Chemotherapy for Histiocytosis

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Dana-Farber Cancer Institute

Must be taking: Aspirin

Must not be taking: Lenalidomide, Antiretrovirals

Disqualifiers: Pregnancy, Active hepatitis, Uncontrolled illness, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 6

Duration: 12 months

12 Participants Needed

Trial Summary

What is the purpose of this trial?

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).

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.

What data supports the effectiveness of the drug Lenalidomide for treating histiocytosis?

Research shows that Lenalidomide, when used with dexamethasone, has helped achieve remission in patients with difficult-to-treat Langerhans cell histiocytosis (LCH), a type of histiocytosis. In several cases, patients experienced significant improvement and remained in remission for months after treatment. ^{1 2 3 4 5}

Is lenalidomide safe for treating histiocytosis?

Lenalidomide, when used with dexamethasone, has shown to be safe with minimal side effects in children with relapsed or refractory Langerhans cell histiocytosis, leading to remission without significant adverse effects. ^{1 2 3 5 6}

How does the drug lenalidomide differ from other treatments for histiocytosis?

Lenalidomide is unique because it is an oral drug that modulates the immune system and has shown effectiveness in achieving complete remission in aggressive, relapsing Langerhans cell histiocytosis, a rare condition with no standard treatment. Unlike thalidomide, lenalidomide does not cause neurotoxicity, making it a safer long-term option. ^{7 8 9 10 11}

Research Team

Eric Jacobsen, MD

Principal Investigator

Dana-Farber Cancer Institute

Eligibility Criteria

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.

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.

Timeline

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

Treatment Details

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.

Participant Groups

Group I: Lenalidomide Experimental Treatment 1 Intervention

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

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

Approved in European Union as Revlimid for:

• Multiple myeloma
• Myelodysplastic syndromes
• Mantle cell lymphoma
• Follicular lymphoma
• Marginal zone lymphoma

Approved in United States as Revlimid for:

• Multiple myeloma
• Myelodysplastic syndromes
• Mantle cell lymphoma
• Follicular lymphoma
• Marginal zone lymphoma

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+

Top Products

>- **Revlimid (lenalidomide)**: Multiple myeloma, myelodysplastic syndromes, and mantle cell lymphoma treatment. - **Pomalyst (pomalidomide)**: Relapsed/refractory multiple myeloma treatment. - **Otezla (apremilast)**: Psoriatic arthritis treatment. - **Thalomid (thalidomide)**: Erythema nodosum leprosum and multiple myeloma treatment.

Jay Backstrom

Celgene

Chief Medical Officer since 2016

MD

Mark Alles

Celgene

Chief Executive Officer since 2016

Bachelor's degree from Lock Haven University of Pennsylvania

Findings from Research

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Lenalidomide: a novel anticancer drug with multiple modalities. Galustian, C., Dalgleish, A.[2019]

References

1. United States pubmed.ncbi.nlm.nih.gov

Excellent remission rates with limited toxicity in relapsed/refractory Langerhans cell histiocytosis with pulse dexamethasone and lenalidomide in children. [2022]

2. England pubmed.ncbi.nlm.nih.gov

Phase 2 study of oral thalidomide-cyclophosphamide-dexamethasone for recurrent/refractory adult Langerhans cell histiocytosis. [2023]

3. Czech Republic pubmed.ncbi.nlm.nih.gov

[Lenalidomide induced therapeutic response in a patient with aggressive multi-system Langerhans cell histiocytosis resistant to 2-chloro-deoxyadenosine and early relapsing after high-dose BEAM chemotherapy with autologous peripheral blood stem cell transplantation]. [2018]

4. France pubmed.ncbi.nlm.nih.gov

[Prognostic factors in histiocytosis X]. [2006]

5. United States pubmed.ncbi.nlm.nih.gov

Successful Treatment of Refractory Langerhans Cell Histiocytosis of the Choroid Plexus in a Child With Pulse Dexamethasone and Lenalidomide. [2022]

6. Japan pubmed.ncbi.nlm.nih.gov

Treatment of Langerhans cell histiocytosis bone lesions with zoledronic acid: a case series. [2021]

7. England pubmed.ncbi.nlm.nih.gov

The potential of immunomodulatory drugs in the treatment of solid tumors. [2018]

8. United States pubmed.ncbi.nlm.nih.gov

Salvage lenalidomide in four rare oncological diseases. [2022]

9. Czech Republic pubmed.ncbi.nlm.nih.gov

[The effect of lenalidomide on rare blood disorders: Langerhans cell histiocytosis, multicentric Castleman disease, POEMS syndrome, Erdheim-Chester disease and angiomatosis]. [2018]

10. New Zealand pubmed.ncbi.nlm.nih.gov

Lenalidomide: a review of its use in patients with transfusion-dependent anaemia due to low- or intermediate-1-risk myelodysplastic syndrome associated with 5q chromosome deletion. [2021]

11. England pubmed.ncbi.nlm.nih.gov

Lenalidomide: a novel anticancer drug with multiple modalities. [2019]