Actively Recruiting
Randomized Study Comparing Cytarabine Monotherapy Versus Standard Vinblastine/Prednisone for Frontline Treatment of Langerhans Cell Histiocytosis
Led by Baylor College of Medicine · Updated on 2025-09-11
124
Participants Needed
11
Research Sites
104 weeks
Total Duration
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AI-Summary
What this Trial Is About
Langerhans Cell Histiocytosis (LCH) is a cancer affecting certain immune cells that can cause tissue damage or lesions in different parts of the body. This research compares two chemotherapy treatments: the standard vinblastine with prednisone versus single-agent cytarabine, aiming to find if cytarabine alone is an effective frontline therapy for LCH. The study also examines how PET scans can better detect LCH involvement and how genetic and protein markers in patients relate to treatment response. Participants will be randomly assigned to receive either vinblastine with prednisone or cytarabine. Cytarabine treatment involves five-day cycles repeated every 21 days for several cycles, followed by continuation therapy lasting up to one year with periodic scans and biopsies. The vinblastine/prednisone group receives oral prednisone and weekly vinblastine infusions, with continuation therapy involving additional medications for high-risk patients. Both treatments include regular scans and bone marrow biopsies if needed, with treatment adjustments based on disease response. During the study, participants will have regular exams, blood and urine tests, biopsies, and imaging such as x-rays, ultrasounds, CT, MRI, and PET scans to monitor disease and treatment effects. Follow-up visits continue for five years after therapy completion, with ongoing evaluations to assess disease status, side effects, and biomarker changes. The main outcome measured is the time to one-year event-free survival, with additional assessments on long-term responses, toxicity, disease activity, and genetic markers.
CONDITIONS
Brief Title
Vinblastine/Prednisone Versus Single Therapy With Cytarabine for Langerhans Cell Histiocytosis (LCH)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patient must have biopsy-confirmed diagnosis of Langerhans cell histiocytosis.
- Patient must be between 0 and 21 years of age.
- Patient must have a Karnofsky or Lansky performance score of 50% or higher.
You will not qualify if you...
- Patient may not have received prior systemic chemotherapy for LCH or any other cancer except short-term steroid pretreatment within defined limits.
- Disease limited to a single skin or bone site is excluded except for CNS-risk or special site lesions, functionally critical lesions with approval, or asynchronous multisite presentation.
- Patient may not have severe kidney disease (creatinine >3 times normal or clearance <50 ml/m2/1.73m2).
- Patient may not have severe liver disease (direct bilirubin >3 mg/dl or AST >500 IU/L) unless due to LCH.
- Female patients who are pregnant or breastfeeding are excluded.
- Patients of reproductive potential must agree to use birth control during the study.
- Patients who are HIV positive are not eligible.
- Patients excluded for lab abnormalities or performance score may be enrolled with special approval.
AI-Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 52 weeks
Participants receive assigned chemotherapy treatment with either cytarabine or vinblastine/prednisone. Treatment includes multiple cycles of medication given intravenously or by mouth according to the assigned group, with scans and tests to monitor response.
Regular visits before each treatment cycle and scans during weeks 6, 12, 24, and end of therapy
Duration - 5 years
Participants are followed for 5 years after completing therapy to monitor health and disease status with periodic exams, blood tests, and scans.
Visits at 1, 3, 6, 9, 12, 18, 24 months, then yearly up to 5 years after treatment
Trial Site Locations
Total: 11 locations
1
Stanford Children's Hospital, Lucile Packard Children's Hospital
Palo Alto, California, United States, 94304
Actively Recruiting
2
Rady Children's Hospital - San Diego
San Diego, California, United States, 92123
Actively Recruiting
3
University of Minnesota/Masonic Cancer Center
Minneapolis, Minnesota, United States, 55455
Withdrawn
4
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Actively Recruiting
5
Lehigh Valley Health Network- Cedar Crest
Allentown, Pennsylvania, United States, 18103
Actively Recruiting
6
Dell Children's Medical Center
Austin, Texas, United States, 78723
Actively Recruiting
7
Cook Children's Health Care System
Fort Worth, Texas, United States, 76104
Actively Recruiting
8
Texas Children's Hospital
Houston, Texas, United States, 77030
Actively Recruiting
9
Vannie Cook Children's Clinic
McAllen, Texas, United States, 78503
Actively Recruiting
10
Children's Hospital of San Antonio
San Antonio, Texas, United States, 78207
Actively Recruiting
11
Children's Hospital of The King's Daughters
Norfolk, Virginia, United States, 23507
Actively Recruiting
Research Team
O
Olive Eckstein, MD
C
Carl E. Allen, MD, PhD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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