Lenalidomide in the Treatment of Refractory Cutaneous Dermatomyositis

Overview

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

Full Title of Study: “Safety and Efficacy of Lenalidomide in the Treatment of Refractory Cutaneous Dermatomyositis”

Study Type

• Study Type: Interventional
• Study Design
  • Allocation: N/A
  • Intervention Model: Single Group Assignment
  • Primary Purpose: Treatment
  • Masking: None (Open Label)
• Study Primary Completion Date: August 1, 2023

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.

Interventions

• Drug: Lenalidomide
  • All subjects will be treated with lenalidomide 5mg/day with a regular follow-up of 24 weeks.

Arms, Groups and Cohorts

• Experimental: Intervention with lenalidomide
  • All subjects will be treated with lenalidomide 5mg/day.

Clinical Trial Outcome Measures

Primary Measures

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

Secondary Measures

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

Participating in This Clinical Trial

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).

Gender Eligibility: All

Minimum Age: 18 Years

Maximum Age: 75 Years

Are Healthy Volunteers Accepted: No

Investigator Details

• Lead Sponsor
  • Jiangxi Provincial People’s Hopital
• Provider of Information About this Clinical Study
  • Principal Investigator: Lihua Duan, Dr – Jiangxi Provincial People’s Hopital
• Overall Official(s)
  • Lihua Duan, MD, Principal Investigator, Jiangxi Provincial People’s Hopital
• Overall Contact(s)
  • Lihua Duan, MD, 0791-86895639, lh-duan@163.com