Lenalidomide plus dexamethasone for Rosai‑Dorfman disease

Efficacy and Safety of the RD Regimen (Lenalidomide, Dexamethasone) in the Treatment of Rosai-Dorfman Disease: A Prospective, Multicenter, Single-Arm Study.

This trial will try lenalidomide combined with dexamethasone to treat adults with Rosai‑Dorfman disease.

Quick facts

• Phase: PHASE2
• Study type: Interventional
• Enrollment: 40 (estimated)
• Ages: 18 Years to 80 Years
• Sex: All
• Sponsor: Cancer Institute and Hospital, Chinese Academy of Medical Sciences (other)
• Locations: 1 site (Beijing)
• Trial ID: NCT07187167 on ClinicalTrials.gov

What this trial studies

This is a phase 2 interventional trial testing an oral lenalidomide and intermittent dexamethasone regimen in adults with confirmed Rosai‑Dorfman disease. Participants receive lenalidomide 25 mg orally on days 1–21 and dexamethasone 40 mg on days 1, 8, 15, and 22 of each 28‑day cycle, for up to 12 cycles. The protocol enrolls treatment‑naive and relapsed/refractory patients with ECOG performance status ≤2 who are judged suitable by their clinicians. The study monitors treatment safety and disease response during and after the treatment period.

Who should consider this trial

Good fit: Adults aged 18–80 with a definitive diagnosis of Rosai‑Dorfman disease, ECOG ≤2, and who are treatment‑naive or relapsed/refractory and able to provide informed consent.

Not a fit: Patients with recent major surgery or radiotherapy, significant cardiac disease (including recent myocardial infarction or NYHA class 3–4 heart failure), pregnant or lactating women, those unable to use reliable contraception, or with significant liver or kidney dysfunction are unlikely to benefit or be eligible.

Why it matters

Potential benefit: If successful, this regimen could offer an effective oral treatment option that controls disease activity and reduces symptoms for some patients with Rosai‑Dorfman disease.

How similar studies have performed: Small case reports and series have suggested benefit from immunomodulatory agents like lenalidomide in Rosai‑Dorfman disease, but randomized or large prospective data are lacking.

Eligibility criteria

Inclusion Criteria:

* Definitively diagnosed adult RDD patients;
* Aged between 18 and 80 years;
* Treatment-naive or refractory/relapsed;
* ECOG performance status score ≤ 2;
* Judged by clinicians as suitable for treatment with this protocol;
* Patients or their families able to understand the study protocol and willing to participate in the study, providing written informed consent.

Exclusion Criteria:

* Subjects who have undergone major surgery within 4 weeks prior to the first dose of the study;
* Subjects who have received radiotherapy within 4 weeks prior to the first dose of the study;
* Subjects with a history of myocardial infarction within the past year;
* Patients with New York Heart Association (NYHA) Class 3 or 4 congestive heart failure, or a history of NYHA Class 3 or 4 congestive heart failure;
* Pregnant or lactating women;
* Patients who cannot strictly practice contraception after participating in the study;
* Abnormal liver and kidney function: creatinine level ≥176.8 μmol/L (2 mg/dL), transaminase and bilirubin levels more than 2 times the upper limit of normal
* Abnormal blood counts: absolute neutrophil count less than 1×10\^9/L, platelet count less than 50×10\^9/L;
* Patients or their families who cannot understand the conditions and objectives of the study;
* Any other situation where the investigator considers the patient unsuitable to participate in this trial.

Where this trial is running

Beijing

• Department of Medical Oncology, National Cancer Center/National Clinical Research Center for Cancer/Cancer Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College — Beijing, China (RECRUITING)

Study contacts

• Study coordinator: Xinxin XX Cao, doctor

Conditions: Rosai-Dorfman Disease