Chemotherapy for Histiocytosis

Age: 18+
Sex: Any
Trial Phase: Phase 2
Sponsor: Dana-Farber Cancer Institute
Must be taking: Aspirin
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 2 JurisdictionsThis treatment is already approved in other countries

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a chemotherapy drug called Lenalidomide to determine its effectiveness in treating certain rare diseases known as histiocytic disorders. These disorders include Langerhans cell histiocytosis, Erdheim-Chester disease, and histiocytic sarcoma, where the body produces too many immune cells. Individuals with these conditions who require systemic treatment may be suitable candidates. Participants must be able to take aspirin as part of the trial and must not have received certain treatments recently. As a Phase 2 trial, the research focuses on evaluating the treatment's effectiveness in an initial, smaller group, offering participants an opportunity to contribute to significant medical advancements.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot participate if you are on other investigational drugs or certain HIV treatments. If you're on corticosteroids, you must be on a stable low dose for at least 2 weeks before starting the study drug.

Is there any evidence suggesting that Lenalidomide is likely to be safe for humans?

Research has shown that lenalidomide is generally well-tolerated by patients. In studies involving individuals with Rosai-Dorfman disease, lenalidomide combined with another drug proved to be safe, with most patients experiencing no serious side effects. Some reported mild issues such as nausea, vomiting, and diarrhea, which are common with many medications.

Another study found that lenalidomide benefited people with Langerhans cell histiocytosis (LCH), a disorder similar to the one being studied in this trial, and it was also considered safe. While some individuals might experience mild side effects, previous research indicates that the drug is generally safe.

These findings suggest that lenalidomide is a promising option for the conditions under study in this trial. However, participants should always discuss potential risks with the study team before joining any trial.12345

Why do researchers think this study treatment might be promising for histiocytosis?

Lenalidomide is unique because it offers a novel approach to treating histiocytosis by modulating the immune system. Unlike standard treatments that often focus on chemotherapy or targeted therapies, lenalidomide works by enhancing the immune response and inhibiting the growth of abnormal cells. Researchers are excited about lenalidomide because it has the potential to be more selective in targeting diseased cells, possibly leading to fewer side effects and improved outcomes for patients. This new mechanism of action represents a promising alternative to existing options.

What evidence suggests that Lenalidomide might be an effective treatment for histiocytosis?

Research has shown that Lenalidomide, the treatment under study in this trial, yields promising results for certain histiocyte disorders. In Langerhans cell histiocytosis (LCH), Lenalidomide has demonstrated potential, particularly in cases unresponsive to standard treatments. Studies have highlighted its effectiveness and safety, offering hope to patients seeking new options. Additionally, in Rosai-Dorfman disease, Lenalidomide achieved an overall response rate of 87%, indicating strong potential for success in similar conditions. These findings support ongoing research into Lenalidomide for treating histiocytic disorders like LCH, Erdheim-Chester disease (ECD), and histiocytic sarcoma (HS).14567

Who Is on the Research Team?

Eric D. Jacobsen, MD - Dana-Farber ...

Eric Jacobsen, MD

Principal Investigator

Dana-Farber Cancer Institute

Are You a Good Fit for This Trial?

Adults over 18 with Langerhans cell histiocytosis, Erdheim-Chester disease, or histiocytic sarcoma needing systemic therapy can join. They must have good organ function and performance status, confirmed diagnosis, detectable disease via scans, and be able to take daily aspirin unless on certain anticoagulants. Participants need to follow birth control guidelines and the Revlimid REMS program.

Inclusion Criteria

I can care for myself but may not be able to do active work.
You have a disease that can be seen on a CT scan, PET scan, bone scan, or MRI.
My condition is confirmed as LCH, ECD, or HS.
See 6 more

Exclusion Criteria

I have been treated with lenalidomide before.
I had cancer before, but it was treated over 5 years ago.
I am not pregnant or breastfeeding.
See 7 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Lenalidomide orally, daily for 21 days of each cycle, with disease assessed every 3 cycles up to 12 cycles

12 months
Disease assessed every 3 cycles

Follow-up

Participants are monitored for progression-free survival and overall survival, with assessments every 3 months for 2 years, then every 6 months

36 months
Every 3 months for 2 years, then every 6 months

What Are the Treatments Tested in This Trial?

Interventions

  • Lenalidomide
Trial Overview The trial is testing Lenalidomide as a potential chemotherapy treatment for three types of histiocyte disorders. It aims to see how effective it is in treating these diseases by monitoring participants' responses to the drug.
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: LenalidomideExperimental Treatment1 Intervention

Lenalidomide is already approved in European Union, United States for the following indications:

🇪🇺
Approved in European Union as Revlimid for:
🇺🇸
Approved in United States as Revlimid for:

Find a Clinic Near You

Who Is Running the Clinical Trial?

Dana-Farber Cancer Institute

Lead Sponsor

Trials
1,128
Recruited
382,000+

Celgene

Industry Sponsor

Trials
649
Recruited
130,000+
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Jay Backstrom profile image

Jay Backstrom

Celgene

Chief Medical Officer since 2016

MD

Mark Alles profile image

Mark Alles

Celgene

Chief Executive Officer since 2016

Bachelor's degree from Lock Haven University of Pennsylvania

Published Research Related to This Trial

Lenalidomide (REVLIMID®) is an effective treatment for multiple myeloma and transfusion-dependent anemia in myelodysplastic syndromes, showing better activity and less neurotoxicity compared to its predecessor, thalidomide.
This drug has multiple beneficial properties, including anti-inflammatory and antiangiogenic effects, and may play a significant role in treating various cancers beyond its current approved uses.
The potential of immunomodulatory drugs in the treatment of solid tumors.Dalgleish, A., Galustian, C.[2018]
Lenalidomide has become a crucial treatment option for multiple myeloma and del-5q myelodysplastic syndromes, and it has been tested for various other cancers, showcasing its broad therapeutic potential.
The drug received FDA approval for use in combination with dexamethasone for previously treated multiple myeloma, highlighting its efficacy and safety in this specific patient population.
Lenalidomide: a novel anticancer drug with multiple modalities.Galustian, C., Dalgleish, A.[2019]

Citations

Lenalidomide and dexamethasone for Rosai-Dorfman diseaseThe overall response rate was 87%, and the 2-year overall survival and progression-free survival were 100% and 69.0%, respectively. Implications of all the ...
Lenalidomide proved effective in multisystem Langerhans ...This new drug demonstrated a therapeutic potential in LCH resistant to some of the conventional treatments, while maintaining a high safety profile.
Lenalidomide proved effective in multisystem Langerhans ...Previous studies on Langerhans'-cell histiocytosis (LCH) were mostly retrospective and patients had been recruited over a long time period.
Acute lymphoblastic leukemia in patients treated with ...Details of 7 out of 20 ALL cases were reported in RCTs (Supplementary Table 5) with a median age at ALL onset of 62.6 [IQR 61.4–66.2] years.
5.pubmed.ncbi.nlm.nih.govpubmed.ncbi.nlm.nih.gov/22103591/
Lenalidomide proved effective in multisystem Langerhans ...Lenalidomide proved effective in multisystem Langerhans cell histiocytosis. Acta Oncol. 2012 Mar;51(3):412-5. doi: 10.3109/0284186X.
Lenalidomide and Dexamethasone for Patients with Rosai ...No patients died or lost follow-up. The estimated 2-year EFS and OS was 67.8% and 100.0%, respectively. To evaluate the prognostic factors of ...
Lenalidomide and Dexamethasone for Patients with Rosai ...8 patients (36.4%) had grade 1 gastrointestinal complications (vomiting, nausea and diarrhea). 6 patients (27.3%) experienced anemia, ...
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