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Langerhans Cell Histiocytosis (LCH)Phase 2NCT02523040

Lenalidomide for Adult Histiocyte Disorders

A Phase 2 study testing Lenalidomide in people with Langerhans Cell Histiocytosis (LCH) and Histiocytoses Erdheim-chester Disease, run by Dana-Farber Cancer Institute at 1 site in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial 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). Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Lenalidomide
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
12 participants
Where
1 siteUnited States
Study dates
Aug 2015to Dec 2026
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.
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  • 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.
  • Able to take aspirin 81 mg daily as prophylactic anticoagulation if not on warfarin, low molecular weight heparin or oral factor Xa inhibitor. Patients intolerant to ASA may use warfarin or low molecular weight heparin at doses designed to treat deep venous thrombosis.
  • All study participants must be registered into the mandatory Revlimid REMS® program, and be willing and able to comply with the requirements of the REMS® program.
  • Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation.
  • Ability to understand and the willingness to sign a written informed consent document.

Who cannot join

  • 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.
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  • 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.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Dana-Farber Cancer Institute

    Boston, Massachusetts, 02115

Other trials in United States

About this study

Official title: A Phase II Study of Lenalidomide for Adult Histiocyte Disorders

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).

Read the detailed description

This research study is a Phase II clinical trial. Phase II clinical trials test the safety and effectiveness of an investigational intervention to learn whether the intervention works in treating a specific disease. "Investigational" means that the intervention is being studied.

The FDA (the U.S. Food and Drug Administration) has not approved lenalidomide for your specific disease but it has been approved for other uses. Lenalidomide is a chemotherapy drug that belongs to a class of drugs called immunomodulatory drugs (IMiDs), which modify a participant's immune response in order to treat cancer. Lenalidomide alters the body's immune system and it may also interfere with the development of tiny blood vessels that help support tumor growth. Therefore, it may reduce or prevent the growth of cancer cells. Lenalidomide has been shown to restore the immune cells' ability to attack and kill tumor cells Lenalidomide is approved by the FDA to treat certain cancers including multiple myeloma and myelodysplastic syndrome.

Being tested
Lenalidomide
Keywords
Langerhans cell histiocytosis (LCH), Erdheim-Chester disease (ECD), Histiocytic Sarcoma (HS)
Medical subject headings
Histiocytosis, Langerhans-Cell, Histiocytic Sarcoma, Erdheim-Chester Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Study groups in NCT02523040
Study groupRoleWhat they receive
LenalidomideExperimentalAfter the screening procedures confirm participation in the research study. - Lenalidomide Oral, Daily for 21 days of each cycle
Receives: Drug: Lenalidomide

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

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

Secondary outcomes (6)

  1. 12-months Progression-free Survival (PFS)

    Progression-free survival (PFS) based on the Kaplan-Meier method is defined as time from registration to progression (PD) or death, censored for patients alive and progression-free at last disease assessment. PD is defined by Histiocyte criteria. Percent PFS is the percent of patients who are alive and progression-free at 12 months.

    Measured: 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.

  2. 12-months Overall Survival (OS)

    Overall survival (OS) based on the Kaplan-Meier method is defined as the time from registration to death, censored for patients alive at last contact. Percent OS is the percent of patients who are alive at 12 months.

    Measured: 12 Months

  3. Incidence of Grade 3-4 Toxicity

    Grade 3-4 adverse events (AE) with treatment attribution of possibly, probably or definite (treatment-related) based on NCI Common Toxicity Criteria for Adverse Events version 3 (CTCAEv3) as reported on case report forms were tabulated by maximum grade. Incidence is the proportion of participants experiencing at least one treatment-related grade 3-4 AE of any type during the time of observation.

    Measured: Up to 12 months on treatment.

  4. Urine Cell Free DNA for BRAF

    Quantitative serial measurements of urine cell free DNA for BRAF mutation as a biomarker of response

    Measured: 12 Months

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

    Serum TNF-alpha levels were quantified per established methods and maximum percent change in level from baseline derived.

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

  6. Plasma Cell Free DNA for BRAF

    Quantitative serial measurements of plasma cell free DNA for BRAF mutation as a biomarker of response

    Measured: 12 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted14 Aug 2015 (estimated)
  2. Study startAug 2015
  3. Primary completionFeb 2024
  4. Study completionDec 2026 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
12 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02523040
Sponsor's protocol ID
15-197
Status last verified
Jul 2026
First submitted
12 Aug 2015
Last updated
23 Jul 2026
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Lenalidomide in people with Langerhans Cell Histiocytosis (LCH) and Histiocytoses Erdheim-chester Disease, run by Dana-Farber Cancer Institute at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 19 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 23 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 25 Apr 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Overall Response Rate (ORR) (time frame: Disease assessed every 3 cycles on treatment up do 12 cycles (approximately 12 months).). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02523040.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Jul 2026; AzTrials last refreshed its copy on 26 Oct 2026.

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