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

Conditions

• Langerhans Cell Histiocytosis

Eligibility Criteria

Sex

ALL

Age

N/A - 21 Years

Healthy Volunteers

Not accepted

Interventions

• Cytarabine — DRUG
  Cytarabine 100 mg/m\^2/day IV for five consecutive days. This five-day cycle will be repeated every 21 days for a total of four cycles for all patients regardless of response. Each new cycle may not begin until absolute neutrophil count (ANC) is ≥ 750/mcL and platelet count is ≥ 75,000/mcL.
• Vinblastine/prednisone — DRUG
  Vinblastine/Prednisone +/- 6-mercaptopurine based on risk category. Patients with high-risk organ involvement (liver, spleen, hematopoeitic system) will receive 6-mercaptopurine during Continuation Therapy as this is the current standard of care treatment. Vinblastine 6 mg/m\^2/dose IV push weekly for patients ≥ 12 months of age. Vinblastine will be dosed at 3 mg/m\^2/dose for patients under 6 months of age, and dosed at 4.5 mg/m\^2/dose for patients 6 months of age to 11.99 months of age. Prednisone (or prednisolone) 20 mg/m2/dose by mouth twice a day

Study Details

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.

Key Dates

Start date

Mar 7, 2016

Status verified

Sep 2025

Primary completion

Jan 31, 2027

Completion

Jan 31, 2029

Study Design

Enrollment

124 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Cytarabine ("experimental") arm
  On this arm, patients will receive single therapy with cytarabine.
• Active Comparator: Vinblastine/prednisone ("standard") arm
  On this arm, patients will receive standard-of-care therapy with vinblastine and prednisone.

Primary Outcome Measure

Time to determine 1-year event-free survival (EFS) of patients treated with cytarabine monotherapy for LCH, compared directly with that of standard-of-care vinblastine/prednisone (Events include progression of LCH, relapse, or death). [ Time Frame: up to 60 months ]

Central Contacts

• Olive Eckstein, MD
  832-822-4242
  [email protected]
• Carl E. Allen, MD, PhD
  832-826-0860
  [email protected]

Locations (11)

Find similar trials in Palo Alto, CA

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