Ignite Creation Date:
2025-12-24 @ 6:35 PM
Ignite Modification Date:
2025-12-29 @ 6:39 AM
Study NCT ID:
NCT07012057
Status:
WITHDRAWN
Last Update Posted:
2025-11-24
First Post:
2025-06-07
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Study of Deucravacitinib for Refractory Adults With Dermatomyositis/Juvenile Dermatomyositis
Sponsor:
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Organization:
Study Overview
Official Title:
A Phase II Open-Label Study of Deucravacitinib for Refractory Adults With Dermatomyositis/ Juvenile Dermatomyositis
Status:
WITHDRAWN
Status Verified Date:
2025-11
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Manufacturer discontinued investigational drug supply.
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Background:
Dermatomyositis (DM) and juvenile dermatomyositis (JDM) are diseases that weaken the immune system. DM and JDM can affect the muscles, skin, joints, and lungs and cause skin rashes and muscle inflammation. Symptoms include weakness, pain, fatigue, and rash. Not everyone responds to current treatments. The FDA has approved a drug called deucravacitinib to treat people with plaque psoriasis. Researchers want to find out if this drug can help people with DM or JDM, too.
Objective:
To test deucravacitinib in people with DM or JDM.
Eligibility:
People aged 18 years and older with DM or JDM.
Design:
Participants will have 9 clinic visits over 28 weeks.
Participants will be screened. They will have a physical exam with blood and urine tests. They will have a test of their heart function. They will complete a short questionnaire about their daily life, pain level, and ability to walk, eat, and do other activities.
Deucravacitinib is a pill taken by mouth twice per day at home. Participants will come to the clinic once every 4 weeks for 24 weeks while they are taking the drug. They will have a final visit 4 weeks after their last dose of the study drug. Blood and urine tests will be repeated during these visits. Each visit may take up to 6 hours.
If the drug is helping them, participants may extend their treatment beyond the first 24 weeks. Then they will visit the clinic every 3 months.
Dermatomyositis (DM) and juvenile dermatomyositis (JDM) are diseases that weaken the immune system. DM and JDM can affect the muscles, skin, joints, and lungs and cause skin rashes and muscle inflammation. Symptoms include weakness, pain, fatigue, and rash. Not everyone responds to current treatments. The FDA has approved a drug called deucravacitinib to treat people with plaque psoriasis. Researchers want to find out if this drug can help people with DM or JDM, too.
Objective:
To test deucravacitinib in people with DM or JDM.
Eligibility:
People aged 18 years and older with DM or JDM.
Design:
Participants will have 9 clinic visits over 28 weeks.
Participants will be screened. They will have a physical exam with blood and urine tests. They will have a test of their heart function. They will complete a short questionnaire about their daily life, pain level, and ability to walk, eat, and do other activities.
Deucravacitinib is a pill taken by mouth twice per day at home. Participants will come to the clinic once every 4 weeks for 24 weeks while they are taking the drug. They will have a final visit 4 weeks after their last dose of the study drug. Blood and urine tests will be repeated during these visits. Each visit may take up to 6 hours.
If the drug is helping them, participants may extend their treatment beyond the first 24 weeks. Then they will visit the clinic every 3 months.
Detailed Description:
Study Description:
Myositis is a heterogeneous family of systemic autoimmune diseases affecting adults and children that can affect the muscle, skin, lungs, joints, and/or gastrointestinal tract. Unfortunately, many patients do not respond completely to current immunosuppressive therapies. Although the pathophysiological mechanisms underlying these diseases are incompletely understood, in adult patients with dermatomyositis (DM) and juvenile dermatomyositis (JDM), a Type 1 interferon gene signature in blood, skin, and muscle correlates with disease activity. Furthermore, recent reports suggest that non-selective inhibitors of the JAK/STAT pathway may be effective in treating patients with refractory DM and JDM. In this study, we will test our hypothesis that inhibition of JAK/STAT pathway using the selective oral TYK2 inhibitor, deucravacitinib, will be efficacious and safe in adult patients with active, treatment-refractory DM/JDM by performing a 24-week, phase II, open-label treatment trial.
At the conclusion of the Treatment Period of the study, all subjects will have the option to receive deucravacitinib in the Extension Portion of the trial.
Objectives:
Primary Objective:
To obtain preliminary data regarding the efficacy of a selective TYK2 inhibitor, deucravacitinib, in adult patients with active, treatment-refractory DM/JDM, as defined by the 2016 ACR/EULAR Criteria for Minimal, Moderate, and Major Clinical Response in Adults with Dermatomyositis and Juvenile Dermatomyositis.
Exploratory Objectives:
* To obtain preliminary data regarding the efficacy of deucravacitinib to improve skin disease activity in adult patients with active, treatment-refractory JDM/DM, as defined by the Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI).
* To obtain preliminary data regarding the frequency and incidence of adverse events in treatment-refractory DM/JDM patients treated with deucravacitinib.
Endpoints:
Primary endpoint:
The number of DM/JDM patients achieving at least a "minimal clinical response" using the total improvement score as defined in the 2016 ACR/EULAR Criteria for Minimal, Moderate, and Major Clinical Response in Adult Dermatomyositis and Polymyositis and Juvenile Dermatomyositis at 24 weeks of treatment.
Exploratory endpoints:
* The number of DM/JDM patients with significantly improved skin disease activity (\>14 point decrease out of 100) as measured by the CDASI at 24 weeks of treatment.
* The frequency and incidence of treatment-related adverse events reported and observed. (Time Frame: At 28 weeks)
Myositis is a heterogeneous family of systemic autoimmune diseases affecting adults and children that can affect the muscle, skin, lungs, joints, and/or gastrointestinal tract. Unfortunately, many patients do not respond completely to current immunosuppressive therapies. Although the pathophysiological mechanisms underlying these diseases are incompletely understood, in adult patients with dermatomyositis (DM) and juvenile dermatomyositis (JDM), a Type 1 interferon gene signature in blood, skin, and muscle correlates with disease activity. Furthermore, recent reports suggest that non-selective inhibitors of the JAK/STAT pathway may be effective in treating patients with refractory DM and JDM. In this study, we will test our hypothesis that inhibition of JAK/STAT pathway using the selective oral TYK2 inhibitor, deucravacitinib, will be efficacious and safe in adult patients with active, treatment-refractory DM/JDM by performing a 24-week, phase II, open-label treatment trial.
At the conclusion of the Treatment Period of the study, all subjects will have the option to receive deucravacitinib in the Extension Portion of the trial.
Objectives:
Primary Objective:
To obtain preliminary data regarding the efficacy of a selective TYK2 inhibitor, deucravacitinib, in adult patients with active, treatment-refractory DM/JDM, as defined by the 2016 ACR/EULAR Criteria for Minimal, Moderate, and Major Clinical Response in Adults with Dermatomyositis and Juvenile Dermatomyositis.
Exploratory Objectives:
* To obtain preliminary data regarding the efficacy of deucravacitinib to improve skin disease activity in adult patients with active, treatment-refractory JDM/DM, as defined by the Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI).
* To obtain preliminary data regarding the frequency and incidence of adverse events in treatment-refractory DM/JDM patients treated with deucravacitinib.
Endpoints:
Primary endpoint:
The number of DM/JDM patients achieving at least a "minimal clinical response" using the total improvement score as defined in the 2016 ACR/EULAR Criteria for Minimal, Moderate, and Major Clinical Response in Adult Dermatomyositis and Polymyositis and Juvenile Dermatomyositis at 24 weeks of treatment.
Exploratory endpoints:
* The number of DM/JDM patients with significantly improved skin disease activity (\>14 point decrease out of 100) as measured by the CDASI at 24 weeks of treatment.
* The frequency and incidence of treatment-related adverse events reported and observed. (Time Frame: At 28 weeks)
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| 002167-AR | None | None | View |