Langerhans cell Histiocytosis (LCH)
Conditions
Interventions
Trade Name: e.g. Decortin, Predni Tablinen, different genercis
Pharmaceutical Form: Tablet
INN or Proposed INN: Prednison
Other descriptive name: PREDNISONE
Concentration unit: mg/m2 milligram(s)/squa
Sponsors
Johann Wolfgang Goethe-University Frankfurt am Main
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Stratum I: •Age: Patients must be less than 18 years of age at the time of enrollment. •No systemic therapy for LCH prior to the standardized induction therapy, e.g., prior to Initial Course 1 (IC-1) and, eventually, Initial Course 2 (IC-2) (pre-study) •Evaluation demonstrates non-active disease after IC-1 (independent of involvement of risk organs) or non-active disease or active disease better (only for patients without involvement of risk organs) after IC-2 [imaging studies need to be referenced regarding treatment response to initial course(s) (pre-study)] Stratum II: •Age: Patients must be older than 2 years and less than 18 years of age at the time of enrollment •No systemic therapy for progression/relapse of LCH prior to the standardized second line therapy, e.g., standardized 24-week second-line initial course (SL-IT), which is NOT part of the study •Evaluation demonstrates non-active disease or active disease better after SL-IT [imaging studies need to be referenced regarding treatment response to second-line initial course (pre-study)]. Both Strata: •Patients must have a referenced histological verification of the diagnosis of LCH (pre-study) •Male subjects able to father children and female subjects of childbearing potential and at risk for pregnancy must agree to use a highly effective method of contraception (e.g., barrier contraception for males, contraceptive pill (“Pill”) for female) throughout the study and for at least 28 days after the last dose of assigned study treatment. •Signed informed consent form Are the trial subjects under 18? yes Number of subjects for this age range: 300 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
Exclusion criteria
Exclusion criteria: Stratum I: •Patients with any active disease after IC-1 or patients with active disease intermediate/worse (for patients without involvement of risk organs) or any active disease (for patients with involvement of risk organs) after Initial Course I Stratum II: •Patients with progressive disease in risk organs •Patients with active disease intermediate/worse after second-line intensive course •Patient suffers from Crohn´s disease or Colitis ulcerosa •History of asthma, urticaria, or other allergic-type reactions after taking acetylsalicylic acid or other non-steroidal anti-inflammatory drugs For both Strata: •Allergy for one of the trial drugs •Pregnancy (patients of child-bearing age must be appropriately tested before chemotherapy) •Breastfeeding •History of severe bleeding or ulcera of the gastrointestinal tract or other relevant bleeding disorders •Participation in other clinical trials according to German drug law (§§40ff AMG)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To reduce reactivation rates and permanent consequences in MS-LCH (Group 1) through prolongation (12 vs. 24 months) and intensification [+/- Mercaptopurine (MP)] of continuation treatment (2x2 factorial randomized trial) To reduce reactivation rates and permanent consequences in a subset of SS-LCH (multifocal bone or isolated “CNS-Risk” lesions (Group 2) through prolongation (6 vs. 12 months) of continuation therapy (randomized trial) To investigate the value continuation therapy (randomized: 24 months of indomethacin vs. MP/MTX) in patients with non-risk organ LCH (both non-responders to first-line regimen and those who experience disease reactivation in non-risk organs after its completion) with respect to achievement of complete disease resolution, prevention of further reactivations and permanent consequences In each treatment arm of both Strata, also overall survival, the incidence of reactivation in risk organs and treatment-related toxicity will be assessed ;Secondary Objective: not applicable;Primary end point(s): Reactivation-free survival;Timepoint(s) of evaluation of this end point: One interim-analysis will be done after 4 years | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Overall survival Incidence of permanent consequences The proportion of patients alive and free of disease without permanent consequences Cumulative incidence of reactivations in risk organs Treatment-related toxicity ;Timepoint(s) of evaluation of this end point: One interim-analysis will be done after 4 years | — |
Countries
Germany
Contacts
Public ContactLCH-Studienzentrale
Universitätsklinikum Frankfurt | Klinik für Kinder- und Jugendmedizin
Outcome results
None listed