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Protocol No. UW16132

Principal Investigator Puccetti, Diane

Phase II/III

Age Group Children

Scope National

Sponsor Type National

Title LCH-IV: International Collaborative Treatment Protocol for Children and Adolescents with Langerhans Cell Histiocytosis

Objective In this study, researchers want to find out if prolonging the duration of chemotherapy to 12 months will be better than 6 months at preventing LCH reactivation and disease-related complications and to learn more about the side effects of chemotherapy. Patients who do not respond to this treatment will be offered alternative risk adapted treatments(e.g. Straum II)

Treatment All participants in this study will first receive 2 drugs called vinblastine and prednisone over a 6 week period for the first course of treatment, at week 7, you will come to the clinic for an exam, imaging scans, and laboratory tests to find out how your disease responded to treatment. The results of this evaluation will determine the next step of treatment

Description The LCH-IV is an international, multicenter, prospective clinical study for pediatric Langerhans Cell Histiocytosis LCH (age < 18 years)

Key Eligibility Stratum I
  • Patients must be less than 18 years of age at the time of diagnosis.
  • Patients must have histological verification of the diagnosis of Langerhans cell histiocytosis according to the protocol criteria
    Stratum II
  • Patients of Stratum I who have:
  • Progressive disease (AD worse) in non-risk organs after 6 weeks (Initial Course AD intermediate or worse in non-risk organs or AD better in risk organs after 12 weeks (Initial Course 2)
  • Disease progression (AD worse) in non-risk organs at any time during continuation treatment Active disease at the end of Stratum I treatment
  • Disease reactivation in non-risk organs at any time after completion of Stratum I treatment
    Stratum III
  • Patients from Stratum I who fulfill the following criteria:
  • AD worse in risk organs after week 6 (after Initial Course 1), or AD worse or AD intermediate in risk organs after week 12 (after Initial Course 2).
  • Presence of unequivocally severe organ dysfunction at the above mentioned evaluation points (hematological dysfunction, liver dysfunction, or both of them) as described by the protocol
    Stratum IV
  • Patients from Stratum I or Stratum III who fulfill the following criteria:
  • AD worse in risk organs after week 6 (after Initial Course 1), or AD worse or AD intermediate in risk organs after week 12 (after Initial Course 2) of Stratum I OR AD worse after the 2nd and 3rd 2-CdA/Ara-C course, and those AD worse or AD intermediate after the 4th 2-CdA/Ara-C course of Stratum III AND Presence of unequivocally severe organ dysfunction at the above mentioned evaluation points (hematological dysfunction, liver dysfunction, or both of them)
  • Adequate organ function: Patients should have adequate hepatic, renal, cardiac and pulmonary function to undergo reduced intensity HCT based upon local institutional guidelines, or at a minimum meet requirements noted in eligibility checklist. However, significant hepatic and pulmonary dysfunction, if secondary to underlying LCH disease activity, will not exclude patients from protocol enrollment and should be discussed with the National PI Coordinator and the Coordinating Principal Investigator.
    Stratum V
  • All patients with verified diagnosis of LCH and MRI findings consistent with ND-CNSLCH irrespective of previous treatments (also those not registered to other Strata ofLCH-IV)
  • Patients with isolated tumorous CNS-LCH (including isolated DI with mass lesion in the hypothalamus-pituitary axis). In patients with already established diagnosis of LCH and radiologic finding of CNS lesions compatible with LCH, a biopsy of the lesion is not obligatory. In all other cases a biopsy of the lesion is needed for inclusion into the study
    Stratum VI -- Patients with newly diagnosed SS-LCH and localization other than "multifocal bone",isolated tumorous CNS lesion, or isolated "CNS-risk" lesion
    Stratum VII -- All patients registered in LCH IV (regardless of treatment) as long as consent for longterm follow-up has not been withheld
    EXCLUSION:
  • Stratum I
  • Pregnancy (patients of child-bearing age must be appropriately tested before chemotherapy)
  • LCH-related permanent consequences (e.g. vertebra plana, sclerosing cholangitis, lung fibrosis, etc.) in the absence of active disease
  • Prior systemic therapy
    Stratum II
  • Patients with progressive disease in risk organs
    Stratum III
  • Isolated sclerosing cholangitis without evidence of active hepatic

  • Applicable Disease Sites Brain/Central Nervous System; Endocrine cancers; Hematologic cancers, other; Liver; Lung; Melanoma/Skin cancer; Sarcoma

    Status Open

    Participating Institutions UW Hospital and Clinics