Bronchiolitis Obliterans Syndrome in Patients post Single or post Double Lung Transplantation MedDRA version: 20.0 Level: LLT Classification code 10049202 Term: Bronchiolitis obliterans System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders
Conditions
Sponsors
Zambon SpA
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Patients who have completed all visits through the End of Treatment Visit in either BOSTON-1 or BOSTON-2, did not withdraw informed consent, and did not prematurely terminate study drug administration. 2. Patients should be on a maintenance regimen of immunosuppressive agents as defined in the Boston 1 and Boston 2 studies, (i.e. tacrolimus, a second agent such as but not limited to mycophenolate mofetil (MMF) or azathioprine, and a systemic corticosteroid such as prednisone as third agent. Concomitant azithromycin for prophylaxis or treatment of BOS is also allowed), according to Investigator judgment. 3. Patients capable of understanding the purposes and risks of the clinical trial, who have given written informed consent and agree to comply with the clinical trial requirements/visit schedules, and who are capable of aerosol inhalation. 4. Women of childbearing potential must have a negative serum or urine pregnancy test within 7 days prior to Visit 1 and must agree to use one of the methods of contraception listed in Appendix II of the protocol through their End of Study Visit. 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 180 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 40
Exclusion criteria
Exclusion criteria: 1. Known hypersensitivity to L-CsA or to cyclosporine A. 2. Patients who experienced an AE related to study drug that led to permanent study drug discontinuation in BOSTON-1 or BOSTON-2. 3. Patients who developed a new malignancy while participating in BOSTON-1 or BOSTON-2, including post-transplant lymphoproliferative disorder, with the exception of treated, localized basal and squamous cell carcinomas. 4. Pregnant women or women who are unwilling to use appropriate birth control to avoid pregnancy through their End of Study Visit. 5. Women who are currently breastfeeding. 6. Receipt of an investigational drug, other than L-CsA, as part of a clinical trial within 4 weeks prior to Visit 1. This is defined as any treatment that is implemented under an Investigational New Drug (IND) or compassionate use. 7. Patients who are currently participating in an interventional clinical trial, other than BOSTON-1 or BOSTON-2. 8. Psychiatric disorders or altered mental status precluding understanding of the informed consent process and/or completion of the necessary procedures. 9. Any co-existing medical condition that in the Investigator’s judgment will substantially increase the risk associated with the patient’s participation in the clinical trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the long-term safety and efficacy of L-CsA plus SoC in the treatment of BOS in single and double lung transplant recipients;Secondary Objective: Not applicable;Primary end point(s): SAFETY 1 Adverse events (AEs) 2 Acute tolerability of IMP after initial dosing (only in patients randomized to SoC in BOSTON-1 or BOSTON-2 study) 3 Clinical laboratory parameters 4 Vital signs 5 Physical examinations 6 Renal function EFFICACY 1 Mean change in forced expiratory volume in 1 second (FEV1) (mL) from baseline to approximately each year until End of Study 2 Mean change in FEV1/forced vital capacity (FVC) from baseline to approximately each year until End of Study 3 Time to progression of BOS, defined as the earliest of the following: o Absolute decrease from baseline in FEV1 = 10% or = 200 mL and absolute decrease in FEV1/FVC of > 5%, OR o Worsening of BOS grade (according to criteria in Verleden 2019), OR o Re-transplantation, OR o Death from respiratory failure 4 Rate of decline of FEV1 from baseline to last treatment day 5 Use of additional immunosuppressive therapy 6 Proportion of patients experiencing re-transplantation due to BOS 7 Proportion of patients with fatal outcome due to respiratory failure 8 Change in BOS severity (according to criteria in Verleden 2019) 9 Mean relative intra-individual change of FEV1 between start of treatment (baseline) and end of study participation 10 Absolute and relative change from baseline in: o Forced mid-expiratory flow (FEF25-75) o Forced Vital Capacity (FVC) 11 Euro Quality of Life Questionnaire (EQ-5D-5L) 12 Hospitalization days;Timepoint(s) of evaluation of this end point: SAFETY 1 Permanent assessment throughout the complete study period 2 Day 1 3, 4, 5 and 6: W0 - EoS EFFICACY From baseline to EoS | — |
Countries
Austria, Belgium, Denmark, France, Germany, Israel, Spain, United Kingdom, United States
Contacts
Public ContactPaola Castellani
Zambon SpA
Outcome results
None listed