Evaluation of Efficacy and Tolerance of Cladribine in Symptomatic Pulmonary Langerhans Cell Histiocytosis

Phase 2

• Conditions: Langerhans Cell Histiocytosis of Lung
• Interventions: Drug: Cladribine

• Registration Number: NCT01473797
• Lead Sponsor: Assistance Publique - Hôpitaux de Paris

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.

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Study First Submitted: 2011-10-14
• Study Start: 2011-11
• Study First Submitted QC: 2011-11-16
• Study First Posted: 2011-11-17
• Primary Completion: 2018-04
• Status Verified: 2021-02
• Last Update Submitted: 2021-02-17
• Last Update Posted: 2021-02-18
• Study Completion: 2022-04

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 10

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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
cladribine	Cladribine	-

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
Variation of nodular and cystic semiquantitative scores in High Resolution Computed Tomography (HRCT)	6 months
Incidence of grade 3 or 4 side effects	6 months
Grade 3 or 4 neutropenia or thrombopenia	6 months
Incidence of infection	6 months
Incidence of secondary malignant disease	4 years
Treatment response	12 months
Absolute variations of FEV1, FVC, residual volume (RV), and Diffusing capacity of the lung for carbon monoxide (DLCO), (expressed in mL)	6 months
Incidence of pneumothorax	12 months
Mortality	12 months, 4 years
Responses to treatment	3 months
Response to treatment of extra pulmonary localizations of the Langerhans disease	6, 9, and 12 months

Trial Locations

Locations (1)

Saint Louis hospital; 🇫🇷 Paris, France