A 12-Week Open-Label Pilot Study to Investigate the Efficacy and Safety of Oral Brepocitinib in Adults With Skin-Predominant Dermatomyositis

PVT-2201-202

This is a prospective, 12-week, open-label, single-arm study. The study population comprises individuals with refractory skin disease characteristic of dermatomyositis with no to minimal muscle involvement. After an up to 8-week Screening Period, eligible participants will receive brepocitinib 30 mg orally (PO) QD for 12 weeks.

2024-06-01

2024-12-01

2025-01-01

Not yet recruiting

Early phase I

5

Inclusion Criteria: * A diagnosis of dermatomyositis according to 2017 EULAR/ACR Classification Criteria for Idiopathic Inflammatory Myopathies * At least 2 of the following skin characteristics of dermatomyositis at screening; * Gottron's papules; * Gottron's sign; * Heliotrope rash; * Nailfold changes; * Photo distributed violaceous erythema; * History of positive dermatomyositis serology (e.g., transcriptional intermediary factor 1-lambda \[TIF1-λ\], nuclear matrix protein 2 \[NXP2\], nucleosome-remodelling deactylase complex, Mi2, melanoma differentiation antigen 5 \[MDA5\], small ubiquitin-like modifier activating enzyme 1/2, or anti-transfer ribonucleic acid \[tRNA\] synthetase). * For participants with onset of dermatomyositis symptoms within 3 years prior to screening, have a documented CT (or PET-CT) scan with contrast of the chest, abdomen, and pelvis, taken after the onset of symptoms and within 1 year prior to screening, without findings suggestive of malignancy. * Current therapy consisting of corticosteroid ≤ 20 mg/day (including a dose of 0 mg \[i.e., not taking corticosteroid\]) of prednisone or equivalent * At most, one systemic non-steroid immunomodulatory/immunosuppressive therapy * Adult subjects (18-75 years old) * Weight \> 40 kg to \< 130 kg, and with a body mass index (BMI) \< 40 kg/m2. Exclusion Criteria: * Dermatomyositis with end-stage organ involvement * Dermatomyositis with irreversible muscle involvement History of: * Any lymphoproliferative disorder * Active malignancy; * History of cancer within 5 years prior to randomization (exceptions for basal cell carcinoma, squamous cell carcinoma, ductal carcinoma in situ of the breast, carcinoma in situ of the uterine cervix, or thyroid carcinoma.) Cancer-associated dermatomyositis * Overlap myositis/connective tissue disease (except for overlap with Sjögren's syndrome) * Participants at a risk of thrombosis or cardiovascular disease * Participants with a high risk for herpes zoster reactivation * Participants with active or recent infections

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 5, 'type': 'ESTIMATED'}}

2024-05-30