A Study to Investigate the Efficacy and Safety of Brepocitinib in Adults with Dermatomyositis (VALOR)

Priovant Therapeutics

Status and phase: Active, not recruiting; Phase 3

Conditions: Dermatomyositis

Treatments: Drug: Brepocitinib; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT05437263

2022-500367-12-00 (Registry Identifier)

PVT-2201-301

Details and patient eligibility

About

This is a phase 3, multicenter, randomized, placebo-controlled, double-blind study of treatment with brepocitinib (TYK2/JAK1 inhibitor) in adults with dermatomyositis (DM). The primary objective of this study is to assess the efficacy of two dose levels of brepocitinib in comparison to placebo, as measured by differences in the Total Improvement Score (TIS). After 52 weeks of double-blind treatment, participants have the option to continue therapy in a 52 week open-label extension phase where all participants will receive brepocitinib.

Enrollment: 241 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• A diagnosis of dermatomyositis according to 2017 EULAR/ACR Classification Criteria for Idiopathic Inflammatory Myopathies
• Adult subjects (18-75 years old)
• Active muscle and skin disease at screening and baseline
• Prior therapy OR current therapy with corticosteroids, hydroxychloroquine, and/or one non-steroid immunosuppressant
• 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

---

241 participants in 3 patient groups, including a placebo group

Brepocitinib Dose Level 1 PO QD

Experimental group

Treatment:

Drug: Brepocitinib

Brepocitinib Dose Level 2 PO QD

Experimental group

Treatment:

Drug: Brepocitinib

Placebo PO QD

Placebo Comparator group

Treatment:

Drug: Placebo