VALOR: A Phase 3 Study to Investigate the Efficacy and Safety of Brepocitinib in Adults With Dermatomyositis

Sponsor

Priovant Therapeutics, Inc. (Industry)

Overall Status

Recruiting

CT.gov ID

NCT05437263

Collaborator

(none)

225

Enrollment

Locations

Arms

Anticipated Duration (Months)

112.5

Patients Per Site

3.9

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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).

Condition or Disease	Intervention/Treatment	Phase
Dermatomyositis	Drug: Brepocitinib Drug: Placebo	Phase 3

Study Design

Study Type:

Interventional

Anticipated Enrollment :

225 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Triple (Participant, Investigator, Outcomes Assessor)

Primary Purpose:

Treatment

Official Title:

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy and Safety of Oral Brepocitinib in Adults With Dermatomyositis

Anticipated Study Start Date :

Jul 1, 2022

Anticipated Primary Completion Date :

Dec 1, 2024

Anticipated Study Completion Date :

Dec 1, 2024

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Brepocitinib Dose Level 1 PO QD	Drug: Brepocitinib Oral Brepocitinib
Experimental: Brepocitinib Dose Level 2 PO QD	Drug: Brepocitinib Oral Brepocitinib
Placebo Comparator: Placebo PO QD	Drug: Placebo Oral Placebo

Arm

Intervention/Treatment

Experimental: Brepocitinib Dose Level 1 PO QD

Drug: Brepocitinib

Oral Brepocitinib

Experimental: Brepocitinib Dose Level 2 PO QD

Drug: Brepocitinib

Oral Brepocitinib

Placebo Comparator: Placebo PO QD

Drug: Placebo

Oral Placebo

Outcome Measures

Primary Outcome Measures

Total Improvement Score (TIS) at Week 52 [52 weeks]
TIS is a composite endpoint based on improvement in the 6 Disease Activity Core Set Measure (CSM) scores and ranges from 0 to 100 (2016 European League Against Rheumatism [EULAR]/American College of Rheumatology [ACR] Myositis Response Criteria)

Secondary Outcome Measures

TIS responder rate after 52 weeks of brepocitinib administration QD, in comparison to placebo [52 weeks]
Proportion of responders, defined as achieving TIS ≥ 40 points (moderate improvement) at Week 52
Time to use of rescue medication through 52 weeks of brepocitinib administration QD, in comparison to placebo [52 weeks]
Time to use of rescue medication through Week 52
MMT-8 after 52 weeks of brepocitinib administration QD, in comparison to placebo [52 weeks]
Change from baseline in MMT-8 score at Week 52

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 74 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

A diagnosis of dermatomyositis according to 2017 EULAR/ACR Classification Criteria for Idiopathic Inflammatory Myopathies
Adult subjects (18-74 years old)
Active muscle and skin disease at screening and baseline
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 screening (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 and cardiovascular disease
Participants with a high risk for herpes zoster reactivation
Participants with active or recent infections