Extracorporeal Photopheresis for Bronchiolitis Obliterans Syndrome

(ECP Trial)

Recruiting at 21 trial locations

Overseen By Mary Clare Derfler, RN, MSN

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Washington University School of Medicine

Must not be taking: T lymphocyte depleting

Disqualifiers: Pregnancy, Lupus, Porphyria, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 24

Duration: 6 months

280 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

The primary aims of this study is to determine the efficacy and tolerability of Extracorporeal Photopheresis (ECP) for the treatment of either Refractory Bronchiolitis Obliterans Syndrome (BOS) patients (258 at cessation of enrollment April 7, 2022) or Newly Diagnosed (22 as of enrollment Hold February 2022) Bronchiolitis Obliterans Syndrome patients after lung transplantation. In compliance with the Centers for Medicare and Medicaid Services' (CMS) Coverage with Evidence Development (CED) decision, the study will collect specified demographic, comorbidity, treatment, and outcome data exclusively for Medicare beneficiaries who are treated with ECP for either refractory or New BOS.

Will I have to stop taking my current medications?

The trial protocol does not specify whether you need to stop taking your current medications. However, if you are currently participating in another clinical trial or using certain investigational agents, you may be excluded from this study.

Is extracorporeal photopheresis (ECP) generally safe for humans?

Extracorporeal photopheresis (ECP) has been used for over 20 years to treat various immune-related conditions and is considered to have a consistent and predictable safety profile. In pediatric patients, it is regarded as relatively safe with few reported side effects, though careful monitoring is necessary. ^{1 2 3 4 5}

How is the treatment extracorporeal photopheresis (ECP) different from other treatments for bronchiolitis obliterans syndrome?

Extracorporeal photopheresis (ECP) is unique because it involves taking blood out of the body, treating it with a special light to modify immune cells, and then returning it to the body, which is different from typical drug treatments. This approach is particularly used for patients with bronchiolitis obliterans syndrome after lung transplantation, where standard treatments may not be effective. ^{1 2 6 7 8}

What data supports the effectiveness of the treatment Extracorporeal Photopheresis (ECP) for Bronchiolitis Obliterans Syndrome?

Several studies suggest that extracorporeal photopheresis (ECP) may help manage bronchiolitis obliterans syndrome (BOS) after lung transplantation by slowing down the decline in lung function, although the data is limited and more research is needed to confirm its effectiveness. ^{1 2 6 7 8}

Who Is on the Research Team?

George Despotis, MD

Principal Investigator

Washington University St. Louis School of Medicine

Are You a Good Fit for This Trial?

This trial is for Medicare recipients aged 18 or older who have had a lung transplant and are diagnosed with Bronchiolitis Obliterans Syndrome (BOS). They must have documented declines in lung function, frequent spirometry tests, and no history of certain cancers or conditions that would affect the study. Pregnant individuals, those allergic to ECP treatment components, or currently in another BOS clinical trial cannot participate.

Inclusion Criteria

I have been diagnosed with BOS, showing a significant decline in lung function.

Regular monitoring of lung function is needed before diagnosing a new lung condition called BOS.

I have received a lung transplant.

Exclusion Criteria

You have a condition that may make it hard for you to do breathing tests.

I have not had ECP therapy in the last 6 months.

My BOS developed either before 2016 or after 2022.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Extracorporeal Photopheresis (ECP) treatment for Bronchiolitis Obliterans Syndrome (BOS). Newly diagnosed BOS patients are randomized to either ECP or standard care.

6 months

24 treatments over 6 months

Follow-up

Participants are monitored for safety and effectiveness after treatment, with spirometry every 30 days for the first year and then annually.

Up to December 2028

Annual visits

Long-term Follow-up

Participants are followed for long-term outcomes, including mortality and quality of life assessments.

Annually up to 5 years

What Are the Treatments Tested in This Trial?

Interventions

• Extracorporeal Photopheresis (ECP)

Trial Overview The study is testing Extracorporeal Photopheresis (ECP) effectiveness on patients with either refractory BOS—those not responding to treatments—or newly diagnosed BOS after lung transplantation. It aims to collect data on how well these patients respond to ECP as per Medicare requirements.

How Is the Trial Designed?

Group I: Standard of Care Comparator Retrospective Chart review Experimental Treatment 1 Intervention

Data for the SOC comparator will be obtained via a retrospective data capture mechanism either medical chart review or electronic medical record query that meet all of the inclusion criteria and through an IRB approved waiver of consent. The SOC comparator data will be identical to the data collected for the Refractory BOS patients with the exception of inclusion of all spirometry values after transplant.

Group II: Refractory Bronchiolitis Obliterans Syndrome (REFRACTORY BOS) Experimental Treatment 1 Intervention

Participants with REFRACTORY BOS will be electronically assigned to either ECP treatment or Observation based on the participant's pre-enrollment Forced Expiratory Volume in 1 second (FEV1). Values from pulmonary function tests from the preceding 12 months will be entered into a web-based treatment allocation which will perform an automated calculation. Patients who have a statistically significant rate of decline within the preceding 6 months, and a derived protocol defined slope, will be assigned to the ECP Treatment arm. If a patient does not meet these criteria, the participant will be assigned to the Observation arm. Two hundred and fifty-eight (258) participants were enrolled with Refractory BOS nationwide.

Group III: Newly Diagnosed Bronchiolitis Obliterans (NEW BOS) Experimental Treatment 1 Intervention

Participants with NEW BOS will be randomized to Early Photopheresis Intervention (EPI) or Control (Standard of Care). EPI patients will receive 24 treatments in a 6-month period and may continue maintenance treatments. Twenty-two (22) NEW BOS participants were enrolled nationwide. The Control group will receive local Standard of Care for the management of BOS. Therapy will involve changes in immunosuppressive agents.

Who Is Running the Clinical Trial?

Washington University School of Medicine

Lead Sponsor

Trials

2,027

Recruited

2,353,000+

Therakos, Inc

Collaborator

Centers for Medicare and Medicaid Services/ Coverage and Analysis Group

Collaborator

Trials

2

Recruited

9,000+

The Foundation for Barnes-Jewish Hospital

Collaborator

Trials

43

Recruited

6,600+

Mallinckrodt

Industry Sponsor

Trials

202

Recruited

15,700+

Mario Saltarelli

Mallinckrodt

Chief Medical Officer

MD

Siggi Olafsson

Mallinckrodt

Chief Executive Officer

PhD in Pharmacy

Centers for Medicare and Medicaid Services

Collaborator

Trials

41

Recruited

31,230,000+

Citations

1. United States pubmed.ncbi.nlm.nih.gov

A prospective interventional study on the use of extracorporeal photopheresis in patients with bronchiolitis obliterans syndrome after lung transplantation. [2022]

2. United States pubmed.ncbi.nlm.nih.gov

Factors Associated With Mortality and Response to Extracorporeal Photopheresis in Lung Allograft Recipients With Bronchiolitis Obliterans Syndrome. [2019]

3. England pubmed.ncbi.nlm.nih.gov

Extracorporeal photopheresis to attenuate decline in lung function due to refractory obstructive allograft dysfunction. [2022]

4. France pubmed.ncbi.nlm.nih.gov

Initiation of extracorporeal photopheresis in lung transplant patients with mild to moderate refractory BOS: A single-center real-life experience. [2022]

5. United States pubmed.ncbi.nlm.nih.gov

The efficacy of photopheresis for bronchiolitis obliterans syndrome after lung transplantation. [2022]

6. United States pubmed.ncbi.nlm.nih.gov

Bacterial contamination rates in extracorporeal photopheresis. [2021]

7. England pubmed.ncbi.nlm.nih.gov

Factual reflections and recommendations on extracorporeal photopheresis in pediatrics. [2018]

8. England pubmed.ncbi.nlm.nih.gov

Extracorporeal photopheresis, a therapeutic option for cutaneous T-cell lymphoma and immunological diseases: state of the art. [2013]