Female subjects affected by Llymphangioleiomyomatosis (LAM) MedDRA version: 19.0 Level: PT Classification code 10049459 Term: Lymphangioleiomyomatosis System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders
Conditions
Interventions
Trade Name: Ofev
Pharmaceutical Form: Capsule, soft
Sponsors
MULTIMEDICA S.P.A.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Written Informed Consent for participating to trial e to genetic tests, 2. Patient aged = 18 years at visit 1, 3. sporadic or TSC associated LAM, classified as ‘‘definite’’ by the European Respiratory Society criteria and /or serum VEGFD level >/= 800 mg/ml, and evidence of a 10% deterioration in FEV1 and /or loss of 80 ml of FEV1 or more in the last year (post bronchodilator). Also LAM patients with proven side effects and/or toxicities/ contraindications to sirolimus therapy will be eligible for this study. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 28 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 2
Exclusion criteria
Exclusion criteria: Previous treatment with nintedanib Other investigational therapy (participation in research trial) received within 8 weeks of visit Thoracic, abdominal, gynecological, neurologic surgical procedures planned to occur during trial period. Pregnant women or women who are breast feeding or of child bearing potential not using two effective methods of birth control (one barrier and one highly effective non-barrier) for at least 1 month prior to enrolment (and until 3 months after treatment end). Female patients will be considered to be of childbearing potential unless surgically sterilised by hysterectomy or bilateral tubal ligation, or postmenopausal for at least two years or if women use a barrier method of contraception includes condom or occlusive cap with spermicidal (foam, gel, film, cream, suppository) or male sterilization (with appropriate post-vasectomy documentation of the absence of sperm in the ejaculate), or if they use an intrauterine device · International normalized ratio (INR) > 2 at visit 1
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective is to demonstrate a reduction of lung function decline, as measured by a change of the yearly rate of decline of FEV1;Secondary Objective: Functional respiratory evaluation after treatment compared to basal -Safety and tolerability -FVC variation compared to basal -DLCO variation compared to basal -Serum VEGF-D variation compared to basal -Efficacy of Nintedanib in angiomyolipoma size reduction (in patients with angiomyolipomas not greater than 5 cm of diameter). -Efficacy of Nintedan;Primary end point(s): The primary outcome measure will be the FEV1 response, which will be assessed as the change in FEV1 (FEV1 slope) in milliliters per month ( post bronchodilator).;Timepoint(s) of evaluation of this end point: 12 and 18 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The proportion of patients achieving a stabilization of the lung function during treatment. A patient will be considered stabilized if her value of the FEV1 measured at 12-month visit will be equal or above the baseline value. - Safety and Tolerability - Rate of decline of FVC over the course of the study - Rate of decline Dlco over the course of the study over the course of the study -VEGF-D levels: change from baseline at the end of treatment period - To assess if nintedanib can reduce renal angiomyolipomas (presence determined from MRI and defined as lesions with a maximum diameter of a least 1 cm). Reduction is defined as a decrease of total volume of target angiomyolipomas (sum of volume of all target lesions indentified at baseline) of at least 30% relative to baseline. New angiomyolipoma = 1 cm in diameter, kidney increases in volume > 20%, and angiomyolipoma related bleeding must be excluded. - To test if nintedanib reduces the number of circulating LAM cells in treated pts and if the loss of circulating LAM cells persists after treatment discontinuation. - Quality of ;Timepoint(s) of evaluation of this end point: 12 and 18 months | — |
Countries
Italy
Contacts
Public ContactServizio di Data Management
MultiMedica SpA
Outcome results
None listed