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

Primary Purpose

Langerhans Cell Histiocytosis of Lung

Status
Unknown status
Phase
Phase 2
Locations
France
Study Type
Interventional
Intervention
Cladribine
Sponsored by
Assistance Publique - Hôpitaux de Paris
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Langerhans Cell Histiocytosis of Lung

Eligibility Criteria

16 Years - 55 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Saint Louis hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

cladribine

Arm Description

Outcomes

Primary Outcome Measures

Cumulated incidence of response to treatment
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

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

Full Information

First Posted
October 14, 2011
Last Updated
February 17, 2021
Sponsor
Assistance Publique - Hôpitaux de Paris
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1. Study Identification

Unique Protocol Identification Number
NCT01473797
Brief Title
Evaluation of Efficacy and Tolerance of Cladribine in Symptomatic Pulmonary Langerhans Cell Histiocytosis
Acronym
ECLA
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

2. Study Status

Record Verification Date
February 2021
Overall Recruitment Status
Unknown status
Study Start Date
November 2011 (undefined)
Primary Completion Date
April 2018 (Actual)
Study Completion Date
April 2022 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Assistance Publique - Hôpitaux de Paris

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Langerhans Cell Histiocytosis of Lung

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Actual)

8. Arms, Groups, and Interventions

Arm Title
cladribine
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Cladribine
Intervention Description
Subcutaneous injections, 0,1 mg/kg/day for 5 days, one course per month for 4 months
Primary Outcome Measure Information:
Title
Cumulated incidence of response to treatment
Description
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
Time Frame
6 months
Secondary Outcome Measure Information:
Title
Responses to treatment
Time Frame
3 months
Title
Absolute variations of FEV1, FVC, residual volume (RV), and Diffusing capacity of the lung for carbon monoxide (DLCO), (expressed in mL)
Time Frame
6 months
Title
Grade 3 or 4 neutropenia or thrombopenia
Time Frame
6 months
Title
Incidence of infection
Time Frame
6 months
Title
Incidence of grade 3 or 4 side effects
Time Frame
6 months
Title
Response to treatment of extra pulmonary localizations of the Langerhans disease
Time Frame
6, 9, and 12 months
Title
Incidence of pneumothorax
Time Frame
12 months
Title
Mortality
Time Frame
12 months, 4 years
Title
Incidence of secondary malignant disease
Time Frame
4 years
Title
Treatment response
Time Frame
at 6 months
Title
Treatment response
Time Frame
9 months
Title
Treatment response
Time Frame
12 months
Title
Variation of nodular and cystic semiquantitative scores in High Resolution Computed Tomography (HRCT)
Time Frame
6 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
16 Years
Maximum Age & Unit of Time
55 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Abdellatif TAZI, MD, PhD
Organizational Affiliation
Assistance Publique - Hôpitaux de Paris
Official's Role
Principal Investigator
Facility Information:
Facility Name
Saint Louis hospital
City
Paris
ZIP/Postal Code
75010
Country
France

12. IPD Sharing Statement

Learn more about this trial

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

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