Vinblastine/Prednisone Versus Single Therapy With Cytarabine for Langerhans Cell Histiocytosis (LCH)

Description

Langerhans Cell Histiocytosis (LCH) is a type of cancer that can damage tissue or cause lesions to form in one or more places in the body. Langerhans cell histiocytosis (LCH) is a cancer that begins in LCH cells (a type of dendritic cell which fights infection). Sometimes there are mutations (changes) in LCH cells as they form. These include mutations of the BRAF gene. These changes may make the LCH cells grow and multiply quickly. This causes LCH cells to build up in certain parts of the body, where they can damage tissue or form lesions. For most patients with LCH, standard-of-care vinblastine/prednisone are used as front-line therapy while cytarabine therapy has been used as therapy for patients who develop recurrence. No alternate treatment strategy has been developed for frontline therapy in LCH. The purpose of this research study is to compare previously used vinblastine/prednisone to single therapy with cytarabine for LCH. We will evaluate the utility of an imaging study called a positron emission tomography (PET) scan to more accurately assess areas of LCH involvement not otherwise seen in other imaging studies as well as response to therapy. We also want to identify if genetic and other biomarkers (special proteins in patient's blood and in patient's cancer) relate to the response of patients LCH to study treatment.

Conditions

Langerhans Cell Histiocytosis

Talk to us

Study Overview

On this page

Study Details

Study overview

Langerhans Cell Histiocytosis (LCH) is a type of cancer that can damage tissue or cause lesions to form in one or more places in the body. Langerhans cell histiocytosis (LCH) is a cancer that begins in LCH cells (a type of dendritic cell which fights infection). Sometimes there are mutations (changes) in LCH cells as they form. These include mutations of the BRAF gene. These changes may make the LCH cells grow and multiply quickly. This causes LCH cells to build up in certain parts of the body, where they can damage tissue or form lesions. For most patients with LCH, standard-of-care vinblastine/prednisone are used as front-line therapy while cytarabine therapy has been used as therapy for patients who develop recurrence. No alternate treatment strategy has been developed for frontline therapy in LCH. The purpose of this research study is to compare previously used vinblastine/prednisone to single therapy with cytarabine for LCH. We will evaluate the utility of an imaging study called a positron emission tomography (PET) scan to more accurately assess areas of LCH involvement not otherwise seen in other imaging studies as well as response to therapy. We also want to identify if genetic and other biomarkers (special proteins in patient's blood and in patient's cancer) relate to the response of patients LCH to study treatment.

Randomization of Cytarabine Monotherapy Versus Standard-of-Care Vinblastine/Prednisone for Frontline Treatment of Langerhans Cell Histiocytosis (TXCH LCH0115)

Vinblastine/Prednisone Versus Single Therapy With Cytarabine for Langerhans Cell Histiocytosis (LCH)

Condition
Langerhans Cell Histiocytosis
Intervention / Treatment

-

Contacts and Locations

Palo Alto

Stanford Children's Hospital, Lucile Packard Children's Hospital, Palo Alto, California, United States, 94304

San Diego

Rady Children's Hospital - San Diego, San Diego, California, United States, 92123

Minneapolis

University of Minnesota/Masonic Cancer Center, Minneapolis, Minnesota, United States, 55455

Columbus

Nationwide Children's Hospital, Columbus, Ohio, United States, 43205

Allentown

Lehigh Valley Health Network- Cedar Crest, Allentown, Pennsylvania, United States, 18103

Austin

Dell Children's Medical Center, Austin, Texas, United States, 78723

Fort Worth

Cook Children's Health Care System, Fort Worth, Texas, United States, 76104

Houston

Texas Children's Hospital, Houston, Texas, United States, 77030

McAllen

Vannie Cook Children's Clinic, McAllen, Texas, United States, 78503

San Antonio

Children's Hospital of San Antonio, San Antonio, Texas, United States, 78207

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • 1. Patient must have biopsy-confirmed diagnosis of Langerhans cell histiocytosis.
  • 2. Patient must be between 0-21 years of age.
  • 3. Patient must have a Karnofsky performance score ≥ 50% or Lansky performance score ≥ 50%.
  • 1. Patient may not have received any prior systemic cytotoxic or other chemotherapies for LCH or any other malignant disorder prior to the initiation of protocol therapy on TXCH LCH0115 with the exception of:
  • 2. Patient may not have disease limited to a single skin or bone site, with the following exceptions:
  • * Central Nervous System (CNS) risk lesions/special site disease: patients with single bone sites that are CNS-risk (sphenoid, mastoid, orbital, zygomatic, ethmoid, maxillary, or temporal bones, the cranial fossa, pituitary gland or neurodegenerative disease) or are "special sites" (odontoid peg, vertebral lesion with intraspinal soft tissue extension) require systemic therapy as standard of care and thus are eligible for the study.
  • * Functionally critical lesions: A single lesion not described above which may cause "functionally critical anatomic abnormality" wherein attempts at local therapy (such as surgical curettage or radiation) would cause unacceptable morbidity. These patients may be enrolled with written approval of the Coordinating Center PI or Vice-Chair and documentation of the rationale justifying systemic therapy.
  • * Asynchronous multisite LCH presentation: A patient may also have any single site of disease involvement at the time of enrollment if they previously had at least one other site of LCH disease in the past (which may have been treated with local therapy/surgery as described), as long as no systemic therapy was previously given per protocol guidelines.
  • 3. Patient may not have severe renal disease (creatinine greater than 3 times normal for age OR creatinine clearance \< 50 ml/m2/1.73m\^2).
  • 4. Patient may not have severe hepatic disease (direct bilirubin greater than 3 mg/dl OR aspartate aminotransferase (AST) greater than 500 IU/L), unless hepatic injury is due to LCH.
  • 5. Female patients may not be pregnant or breastfeeding.
  • 6. Patients of reproductive potential not willing to use an adequate method of birth control for the duration of the study.
  • 7. Patients who are HIV positive may not be enrolled.

Ages Eligible for Study

to 21 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Baylor College of Medicine,

Olive Eckstein, MD, STUDY_CHAIR, Baylor College of Medicine

Study Record Dates

2029-01