|- candidate number||3062|
|- NTR Number||NTR1216|
|- ISRCTN||ISRCTN wordt niet meer aangevraagd|
|- Date ISRCTN created|
|- date ISRCTN requested|
|- Date Registered NTR||13-mrt-2008|
|- Secondary IDs||2007/169 MEC VU University Medical Center Amsterdam|
|- Public Title||Treatment protocol of the first international study for Langerhans Cell Histiocytosis in Adults|
|- Scientific Title||Treatment protocol of the first international study for Langerhans Cell Histiocytosis in Adults|
|- ACRONYM||LCH-A1 |
|- hypothesis||Improvement of standardization in diagnosis and treatment of adult LCH|
|- Healt Condition(s) or Problem(s) studied||Langerhans cell histiocytosis|
|- Inclusion criteria||1. definitive diagnosis of LCH |
2. no prior cytoreductive treatment for LCH
3. age 18-50 years for group 1 and 2
4. age 18-75 years for group 3
|- Exclusion criteria||1. patients with severe impairment of clinical condition including severely impaired pulmonary function, long term oxygen therapy or cor pulmonale. |
2. treatment with immune suppressive agents and/or bisphosphonates within 4 weeks from baseline evaluation
|- mec approval received||yes|
|- multicenter trial||yes|
|- Type||2 or more arms, randomized|
|- planned startdate ||5-okt-2007|
|- planned closingdate||1-okt-2011|
|- Target number of participants||1200|
|- Interventions||- Patients in group 1 (single system disease at risk): treatment with Prednisone, Vinblastine and Mercaptopurine. |
Initial treatment: Prednisone 1 mg/kg/day ( not to exceed 60 mg) as a 4-week course, tapering over a period of 2 weeks.
Vinblastine 6 mg/m2 iv bolus (not to exceed 10 mg), day 1,8,15,22,29,36.
Continuation treatment: starting at day 43 after initial treatment. Mercaptopurine: 30 mg/m2(not to exceed 50 mg) daily until completion of treatment. Prednisone: 1 mg/kg/day (not to exceed 60 mg) day 1-5 every 3 weeks until completion of treatment.
Vinblastine: 6 mg/m2 iv bolus (not to exceed10 mg) day 1 every 3 weeks until completion of treatment. (starting 3 weeks after the last vinblastine injection of the initial treatment.
total duration of treatment is 6 months.
- Patients in group 2, multisystem LCH:treatment with Prednisone, Vinblastine and Mercaptopurine for 6 months versus treatment with Prednisone, Vinblastine and Mercaptopurine for 12 months.
Intervention for group 2 is the same as for group 1; total duration of treatment will be the object of randomization: 6 months vs 12 months.
- Patients in group 3 (isolated pulmonary disease): an observational period of 6 months afer cigarette smoke withdrawal. In case of progression of the symptoms of pulmonary dysfunction, treatment phase will be started with Prednisone monotherapy for 6 monthsat the following dosage:
- 1mg/kg/day (not to exceed 60 mg), daily for 1 month.
- 0.5 mg/kg/day, daily for 1 month.
- 0.25 mg/kg/day daily for 2 months.
- 0.125 mg/kg/day daily for 2 months.
|- Primary outcome||Definition and implementation of an uniform treatment for patients with single system LCH, multisystem LCH and pulmonary isolated LCH; implementation of uniform initial evaluation and stratification criteria.|
|- Secondary outcome||- to explore the therapeutic efficacy on adult patients of the standard regimen for multisystem LCH in children, i.e. the combination of vinblastine and prednisone.
Endpoints will be survival, reactivation-free survival, permanent sequelae.|
- to examine if an extended continuation therapy may reduce disease reactivations occuring after treatment completion in patients with multisystem LCH.
- to describe the natural history of isolated pulmonary disease and in particular the role of smoking cessation on the disease course.
- to explore the therapeutic efficacy of steroid monotherapy in adult patients with isolated pulmonary disease showing disease progression.
|- Timepoints||pre-treatment, at week 6 and every 3 months during treatment (i.e. month 3, month 6 and 9 when appropriate) and then at treatment completion.
thereafter every 6 months for the first 3 years. and once a year during the following 3 years.|
|- Trial web site||N/A|
|- status||open: patient inclusion|
|- CONTACT FOR PUBLIC QUERIES||MD, PhD A.A. Loosdrecht, van de|
|- CONTACT for SCIENTIFIC QUERIES||MD, PhD A.A. Loosdrecht, van de|
|- Sponsor/Initiator ||VU University Medical Center, Department of Hematology|
(Source(s) of Monetary or Material Support)
|VU University Medical Center, Department of Hematology|
|- Brief summary||LCH-A1 study is an international multicenter study for Langerhans Cell Histiocytosis.
This is a rare, tumor-like disease thas has an unpredicatable course and can be fatal. The cause of this disease is unknown.
The targeted number of participants is 1200 patients; for the Netherlands the targeted number is 20 patients.
The study is designed for 3 groups of patients.
Patient population in group 1 are patients with single system multifocal bone lesions or localized special site involvement.
Group 2 consists of patients with multisystem disease and group 3 consists of patients with isolated pulmonary disease.
Treatment for group 1 is 6 months of treatment with Prednisone, Vinblastine and Mercaptopurine.
Treatment for group 2 is the same as in group 1. Patients are randomized for 6 months treatment versus 12 months.
Treatment in group 3 is an observation phase for 6 months after smoking cessation. In case of progression of the symptoms or pulmonary dysfunction 6 months steroid monotherapy is given.
Duration of treatment is for all patients 6 months till 12 months and a follow up period of 6 years.|
|- Main changes (audit trail)|
|- RECORD||12-feb-2008 - 13-mrt-2008|