Lenalidomide for Adult Histiocyte Disorders

NCT02523040 | Active Not Recruiting Phase 2 Results DMC

• 1 other identifier: 15-197
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

This research study is studying a chemotherapy drug Lenalidomide as a possible treatment for one of three histiocyte disorders: Langerhans cell histiocytosis (LCH), Erdheim-Chester disease (ECD), or histiocytic sarcoma (HS).

Conditions

Langerhans Cell Histiocytosis (LCH); Histiocytoses Erdheim-chester Disease; Histiocytic Sarcoma (HS)

Keywords

Langerhans cell histiocytosis (LCH); Erdheim-Chester disease (ECD); Histiocytic Sarcoma (HS)

Outcome Measures

Primary Outcomes (1)

• Overall Response Rate (ORR)

  Proportion of participants achieving either complete resolution of all signs or symptoms which is non-active disease (NAD) status or regression of signs or symptoms along with no new lesions which is better active-disease (AD) status per Histiocyte Society criteria any time on treatment.

  Disease assessed every 3 cycles on treatment up do 12 cycles (approximately 12 months).

Secondary Outcomes (6)

• 12-months Progression-free Survival (PFS)

  Disease assessed on treatment every 3 cycles and in long-term follow-up up to the earlier of 36 months (every 3 months for 2 years then every 6 months) or start of new anti-cancer therapy. Relevant for this endpoint was12-months estimate.

• 12-months Overall Survival (OS)

  12 Months

• Incidence of Grade 3-4 Toxicity

  Up to 12 months on treatment.

• Urine Cell Free DNA for BRAF

  12 Months

• Percent Change in Serum TNF-alpha Levels on Therapy From Baseline up to 12 Cycles

  Measured at baseline, day 1 of cycles 3, 6 ad 12 cycles and within 28 days post-treatment end (up to 13 months).

Study Arms (1)

Lenalidomide

EXPERIMENTAL

After the screening procedures confirm participation in the research study. \- Lenalidomide Oral, Daily for 21 days of each cycle

Drug: Lenalidomide

Interventions

Lenalidomide DRUG

Also known as: Revlimid

Lenalidomide

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients must have histologically or cytologically confirmed LCH, ECD or HS. Confirmation of outside pathology at BWH will be performed but is not mandatory prior to study enrollment (see section 3).
• Detectable disease by at least one of the following modalities: CT, PET, bone scan, or MRI.
• Patients with LCH must require systemic therapy according to the Histiocyte Society LCH Evaluation and Treatment Guidelines (HS 2009)
• \-- Or
• Patients with HS requiring systemic treatment as defined by disease that cannot be surgically resected and/or encompassed in a single radiation field.
• Age 18 years or older.
• ECOG performance status ≤2 (Karnofsky ≥60%, see Appendix A)
• Participants must have normal organ and marrow function as defined below:
• absolute neutrophil count ≥1,000/mcL
• platelets ≥100,000/mcL
• total bilirubin within 1.5 times normal institutional limits
• AST(SGOT)/ALT(SGPT) ≤3 × institutional upper limit of normal
• creatinine within 2 times normal institutional limits
• \--- OR
• creatinine clearance ≥30 mL/min/1.73 m2. Note, dose adjustments are required for CrCl ≥30 mL/min but ≤60 ml/min.

You may not qualify if:

• Prior chemotherapy or radiation within 2 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study or those who have not recovered from adverse events due to agents administered more than 2 weeks earlier.
• Participants who are receiving any other investigational agents.
• Prior treatment with lenalidomide. Patients previously treated with thalidomide who discontinued treatment for reasons aside from an adverse reaction to thalidomide are permitted.
• History of another invasive malignancy unless treated with curative intent 5 years or more prior to study entry. Patients with localized carcinoma of the cervix, non-melanoma skin cancer, or early stage prostate cancer requiring observation only are eligible regardless of timing of diagnosis.
• Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
• Pregnant women are excluded from this study because lenalidomide has known teratogenic effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with lenalidomide, breastfeeding should be discontinued if the mother is treated with lenalidomide.
• Known active hepatitis B (HBV) or hepatitis C (HCV) infection. Patients who are positive only for HBV surface antibody as a result of prior vaccination are eligible. Patients with a positive HBV core antibody but undetectable HBV viral load are eligible.
• HIV-positive participants on combination antiretroviral therapy are ineligible because of the potential for pharmacokinetic interactions with lenalidomide. In addition, these participants are at increased risk of lethal infections when treated with marrow-suppressive therapy. Appropriate studies will be undertaken in participants receiving combination antiretroviral therapy when indicated.
• Concomitant corticosteroids unless patient has been on a stable dose of prednisone (or equivalent) of ≤10 mg daily for at least 2 weeks prior to first dose of study drug.
• Inability to swallow pills.

Sponsors & Collaborators

• Dana-Farber Cancer Institute: lead
• Celgene: collaborator

Study Sites (1)

Dana-Farber Cancer Institute

Boston, Massachusetts, 02115, United States

Location

MeSH Terms

Conditions

Histiocytosis, Langerhans-Cell; Histiocytic Sarcoma; Erdheim-Chester Disease

Interventions

Lenalidomide

Condition Hierarchy (Ancestors)

Lung Diseases, Interstitial; Lung Diseases; Respiratory Tract Diseases; Histiocytosis; Lymphatic Diseases; Hemic and Lymphatic Diseases; Histiocytic Disorders, Malignant; Neoplasms by Histologic Type; Neoplasms; Histiocytosis, Non-Langerhans-Cell

Intervention Hierarchy (Ancestors)

Phthalimides; Phthalic Acids; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Piperidones; Piperidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Isoindoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Results Point of Contact

• Title: Eric Jacobsen, MD
• Organization: Dana-Farber Cancer Institute

eric_jacobsen@dfci.harvard.edu

857-215-1405

Study Officials

• Eric Jacobsen, MD

  Dana-Farber Cancer Institute

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: August 12, 2015
• First Posted: August 14, 2015
• Study Start: August 1, 2015
• Primary Completion: February 1, 2024
• Study Completion: February 1, 2026
• Last Updated: December 10, 2025
• Results First Posted: April 25, 2025

Record last verified: 2025-11

Locations

US (1)