Neoplasms

All Conditions

Blood and Lymph Conditions

Respiratory Tract (Lung and Bronchial) Diseases

Rare Diseases

Sarcoma

Erdheim-Chester Disease

Histiocytosis, Langerhans-Cell

Histiocytosis

Histiocytic Sarcoma

Neoplasms, Connective and Soft Tissue

Neoplasms by Histologic Type

Lymphatic Diseases

Lung Diseases

Lung Diseases, Interstitial

Respiratory Tract Diseases

Histiocytosis, Non-Langerhans-Cell

Histiocytic Disorders, Malignant

Soft Tissue Sarcoma

Lipogranulomatosis

Langerhans Cell Histiocytosis

Non-Langerhans-Cell Histiocytosis

Antineoplastic Agents

All Drugs and Chemicals

Lenalidomide

Immunologic Factors

Angiogenesis Inhibitors

Growth Inhibitors

After the screening procedures confirm participation in the research study.

- Lenalidomide Oral, Daily for 21 days of each cycle

Eric Jacobsen, MD

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).

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.

A Study of Lenalidomide for Adult Histiocyte Disorders

A Phase II Study of Lenalidomide for Adult Histiocyte Disorders

Celgene

Dana-Farber Cancer Institute

Principal Investigator

Symptoms and General Pathology

Nervous System Diseases

Urinary Tract, Sexual Organs, and Pregnancy Conditions

Diseases and Abnormalities at or Before Birth

Recurrence

Carcinoma

Brain Neoplasms

Osteosarcoma

Neuroblastoma

Wilms Tumor

Central Nervous System Neoplasms

Medulloblastoma

Sarcoma, Ewing

Ependymoma

Rhabdomyosarcoma

Rhabdoid Tumor

Nervous System Neoplasms

Germ Cells Tumors

Wilms' Tumor

Ewing Sarcoma

Pineoblastoma

Antirheumatic Agents

Anti-Infective Agents

Anti-Inflammatory Agents

Analgesics

Etoposide

Cyclophosphamide

Sirolimus

Etoposide phosphate

Celecoxib

Temsirolimus

MTOR Inhibitors

Immunosuppressive Agents

Antineoplastic Agents, Alkylating

Alkylating Agents

Enzyme Inhibitors

Anti-Bacterial Agents

Antibiotics, Antitubercular

Clotrimazole

Miconazole

Antifungal Agents

Anti-Inflammatory Agents, Non-Steroidal

Analgesics, Non-Narcotic

Cyclooxygenase 2 Inhibitors

Cyclooxygenase Inhibitors

Participants in this group will receive oral sirolimus and celecoxib in addition to cycles of oral etoposide and cyclophosphamide for up to two years.

The starting dose for sirolimus is 2 mg/m2 once daily. The dose of sirolimus will be individually adjusted to achieve a target serum trough concentration in the range of 10-15 ng/ml. Sirolimus will be given by mouth every day for six weeks (every six weeks is called one cycle) for up to two years or 16 cycles.

Celecoxib 100 mg will be given by mouth twice a day for six weeks (every six weeks is called one cycle) for up to two years or 16 cycles.

Etoposide 50 mg/m2 (maximum dose 100 mg) will be given daily by mouth for the first 3 weeks of a 6 week cycle. Six week cycles will be repeated for up to two years or 16 cycles.

Cyclophosphamide 2.5 mg/Kg (maximum dose 100 mg) will be given daily by mouth for the second 3 weeks of a 6 week cycle. Six week cycles will be repeated for up to two years or 16 cycles.

Thomas Cash, MD

Laura Evdokimo

Lindsey Hoffman, DO

Debra Bertz, CTR, MBA

Emi Caywood, MD

Robin E Ryan, MPH

Kevin Ginn, MD

Cindy Fischer, CCRC

William Petersen, MD

This study aims to determine the efficacy of daily sirolimus and celecoxib, with low dose etoposide alternating with cyclophosphamide for pediatric participants with relapsed or refractory tumors.

This study aims to learn if the combination of oral sirolimus once daily with celecoxib, and with oral etoposide alternating every 21 days with oral cyclophosphamide (metronomic chemotherapy) is effective in shrinking relapsed or refractory tumors in pediatric participants. In addition, this study seeks to learn the length of time this combination can keep the tumor from growing, learn more about the side effects of sirolimus when used in this combination, and to learn if the sirolimus is working by evaluating blood and tumor tissue.

Sirolimus in Combination With Metronomic Chemotherapy in Children With Recurrent and/or Refractory Solid and CNS Tumors

AflacST1502: A Phase II Study of Sirolimus in Combination With Metronomic Chemotherapy in Children With Recurrent and/or Refractory Solid and CNS Tumors

Radiographic response to treatment will be assessed by Response Evaluation Criteria in Solid Tumors (RECIST) for participants with solid tumors via CT or MRI scan. A complete response (CR) is a disappearance of all target and non-target lesions. Partial response (PR) is at least a 30% decrease in the disease measurement compared to the baseline measurement. Stable disease (SD) is neither sufficient shrinkage to qualify for partial response (PR) nor sufficient increase to qualify for progressive disease (PD) taking as reference the smallest disease measurement since baseline. Progressive disease (PD) is at least a 20% increase in the disease measurement compared to baseline, or the appearance of one or more new lesions, or evidence of laboratory or clinical progression.

Radiographic response to treatment will be assessed for participants with CNS tumors via CT or MRI scan. Response criteria are assessed based on the lesion of the longest diameter and its longest perpendicular diameter. Development of new disease or progression in any established lesions is considered progressive disease, regardless of response in other lesions. Complete response (CR) is the the disappearance of all target lesions. Partial response (PR) is greater than or equal to 50% decrease decrease in the sum of the products of the two perpendicular diameters of all target lesions, taking into reference the baseline measurement. Stable disease (SD) is neither a sufficient decrease in the sum of the products of the two perpendicular diameters of all target lesions to qualify for PR, nor sufficient increase in a single target lesion to qualify for progressive disease (PD).

The adverse events associated with sirolimus in combination with metronomic chemotherapy administered on this schedule will be defined and evaluated throughout the treatment period.

Cannonball Kids' Cancer Foundation

Hyundai Hope On Wheels

Emory University

Associate Professor

Immune System Diseases

Infections

Digestive System Diseases

Nutritional and Metabolic Diseases

Syndrome

Leukemia

Leukemia, Myeloid

Leukemia, Myeloid, Acute

Preleukemia

Leukemia, Myelomonocytic, Juvenile

Myelodysplastic Syndromes

Anemia, Sickle Cell

Precursor Cell Lymphoblastic Leukemia-Lymphoma

Leukemia, Lymphoid

Anemia

Lymphoma

Thrombocytopenia

Thalassemia

beta-Thalassemia

Acquired Immunodeficiency Syndrome

HIV Infections

Immunologic Deficiency Syndromes

Anemia, Aplastic

Leukemia, Myelogenous, Chronic, BCR-ABL Positive

Neutropenia

Lymphocytosis

Bone Marrow Failure Disorders

Pancytopenia

Macrophage Activation Syndrome

Hemoglobinopathies

Primary Immunodeficiency Diseases

Shwachman-Diamond Syndrome

Anemia, Diamond-Blackfan

Hematologic Diseases

Bone Marrow Diseases

Precancerous Conditions

Lymphoproliferative Disorders

Immunoproliferative Disorders

Myeloproliferative Disorders

Chronic Disease

Disease Attributes

Myelodysplastic-Myeloproliferative Diseases

Myeloid Leukemia

Acute Myeloid Leukemia

Acute Non Lymphoblastic Leukemia

Chronic Myelomonocytic Leukemia

Juvenile Myelomonocytic Leukemia

Sickle Cell Anemia

Acute Lymphoblastic Leukemia

Lymphoblastic Lymphoma

Chronic Graft Versus Host Disease

Lymphosarcoma

Beta-thalassemia

Aplastic Anemia

Chronic Myeloid Leukemia

Granulocytopenia

Acute Graft Versus Host Disease

Diamond-Blackfan Anemia

Familial Hemophagocytic Lymphohistiocytosis

Severe Congenital Neutropenia

Sickle Beta Thalassemia

Chronic Myeloproliferative Disorders

Myelodysplastic/myeloproliferative Disease

Pharmaceutical Solutions

Hematinics

Antiemetics

Neuroprotective Agents

Gastrointestinal Agents

Tacrolimus

Fludarabine

Fludarabine phosphate

Melphalan

Prednisolone

Busulfan

Liver Extracts

Methylprednisolone

Thiotepa

Methylprednisolone Acetate

Methylprednisolone Hemisuccinate

Prednisolone acetate

Prednisolone hemisuccinate

Prednisolone phosphate

Alemtuzumab

Calcineurin Inhibitors

Glucocorticoids

Hormones

Hormone Antagonists

Protective Agents

Antineoplastic Agents, Hormonal

Antineoplastic Agents, Immunological

Patients will start their pre-conditioning regimen on 8 days before scheduled transplant. Fractionated total body irradiation (TBI) will be administered twice daily on the 6th, 7th, and 8th before transplant. Patients will receive Thiotepa on the 4th and 5th day before transplant, Cyclophosphamide on the 2nd and 3rd day before transplant, and Alemtuzumab on the 1st-5th day(s) before transplant. Then the stem cell infusion will be performed (allogeneic family member or ≥ 8/10 HLA matched adult unrelated donor peripheral blood stem cell transplantation with CD34 Selection using CliniMACS CD34+ Reagent System). GVHD prophylaxis will consist of tacrolimus only. Tacrolimus administration will begin on the day after transplant, and methylprednisolone will start on day -5.

Patients will start their pre-conditioning regimen 9 days before their scheduled transplant. Patients will receive busulfan twice daily on the 5th-8th day before transplant, and Melphalan on the 2nd-4th days before transplant and Alemtuzumab on the 1st-5th day before transplant. Subjects will then undergo with their stem cell infusion (allogeneic family member or ≥ 8/10 HLA matched adult unrelated donor peripheral blood stem cell transplantation with CD34 Selection using CliniMACS CD34+ Reagent System) and GVHD prophylaxis will consist of tacrolimus only. Tacrolimus administration will begin on the day after their transplant, and methylprednisolone will start on day -5.

Patients will begin tacrolimus 8 days pre-transplant, and then will receive alemtuzumab on the 3rd-7th day pre-transplant; busulfan twice daily on the 5th-8th day pre-transplant; and fludarabine on the 2nd-7th day pre-transplant. Methylprednisolone will start on day -7.The stem cell infusion will be performed (with CD34 Selection using CliniMACS CD34+ Reagent System). GVHD prophylaxis will consist of tacrolimus or sirolimus. For patients with a history of hepatic toxicity and/or high-risk for veno-occlusive disease or other liver toxicity post stem cell transplant, melphalan at 70 mg/m2 will be substituted for Busulfan, followed by fludarabine on the 2nd-7th day before transplant and alemtuzumab on the 3rd-7th day before transplant.

The CliniMACS® Reagent System (Miltenyi Biotec, Germany), is a semi-automated immunomagnetic cell selection medical device that is used in vitro to select and enrich specific cell populations in a closed, sterile environment. The system is comprised of a computer controlled medical device containing a permanent magnet, a closed-system sterile tubing set containing columns coated with a ferromagnetic matrix, and a magnetic cell specific labeling reagent.

CliniMACS CD34+ Reagent System

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
A Study of Lenalidomide for Adult Histiocyte Disorders	NCT02523040	Langerhans Cell... Histiocytoses E... Histiocytic Sar...	Lenalidomide	18 Years -	Dana-Farber Cancer Institute
A Study of Lenalidomide for Adult Histiocyte Disorders	NCT02523040	Langerhans Cell... Histiocytoses E... Histiocytic Sar...	Lenalidomide	18 Years -	Dana-Farber Cancer Institute
Sirolimus in Combination With Metronomic Chemotherapy in Children With Recurrent and/or Refractory Solid and CNS Tumors	NCT02574728	Cancer	Sirolimus Celecoxib Etoposide Cyclophosphamid...	12 Months - 30 Years	Emory University
CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant	NCT02061800	Chronic Myeloid... Acute Myelogeno... Myelodysplastic... Juvenile Myelom... Acute Lymphobla... Lymphoma (Hodgk...	CliniMACS CD34+... Thiotepa Cyclophosphamid... Alemtuzumab Tacrolimus Melphalan Busulfan Fludarabine Methylprednisol...	- 22 Years	Columbia University
Cobimetinib in Refractory Langerhans Cell Histiocytosis (LCH), and Other Histiocytic Disorders	NCT04079179	Langerhan's Cel... Juvenile Xantho... Erdheim-Chester... Rosai Dorfman D... Neuro-Degenerat... Histiocytic Sar... Histiocytic Dis...	Cobimetinib	-	Baylor College of Medicine
A Study of Lenalidomide for Adult Histiocyte Disorders	NCT02523040	Langerhans Cell... Histiocytoses E... Histiocytic Sar...	Lenalidomide	18 Years -	Dana-Farber Cancer Institute

Spots Global Cancer Trial Database for langerhans cell histiocytosis (lch)