active Hepatitis B or C\n- Systemic corticosteroid or immunosuppressant use for at least 7 days in the previous 3 months inclusion/randomization\n- History of hydroxychloroquine treatment for sarcoidosis\n- Current pregnancy\n- Breastfeeding\n- Inability to respond to questionnaires despite aid\n- Patient under guardianship or curatorship\n- Patient under judicial protection\n- Participation in another interventional therapeutic trial within 6 months before inclusion\n- Cardiomypathy with heart failure\n- Other conditions affecting respiratory function: moderate to very severe COPD, uncontrolled asthma, obesity (BMI >30), fibrotic pulmonary disease, pulmonary neoplasia\n- Hydroxychloroquine contraindications (hypersensitivity, severe retinal or cataract, unilateral blindness, myasthenia gravis, QTc prolongation, concurrent QT-prolonging drugs)\n- Tamoxifen use\n- Renal insufficiency with clearance <60ml/min\n- History of retinopathy or maculopathy\n- Corticosteroid contraindications (hypersensitivity, infections, evolving virosis, glaucoma, uncontrolled psychosis, live vaccines, uncontrolled diabetes, uncontrolled hypertension)\n- Acute porphyria risk"}

Primary endpoints

• {"endpoint_text":"- The difference in forced vital capacity (FVC) percentage of predicted value between baseline and 6 months","definition_or_measurement_approach":"Difference in forced vital capacity (FVC) expressed as percentage of predicted value between inclusion (baseline) and month 6 (change in FVC % predicted from baseline to 6 months)."}

Secondary endpoints

• {"endpoint_text":"- General Quality of Life: Short Form 36 item (SF-36) questionnaire","definition_or_measurement_approach":"Measured using the SF-36 questionnaire."}
• {"endpoint_text":"- Respiratory Quality of Life: St George's Respiratory Questionnaire (SGRQ)","definition_or_measurement_approach":"Measured using the St George's Respiratory Questionnaire (SGRQ)."}
• {"endpoint_text":"- Fatigue: Score Fatigue assessment scale (FAS)","definition_or_measurement_approach":"Measured using the Fatigue Assessment Scale (FAS) score."}
• {"endpoint_text":"- Hydroxychloroquine Treatment Side Effects: digestive issues (nausea, vomiting), allergic and skin disorders, ocular effects","definition_or_measurement_approach":"Assessment of reported adverse events including digestive issues (nausea, vomiting), allergic/skin disorders, and ocular effects."}
• {"endpoint_text":"- Corticosteroid Treatment Side Effects : weight, blood pressure, Cushingoid facies, infection episodes, sleep disorders (Pittsburgh Sleep Quality Index - PSQI), anxiety disorders (Hospital Anxiety and Depression Scale - HADS), fasting blood glucose, HbA1c, lipid profile (total cholesterol, TG, HDL-C, LDL-C), nutritional assessment when done as part of care","definition_or_measurement_approach":"Assessment of multiple corticosteroid-related safety measures: physical signs, infection episodes, PSQI, HADS, fasting glucose, HbA1c, full lipid profile, and nutritional assessment when available."}
• {"endpoint_text":"- Treatment Adherence: GIRERD self-questionnaire","definition_or_measurement_approach":"Adherence measured by the GIRERD self-questionnaire."}
• {"endpoint_text":"- Respiratory Function: Measure of FVC, Forced Expiratory Volume in 1 second (FEV1), Carbon Monoxide Diffusing Capacity (DLCO), oxygen saturation, 6-minute walk test (6MWT). The 3-month follow-up will be aimed at assessing the patient's early respiratory progress, as is done in routine practice","definition_or_measurement_approach":"Objective respiratory measures: FVC, FEV1, DLCO, oxygen saturation, and 6-minute walk test; 3-month visit for early respiratory assessment."}
• {"endpoint_text":"- Respiratory Symptoms: Dyspnea scores and scales: mMRC, Baseline and Transition Dyspnea Index (BDI-TDI), Dyspnea-12; Cough and Sputum Assessment Questionnaire (CASA-Q)","definition_or_measurement_approach":"Symptom assessment using dyspnea scales (mMRC, BDI-TDI, Dyspnea-12) and CASA-Q for cough/sputum."}
• {"endpoint_text":"- Activity and flares of thoracic and extrathoracic sarcoidosis : Organ involvement assessed clinically using the Wasog sarcoidosis organ assessment instrument adapted by Bickett et al. (Score form)","definition_or_measurement_approach":"Organ involvement scored clinically using the WASOG sarcoidosis organ assessment instrument adapted by Bickett et al."}
• {"endpoint_text":"- Response to treatment : disappearance/appearance/worsening/improvement/stability of organ involvement using the extra-Pulmonary physician Organ Severity Tool (ePOST)(11), Sarcoidosis-Disease Activity Index (S-DAI)(12)","definition_or_measurement_approach":"Response assessed by changes in organ involvement via ePOST and S-DAI instruments."}
• {"endpoint_text":"- Use of immunosuppressants or increased corticosteroid dose during the study","definition_or_measurement_approach":"Recorded use of additional immunosuppressants or increases in corticosteroid dosing during the study."}
• {"endpoint_text":"- Biological activity assessed by serum angiotensin-converting enzyme (ACE) levels, calcium, lymphocyte count in complete blood count","definition_or_measurement_approach":"Laboratory measures: serum ACE, calcium, and lymphocyte count on CBC."}

France

Earliest CTIS Part Ii Submission Date

14-10-2024

Latest Decision Or Authorization Date

24-11-2025

Processing Time Days

406

Number Of Sites

39

Number Of Participants

200

Primary sponsor

Full Name

Assistance Publique Hopitaux De Paris

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

France