Phase II Study of Clofarabine in Patients With Recurrent or Refractory Langerhans Cell Histiocytosis

Status: Recruiting
Phase: Phase 2
DFCI Protocol ID: 15-005

This research study is evaluating a drug called clofarabine as a possible treatment for Langerhans Cell Histiocytosis( LCH )

Conducting Institutions:

Dana-Farber Cancer Institute, Children's Hospital Boston, Brigham and Women's Hospital, Massachusetts General Hospital

Overall PI:

Barbara Degar, MD, Dana-Farber Cancer Institute

Site-responsible Investigators:

David Ebb, MD, Massachusetts General Hospital

Contacts:

Dana-Farber Cancer Institute: Childrens Hospital Pediatric Clinical Translation Investigation Program CTIP, ctip@partners.org
Massachusetts General Hospital: Cancer Trials Call Center, 877-789-6100

Eligibility Criteria

Inclusion Criteria:

  -  Prior diagnosis of Langerhans Cell Histiocytosis established by standard diagnostic
     criteria and confirmed histologically

  -  Evidence of disease reactivation or progression after standard treatment against LCH
     (histological confirmation is not required)

  -  Performance Score > 70% (use Lansky score for age < 16 and Karnofsky score for age =
     >16).

  -  Patients of all ages will be eligible.

  -  Provide signed written informed consent.

  -  Patients have failed first line treatment with prednisone and vinblastine. There is
     no limitation of amount or the type of prior therapy or drugs.

  -  Patients with clinical evidence of involvement of hematopoietic system, liver or
     spleen, have failed salvage treatment with cladribine/cytarabine or are not
     considered to be eligible for such treatment.

  -  Female patients of childbearing potential must have a negative serum pregnancy test
     within 14 days prior to enrollment. Male and female patients must use an effective
     contraceptive method during the study and for a minimum of 6 months after study
     treatment.

  -  Capable of understanding the investigational nature, potential risks and benefits of
     the study, and able to provide valid informed consent.

  -  Participants must have adequate marrow functions as defined below, except those with
     involvement of hematopoietic system for whom these criteria can be waived:

       -  Absolute neutrophil count ≥ 750/µL

       -  Platelets ≥75,000/µL

  -  Participants must have adequate organ functions as defined below:

       -  Total bilirubin ≤ 2.5x institutional upper limit of normal

       -  AST (SGOT)/ALT (SGPT) < 2.5 X institutional upper limit of normal unless it is
  related to involvement by LCH

       -  Adequate renal function defined as:

    -  A serum creatinine ≤ institutional upper limit of normal or

    -  Creatinine clearance or radioisotope GFR ≥60 mL/min/1.73 m2

       -  Alkaline phosphatase ≤ 2.5 x institutional upper limit of normal

Exclusion Criteria:

  -  Participants who have had chemotherapy or radiotherapy within 2 weeks (6 weeks for
     nitrosoureas or mitomycin C) prior to entering the study or those who have not
     recovered from adverse events due to agents administered more than 2 weeks earlier.

  -  Participants may not be receiving any other investigational agents targeting LCH.

  -  Use of alternative medications (e.g., herbal or botanical that could interfere with
     clofarabine) is not permitted during the entire study period.

  -  Uncontrolled intercurrent illness including, but not limited to ongoing or active
     infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac
     arrhythmia, or psychiatric illness/social situations that would limit compliance with
     study requirements.

  -  Pregnant women are excluded from this study because clofarabine has potential for
     teratogenic or abortifacient effects. Because there is an unknown but potential risk
     of adverse events in nursing infants, breastfeeding should be discontinued if the
     mother is treated with clofarabine.

  -  Individuals with a history of a different malignancy are ineligible except for the
     following circumstances. Individuals with a history of other malignancies are
     eligible if they have been disease-free for at least 5 years and are deemed by the
     investigator to be at low risk for recurrence of that malignancy. Individuals with
     the following cancers are eligible if diagnosed and treated within the past 5 years:
     cervical cancer in situ, and basal cell or squamous cell carcinoma of the skin.

  -  Patients with a history of prior hematopoietic stem cell transplantation(HSCT),
     elevated conjugated serum bilirubin at study entry, uncontrolled systemic fungal,
     bacterial, or other infection, a history of hepatitis B or C infection or a history
     of cirrhosis.

  -  Individuals who are known to be HIV-positive on combination antiretroviral therapy.

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