An Open Label Follow-Up Study to Evaluate the Long Term Safety and Efficacy of L-CSA in Patients with a Diagnosis of CLAD-BOS after they have completed the participation to BOSTON 1 and BOSTON 2 studies
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 81
- 试验地点
- 12
- 主要终点
- SAFETY: Adverse events (AEs)
研究概览
简要总结
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, who have completed either BOSTON-1 or BOSTON-2 study, and have taken the decision of continuing BOSTON- 3, based on their individual benefit risk considerations as discussed with their study doctor.
入排标准
- 年龄范围
- 18 years 至 65+ years（65+ Years, 18-64 Years）
- 接受健康志愿者
- 是
入选标准
- •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 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), or according to Investigator judgment.
- •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 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.
排除标准
- •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-
- •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.
- •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
- •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-
- •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.
结局指标
主要结局
SAFETY: Adverse events (AEs)
SAFETY: Adverse events (AEs)
SAFETY: Acute tolerability of IMP after initial dosing (only in patients randomized to SoC in BOSTON-1 or BOSTON-2 study)
SAFETY: Acute tolerability of IMP after initial dosing (only in patients randomized to SoC in BOSTON-1 or BOSTON-2 study)
SAFETY: Clinical laboratory parameters
SAFETY: Clinical laboratory parameters
SAFETY: Vital signs
SAFETY: Vital signs
SAFETY: Physical examinations
SAFETY: Physical examinations
SAFETY: Renal function
SAFETY: Renal function
EFFICACY: Mean change in forced expiratory volume in 1 second (FEV1) (mL) from baseline to approximately each year until End of Study
EFFICACY: Mean change in forced expiratory volume in 1 second (FEV1) (mL) from baseline to approximately each year until End of Study
EFFICACY: Mean change in FEV1/forced vital capacity (FVC) from baseline to approximately each year until End of Study
EFFICACY: Mean change in FEV1/forced vital capacity (FVC) from baseline to approximately each year until End of Study
EFFICACY: 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
EFFICACY: 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
EFFICACY: Rate of decline of FEV1 from baseline to last treatment day
EFFICACY: Rate of decline of FEV1 from baseline to last treatment day
EFFICACY: Use of additional immunosuppressive therapy
EFFICACY: Use of additional immunosuppressive therapy
EFFICACY: Proportion of patients experiencing re-transplantation due to BOS
EFFICACY: Proportion of patients experiencing re-transplantation due to BOS
EFFICACY: Proportion of patients with fatal outcome due to respiratory failure
EFFICACY: Proportion of patients with fatal outcome due to respiratory failure
EFFICACY: Change in BOS severity (according to criteria in Verleden 2019)
EFFICACY: Change in BOS severity (according to criteria in Verleden 2019)
EFFICACY: Mean relative intra-individual change of FEV1 between start of treatment (baseline) and end of study participation
EFFICACY: Mean relative intra-individual change of FEV1 between start of treatment (baseline) and end of study participation
EFFICACY: Euro Quality of Life Questionnaire (EQ-5D-5L)
EFFICACY: Euro Quality of Life Questionnaire (EQ-5D-5L)
EFFICACY: Hospitalization days
EFFICACY: Hospitalization days
EFFICACY: Absolute and relative change from baseline in: o Forced mid-expiratory flow (FEF25-75) o Forced Vital Capacity (FVC)
EFFICACY: Absolute and relative change from baseline in: o Forced mid-expiratory flow (FEF25-75) o Forced Vital Capacity (FVC)
次要结局
未报告次要终点
研究者
Ferdinando Ceravolo
Scientific
Zambon S.p.A.