CH-IV Treatment Protocol for Children and Adolescents withLANGERHANS CELL HISTIOCYTOSIS
- Conditions
- angerhans cell histiocytosisTherapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2011-001699-20-GR
- Lead Sponsor
- St. Anna Kinderkrebsforschung
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 1400
•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 criteria described in Section 6.1,
Patients with reproductive potential must agree to use effective contraception during the period of therapy,
Signed informed consent
•Stratum II
Patients of Stratum I who have progressive disease (AD worse) in non-risk organs after Initial Course 1,
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,
Disease reactivation in risk-organs, who do not meet organ dysfunction criteria (acc. table XI) at any time or after completion of Stratum I Treatment,
Patients with reproductive potential must agree to use effective contraception during the period of therapy
•Stratum III
Patients from Stratum I who fulfill the following criteria:
AD intermediate or 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 (hematological dysfunction, liver dysfunction, or both of them) as defined in Table XI,
Patients with reproductive potential must agree to use effective contraception during the period of therapy
•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 (hematological dysfunction, liver dysfunction, or both of them) as defined in Table XI (see Section 10.3.1),
Signed informed consent,
Adequate organ function,
Presence of unequivocally severe organ dysfunction (hematological dysfunction, liver dysfunction, or both of them),
Patients with reproductive potential must agree to us effective contraception during the period of therapy
•Stratum V
All patients with verified diagnosis of LCH and MRI findings consistent with ND- CNS-LCH irrespective of previous treatments,
OR
Patients with isolated tumorous CNS-LCH (including isolated DI with mass lesion in the hypothalamus-pituitary axis),
Patients with reproductive potential must agree to use effective contraception during the period of therapy
•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 long- term follow-up has not been withheld
Are the trial subjects under 18? yes
Number of subjects for this age range: 1400
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
•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, except systemic steroids given up to one week
•Stratum II
Patients with progressive disease in risk organs,
Permanent consequences (e.g. sclerosing cholangitis, lung fibrosis, etc.) without evidence of active LCH in the same organ or in any other locations,
No written consent of the patient or his/her parents or legal guardian
•Stratum III
Isolated sclerosing cholangitis without evidence of active hepatic LCH as the only evidence of risk organ involvement,
Inadequate renal function as defined by serum creatinine > 3x normal for age
•Stratum IV
Pulmonary failure (requiring mechanical ventilation) not due to active LCH,
Isolated liver sclerosis or pulmonary fibrosis, without active LCH,
Uncontrolled active life-threatening infection.
Decreased renal function with a GFR of less than 50ml/1.73m2/min,
Pregnancy or active breast feeding,
Failure to provide signed informed consent
•Stratum V
Patients without any evidence of isolated tumorous CNS-LCH lesions or ND-CNS lesions
•Stratum VI
Patients with single-system LCH who have an isolated tumorous CNS lesion (eligible for Stratum V),
Patients with isolated CNS-risk or multifocal bone lesion (eligible for Stratum I, Group 2)
•Stratum VII
no consent for long-term follow up available
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method