## Protocol Section
### Identification Module
**NCT ID:** NCT06433999

**Brief Title:** A 12-Week Open-Label Study to Investigate the Efficacy and Safety of Brepocitinib in Adults With Skin-Predominant Dermatomyositis

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

#### Organization Study ID Info
**ID:** PVT-2201-202
#### Organization
**Class:** INDUSTRY
**Full Name:** Priovant Therapeutics, Inc.
### Status Module
#### Completion Date
**Date:** 2025-01

**Type:** ESTIMATED

#### Last Update Post Date
**Date:** 2024-05-30

**Type:** ACTUAL

**Last Update Submit Date:** 2024-05-23

**Overall Status:** NOT_YET_RECRUITING

#### Primary Completion Date
**Date:** 2024-12

**Type:** ESTIMATED

#### Start Date
**Date:** 2024-06

**Type:** ESTIMATED

**Status Verified Date:** 2024-05

#### Study First Post Date
**Date:** 2024-05-30
**Type:** ACTUAL
**Study First Submit Date:** 2024-05-23
**Study First Submit QC Date:** 2024-05-23
### Sponsor Collaborators Module

#### Lead Sponsor

**Class:** INDUSTRY

**Name:** Priovant Therapeutics, Inc.

#### Responsible Party

**Type:** SPONSOR

### Oversight Module

**Is FDA Regulated Device:** False

**Is FDA Regulated Drug:** True

**Oversight Has DMC:** True

### Description Module

**Brief Summary:** 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.

### Conditions Module

**Conditions:**

- Dermatomyositis

- Dermatomyositis, Adult Type

**Keywords:**

- skin-predominant dermatomyositis

- dermatomyositis

### Design Module

#### Design Info

**Allocation:** NA

**Intervention Model:** SINGLE_GROUP

##### Masking Info

**Masking:** NONE

**Primary Purpose:** TREATMENT

#### Enrollment Info

**Count:** 5

**Type:** ESTIMATED

**Phases:**

- PHASE2

**Study Type:** INTERVENTIONAL

### Arms Interventions Module

#### Arm Group 1

**Intervention Names:**

- Drug: Brepocitinib 30 mg

**Label:** Arm 1

**Type:** EXPERIMENTAL

### Interventions

#### Intervention 1

**Arm Group Labels:**

- Arm 1

**Description:** Oral Brepocitinib 30 mg PO QD

**Name:** Brepocitinib 30 mg

**Type:** DRUG

### Outcomes Module

#### Primary Outcomes

**Measure:** The primary endpoint is the change in CDASI-A score from baseline through Week 12

**Time Frame:** 12 weeks

### Eligibility Module

**Eligibility Criteria:** 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

**Maximum Age:** 75 Years

**Minimum Age:** 18 Years

**Sex:** ALL

**Standard Ages:**

- ADULT

- OLDER_ADULT

### Contacts Locations Module

#### Central Contacts

**Contact 1:**

**Email:** lindsey.rios@priovanttx.com

**Name:** Lindsey Rios, BS

**Phone:** (860) 307-5311

**Role:** CONTACT

**Contact 2:**

**Email:** matt.cascino@priovanttx.com

**Name:** Matt Cascino, MD

**Role:** CONTACT

#### Overall Officials

**Official 1:**

**Affiliation:** Mayo Clinic

**Name:** Mangold, MD

**Role:** PRINCIPAL_INVESTIGATOR

### IPD Sharing Statement Module

**IPD Sharing:** NO

## Derived Section

### Condition Browse Module - Ancestors

- ID: D000017285

- Term: Polymyositis

- ID: D000009220

- Term: Myositis

- ID: D000009135

- Term: Muscular Diseases

- ID: D000009140

- Term: Musculoskeletal Diseases

- ID: D000009468

- Term: Neuromuscular Diseases

- ID: D000009422

- Term: Nervous System Diseases

- ID: D000003240

- Term: Connective Tissue Diseases

- ID: D000012871

- Term: Skin Diseases

### Condition Browse Module - Browse Branches

- Abbrev: BC05

- Name: Musculoskeletal Diseases

- Abbrev: BC10

- Name: Nervous System Diseases

- Abbrev: BC17

- Name: Skin and Connective Tissue Diseases

- Abbrev: All

- Name: All Conditions

- Abbrev: Rare

- Name: Rare Diseases

### Condition Browse Module - Browse Leaves

- ID: M7077

- Name: Dermatomyositis

- Relevance: HIGH

- As Found: Dermatomyositis

- ID: M19579

- Name: Polymyositis

- Relevance: LOW

- As Found: Unknown

- ID: M12172

- Name: Myositis

- Relevance: LOW

- As Found: Unknown

- ID: M12092

- Name: Muscular Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M12097

- Name: Musculoskeletal Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M12411

- Name: Neuromuscular Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M6464

- Name: Connective Tissue Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M15674

- Name: Skin Diseases

- Relevance: LOW

- As Found: Unknown

- ID: T1814

- Name: Dermatomyositis

- Relevance: HIGH

- As Found: Dermatomyositis

- ID: T4623

- Name: Polymyositis

- Relevance: LOW

- As Found: Unknown

- ID: T3001

- Name: Idiopathic Inflammatory Myopathy

- Relevance: LOW

- As Found: Unknown

### Condition Browse Module - Meshes

- ID: D000003882

- Term: Dermatomyositis

### Misc Info Module

- Version Holder: 2024-05-31
