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

• Sponsor: Assistance Publique - Hôpitaux de Paris (Other)
• Overall Status: Active, not recruiting
• CT.gov ID: NCT01473797
• Collaborator: (none)
• Enrollment: 10
• Location: 1
• Arm: 1
• Duration (Months): 125
• Patients Per Site Per Month: 0.1

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
Langerhans Cell Histiocytosis of Lung	Drug: Cladribine	Phase 2

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.

Study Design

• Study Type: Interventional
• Actual Enrollment: 10 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Evaluation of Efficacy and Tolerance of Cladribine in Symptomatic Patients With Pulmonary Langerhans Cell Histiocytosis and Impairment of Lung Function
• Study Start Date: Nov 1, 2011
• Actual Primary Completion Date: Apr 1, 2018
• Anticipated Study Completion Date: Apr 1, 2022

Arms and Interventions

Arm	Intervention/Treatment
Experimental: cladribine	Drug: Cladribine; Subcutaneous injections, 0,1 mg/kg/day for 5 days, one course per month for 4 months

Outcome Measures

Primary Outcome Measures

1. 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

1. Responses to treatment [3 months]

2. Absolute variations of FEV1, FVC, residual volume (RV), and Diffusing capacity of the lung for carbon monoxide (DLCO), (expressed in mL) [6 months]

3. Grade 3 or 4 neutropenia or thrombopenia [6 months]

4. Incidence of infection [6 months]

5. Incidence of grade 3 or 4 side effects [6 months]

6. Response to treatment of extra pulmonary localizations of the Langerhans disease [6, 9, and 12 months]

7. Incidence of pneumothorax [12 months]

8. Mortality [12 months, 4 years]

9. Incidence of secondary malignant disease [4 years]

10. Treatment response [at 6 months]

11. Treatment response [9 months]

12. Treatment response [12 months]

13. Variation of nodular and cystic semiquantitative scores in High Resolution Computed Tomography (HRCT) [6 months]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 16 Years to 55 Years
• Sexes Eligible for Study: All
• Accepts 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Saint Louis hospital	Paris		France	75010

Sponsors and Collaborators

• Assistance Publique - Hôpitaux de Paris

Investigators

• Principal Investigator: Abdellatif TAZI, MD, PhD, Assistance Publique - Hôpitaux de Paris

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Assistance Publique - Hôpitaux de Paris
• ClinicalTrials.gov Identifier: NCT01473797
• Other Study ID Numbers: AOM10182; 2010-023344-32
• First Posted: Nov 17, 2011
• Last Update Posted: Feb 18, 2021
• Last Verified: Feb 1, 2021

• Additional relevant MeSH terms: Histiocytosis, Langerhans-Cell; Histiocytosis; Cladribine

Study Results

No Results Posted as of Feb 18, 2021