Lenalidomide and Dexamethasone for Rosai-Dorfman Disease

Lenalidomide and Dexamethasone for Rosai-Dorfman Disease: A Single Arm, Single Center, Prospective Phase 2 Study

Rosai-Dorfman Disease (RDD) is a rare, heterogeneous histiocytic disorder. Because of the rarity of RDD and a lack of prospective randomized trials, the treatment strategy for RDD is mostly based on retrospective study. Steroid was suggested as frontline treatment as RDD with only lymph nodes involvement. Studies showed thalidomide or lenalidomide showed some effect in recurrent/refractory skin RDD. The investigators want to analyze the efficacy and toxicity of lenalidomide combined with dexamethasone regimens in the treatment of RDD among adult patients at our hospital.

Study Overview

• Status: Recruiting
• Conditions: Rosai-Dorfman Disease
• Intervention / Treatment: Drug: lenalidomide and dexamethasone

• Study Type: Interventional
• Enrollment (Anticipated): 16
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Xinxin Cao; Phone Number: 18618315968; Email: caoxinxin@pumch.cn

Study Locations

• China
  • Beijing, China
    • Recruiting
    • Peking Union Medical College Hospital
    • Contact: Xinxin Cao

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 80 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Histologically confirmed diagnosis of RDD
• Age ≥18 years
• Eastern Cooperative Oncology Group (ECOG) performance status 0-2.
• Patients must have adequate renal, liver, and bone marrow function as defined by the following criteria:

Absolute neutrophil count ≥1500 cells per mm3 Platelet count ≥100000 cells per mm3 Creatinine clearance [according to Cockcroft formula] ≥60 mL/min. Aspartate aminotransferase and alanine aminotransferase ≤2·5×upper limit of normal [ULN], and total bilirubin ≤2·5×ULN; or ≤10×ULN in the case of known liver involvement by RDD.

• No active or untreated infection.
• No cardiac abnormalities.
• Subject provide written informed consent.
• A female is eligible to enter and participate in this study if she is of:

Non-childbearing potential including ω Any female who has had a surgical procedure rendering her incapable of becoming pregnant.

ω Subjects have experienced total cessation of menses for more than 1 year and be greater than 45 years in age.

⎫ Childbearing potential, including any female who has had a negative serum pregnancy test within 2 weeks prior to the first dose of study treatment, and agrees to use adequate contraception.

• Male subjects must use an effective barrier method of contraception during the study and for 90 days following the last course of MA if sexually active with a childbearing potential

Exclusion Criteria:

• Patients had concurrent malignancies.
• History of myocardial infarction, or unstable angina, or New York Heart Association (NYHA) Grade III-IV within 6 months prior to Day 1.
• Women who were pregnant or of childbearing potential.
• Known HIV seropositive, active hepatitis C infection, and/or hepatitis B (defined as HCV RNA≥1000 copies or HBV DNA ≥1000 copies at screening).
• Major surgical procedure within 28 days prior to the first dose of study treatment.
• Presence of uncontrolled infection.
• Evidence of active bleeding or bleeding diathesis.
• Any serious and/or unstable pre-existing medical, psychiatric, or other condition that could interfere with subject's safety, provision of informed consent, or compliance to study procedures.

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: RD; lenalidomide and dexamethasone	Drug: lenalidomide and dexamethasone; lenalidomide 25mg d1-21 and dexamethasone 20-40mg d1，8，15，22

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Event-free survival	Events were defined as a poor response to RD, reactivation after RD therapy and death from any cause.	the duration from initiation of treatment to the date of first documented event or date of death from any cause, whichever come first, assessed up to 5 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall response rate	the cumulative number of patients with either non-active disease or regressive disease (signs and symptoms were improved with no new lesions) after RD	on 12 months
Overall survival		the duration from initiation of RD treatment to the date of death or last follow-up, assessed up to 5 years

Collaborators and Investigators

• Sponsor: Peking Union Medical College Hospital
• Investigators: Study Chair: Dao-bin Zhou, Peking Union Medical College Hospital

Study record dates

Study Major Dates

• Study Start (Actual): June 8, 2021
• Primary Completion (Anticipated): December 31, 2023
• Study Completion (Anticipated): December 31, 2024

Study Registration Dates

• First Submitted: June 7, 2021
• First Submitted That Met QC Criteria: June 7, 2021
• First Posted (Actual): June 14, 2021

Study Record Updates

• Last Update Posted (Actual): June 16, 2021
• Last Update Submitted That Met QC Criteria: June 11, 2021
• Last Verified: June 1, 2021

More Information

Terms related to this study

• Keywords: lenalidomide; dexamethasone; Rosai-Dorfman Disease
• Additional Relevant MeSH Terms: Lymphatic Diseases; Histiocytosis, Non-Langerhans-Cell; Histiocytosis; Histiocytosis, Sinus; Physiological Effects of Drugs; Autonomic Agents; Peripheral Nervous System Agents; Anti-Inflammatory Agents; Antineoplastic Agents; Immunologic Factors; Antiemetics; Gastrointestinal Agents; Glucocorticoids; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Antineoplastic Agents, Hormonal; Angiogenesis Inhibitors; Angiogenesis Modulating Agents; Growth Substances; Growth Inhibitors; Dexamethasone; Lenalidomide
• Other Study ID Numbers: PUMCHRDD1

Drug and device information, study documents

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