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

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: Evaluation of Efficacy and Tolerance of Cladribine in Symptomatic Patients With Pulmonary Langerhans Cell Histiocytosis and Impairment of Lung Function

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 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.
Sponsor: Assistance Publique - Hôpitaux de Paris

Current Primary Outcome: Cumulated incidence of response to treatment [ Time Frame: 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


Original Primary Outcome: Cumulated incidence of response to treatment [ Time Frame: 6 months ]

response to treatment after 6 months is defined as

  • ≥10% improvement of FVC
  • and/or ≥10% improvement of postbronchodilator FEV1 and ≥200ml


Current Secondary Outcome:

  • Responses to treatment [ Time Frame: 3 months ]
  • 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 ]
  • Grade 3 or 4 neutropenia or thrombopenia [ Time Frame: 6 months ]
  • Incidence of infection [ Time Frame: 6 months ]
  • Incidence of grade 3 or 4 side effects [ Time Frame: 6 months ]
  • Response to treatment of extra pulmonary localizations of the Langerhans disease [ Time Frame: 6, 9, and 12 months ]
  • Incidence of pneumothorax [ Time Frame: 12 months ]
  • Mortality [ Time Frame: 12 months, 4 years ]
  • Incidence of secondary malignant disease [ Time Frame: 4 years ]
  • Treatment response [ Time Frame: at 6 months ]
  • Treatment response [ Time Frame: 9 months ]
  • Treatment response [ Time Frame: 12 months ]
  • Variation of nodular and cystic semiquantitative scores in High Resolution Computed Tomography (HRCT) [ Time Frame: 6 months ]


Original Secondary Outcome:

  • Responses to treatment [ Time Frame: 3 months ]
  • Absolute variations of FEV1, FVC, RV, and DLCO(mL) [ Time Frame: 6 months ]
  • Grade 3 or 4 neutropenia or thrombopenia [ Time Frame: 6 months ]
  • Incidence of infection [ Time Frame: 6 months ]
  • Incidence of grade 3 or 4 side effects [ Time Frame: 6 months ]
  • Response to treatment of extra pulmonary LCH localizations [ Time Frame: 6, 9, and 12 months ]
  • Incidence of pneumothorax [ Time Frame: 12 months ]
  • Mortality [ Time Frame: 12 months, 4 years ]
  • Incidence of secondary malignant disease [ Time Frame: 4 years ]
  • Treatment response [ Time Frame: at 6 months ]
  • Treatment response [ Time Frame: 9 months ]
  • Treatment response [ Time Frame: 12 months ]
  • Variation of nodular and cystic semiquantitative scores in HRCT [ Time Frame: 6 months ]


Information By: Assistance Publique - Hôpitaux de Paris

Dates:
Date Received: October 14, 2011
Date Started: November 2011
Date Completion: April 2018
Last Updated: September 28, 2016
Last Verified: September 2016