Clinical trial NCT01473797: Evaluation of Efficacy and Tolerance of Cladribine in Symptomatic Pulmonary Langerhans Cell Histiocytosis
Official title: Evaluation of Efficacy and Tolerance of Cladribine in Symptomatic Patients With Pulmonary Langerhans Cell Histiocytosis and Impairment of Lung Function
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Langerhans Cell Histiocytosis of Lung
Lead sponsor: Assistance Publique - Hôpitaux de Paris
Sponsor class: OTHER
First posted: 2011-11-17
Enrollment: 10
Sex: ALL
Minimum age (years): 16
Maximum age (years): 55
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Cumulated incidence of response to treatment
timeFrame: 6 months
Secondary outcomes:
measure: Responses to treatment
timeFrame: 3 months
measure: Absolute variations of FEV1, FVC, residual volume (RV), and Diffusing capacity of the lung for carbon monoxide (DLCO), (expressed in mL)
timeFrame: 6 months
measure: Grade 3 or 4 neutropenia or thrombopenia
timeFrame: 6 months
measure: Incidence of infection
timeFrame: 6 months
measure: Incidence of grade 3 or 4 side effects
timeFrame: 6 months
measure: Response to treatment of extra pulmonary localizations of the Langerhans disease
timeFrame: 6, 9, and 12 months
measure: Incidence of pneumothorax
timeFrame: 12 months
measure: Mortality
timeFrame: 12 months, 4 years
measure: Incidence of secondary malignant disease
timeFrame: 4 years
measure: Treatment response
timeFrame: at 6 months
measure: Treatment response
timeFrame: 9 months
measure: Treatment response
timeFrame: 12 months
measure: Variation of nodular and cystic semiquantitative scores in High Resolution Computed Tomography (HRCT)
timeFrame: 6 months
Other outcomes:
Locations:
city: Paris
country: France
facility: Saint Louis hospital
Study officials:
affiliation: Assistance Publique - Hôpitaux de Paris
name: Abdellatif TAZI, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: