NCT03657342

Efficacy + Safety of Liposome Cyclosporine A to Treat Bronchiolitis Obliterans Post Single Lung Transplant (BOSTON-1)

A Phase III Clinical Trial to Demonstrate Efficacy / Safety of Liposomal Cyclosporine A + Standard of Care (SoC) vs SoC Alone in Treating Chronic Lung Allograft Dysfunction / Bronchiolitis Obliterans in Patients Post Single Lung Transplant

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 62 (actual)
• Sponsor: Zambon SpA · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The objective of this trial is to assess the efficacy and safety of aerosolized liposomal cyclosporine A (L-CsA) as add-on therapy to standard of care (SoC) as compared to SoC alone in single lung transplant recipients with chronic lung allograft dysfunction (CLAD)-bronchiolitis obliterans syndrome (BOS).

Detailed description

BOSTON-1 was a Phase III, prospective, multicenter, randomized, open-label, controlled clinical trial of L-CsA for the treatment of BOS in adults diagnosed with CLAD-BOS following single lung transplant. Eligible patients were randomized to receive either L-CsA (5 mg) via the PARI eFlow® Device (L-CsA eFlow) twice daily plus SoC treatment or SoC alone for a period of 48 weeks. Regardless of treatment allocation, all patients continued to receive their SoC regimen for maintenance of the lung allograft. Maintenance immunosuppressive therapy including tacrolimus, a second agent such as, but not limited to, MMF or azathioprine, and a systemic corticosteroid such as prednisone as third agent was administered according to institutional standards. Up to 11 study visits (screening, V1 through V10) were planned. Spirometry was measured at all visits according to American Thoracic Society/European Respiratory Society 2005 guidelines. Spirometry measurements on-site were performed by pulmonary function technicians, respiratory therapists, or physiotherapists who were blinded to each patient's study treatment assignment. Safety assessments at every study visit included physical examination, vital signs, adverse event (AE) reporting, and clinical laboratory tests. Acute tolerability of L-CsA was assessed by spirometry before and 1 hour and 4 hours after inhalation of L-CsA at initial dosing. All patients who completed the study were eligible to continue in an open-label extension trial of L-CsA (BOSTON-3 \[Study BT-L-CsA-303-FU\]).

Conditions

• Bronchiolitis Obliterans
• Chronic Rejection of Lung Transplant
• Lung Transplant Rejection
• Lung Transplant; Complications
• Lung Transplant Failure and Rejection
• Chronic Lung Allograft Dysfunction

Interventions

Type	Name	Description
DRUG	Liposomal Cyclosporine A	This formulation is developed for inhalation use and delivered via the PARI eFlow® Device, which is a new technology of nebulizing liquid drugs with a perforated vibrating membrane resulting in an aerosol with a low ballistic momentum and a high percentage of droplets in a respirable size range of 3-5 μm
DRUG	standard of care	Standard of Care Therapy. Eligible patients should be on a maintenance regimen of immunosuppressive agents including tacrolimus, a second agent such as but not limited to MMF or azathioprine, and a systemic corticosteroid such as prednisone as third agent. The regimen must be stable within 4 weeks prior to randomization with respect to the therapeutic agents. Patients receiving azithromycin for prophylaxis or treatment of BOS, must be on a stable regimen for a least 4-weeks prior to randomization and will continue to receive azithromycin during the trial as deemed appropriate by the investigator.

Timeline

• Start date: 2019-03-26
• Primary completion: 2024-04-16
• Completion: 2024-04-16
• First posted: 2018-09-05
• Last updated: 2025-10-27
• Results posted: 2025-10-27

Locations

39 sites across 7 countries: United States, Belgium, France, Germany, Israel, Spain, United Kingdom

Regulatory

• FDA-regulated drug study