Extension Trial on Efficacy / Safety of L-CsA + SoC in Treating BOS in Post Single or Double Lung Transplant (BOSTON-3)

NCT04039347 | Active Not Recruiting Phase 3 DMC FDA Drug

• 2 other identifiers: BT - L-CsA - 303 - FU2019-002987-29
• Study Type: interventional
• Target: 262
• Locations: 9 countries
• Sites: 37

Brief Summary

The objective of the trial is to assess the long-term safety and efficacy of L-CsA plus Standard of Care (SoC) in the treatment of BOS in single (SLT) and double lung transplant (DLT) recipients.

Conditions

Bronchiolitis Obliterans; Obliterative Bronchiolitis; Bronchiolitis Obliterans Syndrome

Keywords

single and double transplant

Outcome Measures

Primary Outcomes (1)

• Mean change in FEV1 from Baseline to Week 24

  FEV1 is the Forced Expiratory Volume in One Second

  Baseline to Week 24

Secondary Outcomes (5)

• Mean change in FEV1 from Baseline to Week 48

  Baseline to Week 48

• Mean change in FEV1 from Baseline to End of Study

  Baseline to end of study, approximately 2 years

• Mean change in FEV1/FVC from Baseline to Week 24

  Baseline to Week 24

• Mean change in FEV1/FVC from Baseline to Week 48

  Baseline to Week 48

• Time to Progression of BOS

  Baseline to End of Study, approximately 2 years

Other Outcomes (4)

• Adverse events

  Baseline through end of study, approximately 2 years

• Acute tolerability of L-CsA as measured by change in FEV1 at 1 hour and 4 hours after first inhalation of L-CsA

  First treatment with L-CsA

• Acute tolerability of L-CsA as measured by number of patients with treatment-related adverse events

  Baseline through end of treatment, approximately 2 years

Study Arms (2)

L-CsA 5 mg plus Standard of Care

EXPERIMENTAL

L-CsA 5 mg twice daily plus Standard of Care for up to 144 weeks for patients post Single Lung Transplant

Drug: Liposomal Cyclosporine A 5 mg

L-CsA 10 mg plus Standard of Care

EXPERIMENTAL

L-CsA 10 mg twice daily plus Standard of Care for up to 144 weeks for patients post Double Lung Transplant

Drug: Liposomal Cyclosporine A 10 mg

Interventions

Liposomal Cyclosporine A 5 mg DRUG

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

Also known as: L-CsA

L-CsA 5 mg plus Standard of Care

Liposomal Cyclosporine A 10 mg DRUG

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

Also known as: L-CsA

L-CsA 10 mg plus Standard of Care

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• Patients should be on a three-drug maintenance regimen of immunosuppressive agents including tacrolimus or another CNI, a second agent such as but not limited to MMF or azathioprine, and a systemic corticosteroid such as prednisone.
• 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.
• Women of childbearing potential must have a negative serum pregnancy test within 7 days prior to Visit 1 and must agree to use one of the methods of contraception listed in Appendix II through their End of Study Visit.

You may not qualify if:

• Known hypersensitivity to L-CsA or to cyclosporine A.
• Patients who experienced an AE related to study drug that led to permanent study drug discontinuation in BOSTON-1 or BOSTON-2.
• Patients with new onset of 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.
• Pregnant women or women who are unwilling to use appropriate birth control to avoid pregnancy through their End of Study Visit.
• Women who are currently breastfeeding.
• 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.
• Patients who are currently participating in an interventional clinical trial, other than BOSTON-1 or BOSTON-2.
• Psychiatric disorders or altered mental status precluding understanding of the informed consent process and/or completion of the necessary procedures.
• 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.

Sponsors & Collaborators

• Zambon SpA: lead

Study Sites (37)

Banner - University Medical Center

Phoenix, Arizona, 85006, United States

Location

David Geffen School of Medicine at UCLA

Los Angeles, California, 90095, United States

Location

UCSF

San Francisco, California, 94143, United States

Location

UCSF Center for Advanced Lung Disease

Stanford, California, 94305, United States

Location

University of Florida Dept of Pulmonary Medicine

Gainesville, Florida, 32610, United States

Location

Indiana University

Indianapolis, Indiana, 46202, United States

Location

UK Albert B. Chandler Hospital

Lexington, Kentucky, 40508, United States

Location

University of Maryland

Baltimore, Maryland, 21201, United States

Location

Johns Hopkins University Hospital

Baltimore, Maryland, 21287, United States

Location

Barnes-Jewish Hospital

St Louis, Missouri, 63110, United States

Location

Columbia University Medical Center

New York, New York, 10032, United States

Location

Duke University

Durham, North Carolina, 27710, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

OSU Wexner Medical Center

Columbus, Ohio, 43210, United States

Location

Temple University Hospital

Philadelphia, Pennsylvania, 19140, United States

Location

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15232, United States

Location

Baylor University Medical Center

Dallas, Texas, 75246, United States

Location

University of Texas Southwestern Medical Center

Dallas, Texas, 75390, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Houston Methodist Hospital

Houston, Texas, 77030, United States

Location

Waehringer Guertel

Vienna, Austria

Location

Hôpital Erasme

Brussels, 1070, Belgium

Location

Universitair Ziekenhuis Leuven

Leuven, 3000, Belgium

Location

Copenhagen University Hospital

Copenhagen, Denmark

Location

Hopital Marie Lannelongue

Le Plessis-Robinson, 92350, France

Location

CHU Hopital Nord

Marseille, France

Location

Hopitaux Universitaires de Strasbourg

Strasbourg, France

Location

Hannover Medical School

Hanover, Germany

Location

LMU Klinikum Groshadern

Munich, Germany

Location

Rabin Medical Center

Petah Tikva, Israel

Location

Complexo Hospitalario de A Coruna

A Coruña, Spain

Location

Hospital Universitari Vall d'Hebron

Barcelona, Spain

Location

Hospital Universitario Puerta de Hierro - Unidad de Trasplante Pulmonar

Madrid, 28222, Spain

Location

Hospital Marques de Valdecilla

Santander, Spain

Location

Unidad de Trasplante Pulmonar del Hospital La Fe

Valencia, 46026, Spain

Location

Royal Papworth Hospital NHS Foundation Trust

Cambridge, CB23 3RE, United Kingdom

Location

University of Manchester

Manchester, United Kingdom

Location

MeSH Terms

Conditions

Bronchiolitis Obliterans; Bronchiolitis Obliterans Syndrome

Condition Hierarchy (Ancestors)

Bronchiolitis; Bronchitis; Bronchial Diseases; Respiratory Tract Diseases; Lung Diseases, Obstructive; Lung Diseases; Organizing Pneumonia; Graft vs Host Disease; Immune System Diseases

Study Officials

• Paola R Castellani, MD

  Zambon SpA, Chief Medical Officer

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 19, 2019
• First Posted: July 31, 2019
• Study Start: March 12, 2020
• Primary Completion: December 1, 2025
• Study Completion: December 1, 2025
• Last Updated: January 27, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (20); DE (2); DK (1); ES (5); GB (2); FR (3); IL (1); AU (1); BE (2)