Langerhans Cell Histiocytosis of Lung
Conditions
Brief summary
ECLA is a phase II, multicenter study testing sub cutaneous cladribine 0.1mg/kg/j during 5 days, administrated every month for 4 courses, in symptomatic adult patients with pulmonary Langerhans cell histiocytosis and impairment of lung function patients.
Detailed description
ECLA is a phase II, multicenter study testing sub cutaneous cladribine 0.1mg/kg/j during 5 days, administrated every month for 4 courses, in symptomatic adult patients with pulmonary Langerhans cell histiocytosis and impairment of lung function patients.
Interventions
DRUGCladribine
Subcutaneous injections, 0,1 mg/kg/day for 5 days, one course per month for 4 months
Sponsors
Assistance Publique - Hôpitaux de Paris
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
16 Years to 55 Years
Healthy volunteers
No
Inclusion criteria
* Age 16 to 55 yr * Histologically proven pulmonary Langerhans cell histiocytosis ( patients with presumptive diagnosis whose lung function precludes lung biopsy may be included after revision of their medical record at the national reference center for Langerhans cell histiocytosis) * Symptomatic pulmonary Langerhans cell histiocytosis (NYHA dyspnea class ≥2) with: * irreversible airflow obstruction (FEV1/FVC\<70%) with postbronchodilator FEV1 comprised between 30 and 70% of predicted * and/or decrease ≥15% in FEV1, FVC or DLCO as compared to baselines values in the year preceding the inclusion * Signed written informed consent
Exclusion criteria
* Women at childbearing age without adequate contraception or wishing breastfeeding * Male without adequate contraception during the study * Dyspnea due to severe pulmonary arterial hypertension (PAP≥35mmHg) confirmed by cardiac right catheterism * Previous malignancy * Current infectious disease * Renal failure * Liver failure * Severe alteration of lung * Hematologic disease unrelated to Langerhans cell histiocytosis * Epilepsy * Hepatic, spleen or hematology involvement by Langerhans cell histiocytosis * Pneumothorax within a month previously to inclusion * Previous treatment with cladribine * Contra indication to the use of cladribine * Previous myelosuppressive treatment * Simultaneous participation to another interventional clinical trial
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Cumulated incidence of response to treatment | 6 months | response to treatment after 6 months is defined as * ≥10% improvement of forced vital capacity (FVC) * and/or ≥10% improvement of postbronchodilator forced expiratory volume (FEV1) and ≥200ml |
Secondary
| Measure | Time frame |
|---|---|
| Absolute variations of FEV1, FVC, residual volume (RV), and Diffusing capacity of the lung for carbon monoxide (DLCO), (expressed in mL) | 6 months |
| Grade 3 or 4 neutropenia or thrombopenia | 6 months |
| Incidence of infection | 6 months |
| Incidence of grade 3 or 4 side effects | 6 months |
| Response to treatment of extra pulmonary localizations of the Langerhans disease | 6, 9, and 12 months |
| Responses to treatment | 3 months |
| Mortality | 12 months, 4 years |
| Incidence of secondary malignant disease | 4 years |
| Treatment response | at 6 months |
| Variation of nodular and cystic semiquantitative scores in High Resolution Computed Tomography (HRCT) | 6 months |
| Incidence of pneumothorax | 12 months |
Countries
France
Outcome results
None listed