Lenalidomide in the Treatment of Refractory Cutaneous Dermatomyositis

Safety and Efficacy of Lenalidomide in the Treatment of Refractory Cutaneous Dermatomyositis

The goal of this study is to evaluate the efficacy and safety of lenalidomide in the treatment of patients with active cutaneous DM.

Study Overview

• Status: Not yet recruiting
• Conditions: Dermatomyositis
• Intervention / Treatment: Drug: Lenalidomide

Detailed Description

Dermatomyositis (DM) are systemic immune-mediated inflammatory diseases which commonly affected the skin and musculoskeletal system. The cutaneous manifestations of DM are the most important aspect of this disease. Treatment of these cutaneous manifestations is challenging and currently no universally effective drugs exists. Lenalidomide, a second-generation derivative of thalidomide, has a role as an angiogenesis inhibitor, an antineoplastic agent and an immunomodulator. Reports on refractory cutaneous manifestations of systemic lupus erythematosus have been mostly performed. The clinical trials of lenalidomide for cutaneous DM are lacking. This is a single-centre, prospective, open-label, single-arm study with Lenalidomide 5mg/day added to the background treatment of GCs and immunosuppressants in DM to evaluate the efficacy and safety of lenalidomide in the treatment of cutaneous DM.

• Study Type: Interventional
• Enrollment (Anticipated): 10
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Lihua Duan, MD; Phone Number: 0791-86895639; Email: lh-duan@163.com
• Study Contact Backup: Name: Jiao Luo, MD; Phone Number: 0791-86895639; Email: luojiao02@163.com

Study Locations

• China
  • Jiangxi
    • Nanchang, Jiangxi, China, 330000
      • Department of Rheumatology and Clinical Immunology, Jiangxi Provincial People's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 75 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Male or non-pregnant、non-nursing female.
2. Age 18 to 75 years at the time of signing informed consent.
3. Fulfilling 2017 ACR/EULAR IIMs classification criteria for DM or ADM.
4. Disease activity with skin damage (CDASI ≥12) despite treatment with corticosteroids, immunosuppressive agents or biologics for more than 3 months.
5. Written informed consent obtained before taking part in the study.

Exclusion Criteria:

1. Pregnant women or breastfeeding mothers, Male and female patients with recent fertility requirements.
2. Skin and mucosal lesions should exclude erythema multiforme, syphilis, Sweet disease, Stevens-Johnson syndrome, acne vulgaris, herpes simplex infection, periodic granulocytopenia, and acquired immunodeficiency.
3. Severe Concomitant disease: including heart failure (≥level Ⅲ, NYHA), respiratory failure, myelosuppression (WBC<3.0×109/L or N<1.5×109/L, HGB≤85g/L, PLT<100×109/L), peripheral neuropathy.
4. Acute severe infections such as sepsis and cellulitis, active hepatitis B or C virus infection, active tuberculosis, and history of a positive test for, or any clinical suspicion of, human immunodeficiency virus (HIV).
5. Patients with risk factors for myocardial infarction (including a history of thrombosis), hypercoagulability, or with history of venous thromboembolism, including deep vein thrombosis and pulmonary embolism.
6. Patients with allergies or contraindications to lenalidomide or thalidomide.
7. Uncontrolled or rapidly progressive myositis or interstitial lung disease at the discretion of the investigator which is likely to warrant escalation in therapy beyond permitted background medications.
8. Recurrent or chronic bacterial, viral, fungal, mycobacterial, or other infections including HIV.
9. History of recurrent herpes zoster, disseminated (multi-dermatomal) herpes zoster, disseminated herpes simplex or ophthalmic zoster. Herpes zoster lesions within 90 days prior to screening.
10. Primary or secondary immunodeficiency.
11. Current uncontrolled renal, gastrointestinal, endocrine, pulmonary, cardiac, or neurologic disease, which, in the opinion of the investigator, might place the patient at unacceptable risk for participation in this study.
12. History of alcohol, drug, or chemical abuse within one year prior to signing the informed consent form.
13. Major surgery within 8 weeks prior to Screening or planned major surgery at any time during participation in the study.
14. Immunization with a live/attenuated vaccine within 4 weeks prior to Screening.
15. History of malignant tumor within 5 years prior to screening (stable disease for more than 6 months after completion of tumor treatment program can be enrolled).

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Intervention with lenalidomide; All subjects will be treated with lenalidomide 5mg/day.	Drug: Lenalidomide; All subjects will be treated with lenalidomide 5mg/day with a regular follow-up of 24 weeks.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Cutaneous Disease Activity Severity Index (CDASI) activity score	Change in Cutaneous Disease Activity Severity Index (CDASI) activity score (0-100), higher scores mean a worse outcome	24 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Cutaneous Disease Activity Severity Index (CDASI) activity score	Change in Cutaneous Disease Activity Severity Index (CDASI) activity score (0-100), higher scores mean a worse outcome	12 weeks
International Myositis Assessment & Clinical Studies Group (IMACS) Disease Activity Core Set Measures	Change in International Myositis Assessment & Clinical Studies Group (IMACS) Disease Activity Core Set Measures	12 weeks and 24 weeks
SF-36	Change in SF-36	12 weeks and 24 weeks
Dermatology Life Quality Index (DLQI)	Change in Dermatology Life Quality Index (DLQI)	12 weeks and 24 weeks
Adverse event	Adverse event monitoring	24 weeks

Collaborators and Investigators

• Sponsor: Jiangxi Provincial People's Hopital
• Investigators: Principal Investigator: Lihua Duan, MD, Jiangxi Provincial People's Hopital

Study record dates

Study Major Dates

• Study Start (Anticipated): August 1, 2022
• Primary Completion (Anticipated): August 1, 2023
• Study Completion (Anticipated): September 1, 2023

Study Registration Dates

• First Submitted: July 28, 2022
• First Submitted That Met QC Criteria: August 3, 2022
• First Posted (Actual): August 4, 2022

Study Record Updates

• Last Update Posted (Actual): August 4, 2022
• Last Update Submitted That Met QC Criteria: August 3, 2022
• Last Verified: August 1, 2022

More Information

Terms related to this study

• Keywords: Cutaneous Dermatomyositis, Lenalidomide
• Additional Relevant MeSH Terms: Nervous System Diseases; Skin Diseases; Musculoskeletal Diseases; Connective Tissue Diseases; Muscular Diseases; Neuromuscular Diseases; Polymyositis; Myositis; Dermatomyositis; Physiological Effects of Drugs; Antineoplastic Agents; Immunologic Factors; Angiogenesis Inhibitors; Angiogenesis Modulating Agents; Growth Substances; Growth Inhibitors; Lenalidomide
• Other Study ID Numbers: DLH88086

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No