A Open Label, Post Marketing Surveillance Study Following Transfusion of INTERCEPT Platelet Components

NCT02549222 | Completed

• 1 other identifier: CLI 00112
• Study Type: observational
• Target: 2,291
• Locations: 1 country
• Sites: 15

Brief Summary

This study is a prospective, non-randomized sequential cohort, open label, multi-center, non-inferiority, Phase IV surveillance study following transfusion of INTERCEPT PCs. The patient population will be hematology-oncology patients, including those undergoing hematopoietic stem cell transplant (HSCT), expected to require one or more PC transfusions. For each participating center, the study will start with a brief pilot run-in period with a group of at least 5 patients exposed only to conventional PCs. The purpose of this pilot run-in is to evaluate study logistics and data collection methods within each study center. Data from the pilot phase will be included in the data analysis for the treatment comparison. After the pilot run-in period, the study will be conducted in two sequential patient cohorts: 1) the Control cohort during which study patients will receive only conventional PCs, and 2) the INTERCEPT cohort during which patients will receive only INTERCEPT PCs. Patient enrollment at each Center will be monitored to target similar numbers of patients into the Control and Test Cohorts within each center. Centers may enroll Control and Test patients in ratios that vary from 2:1 to 1:2 due to institutional requirements to move rapidly to full INTERCEPT implementation, or due to availability issues with either Test or Control components. Within each Center, patient enrollment will be stratified in four categories: (1) chemotherapy only; and by use of conditioning regimens for hematopoietic stem cell transplantation (HSCT) in (2) myeloablative conditioning, (3) non-myeloablative conditioning, and (4) reduced intensity using the Center for International Blood and Marrow Transplant Research (CIBMTR) criteria. Note time from last chemotherapy treatment to first study transfusion should be no more than 30 days. To ensure both Test and Control cohorts have a similar allocation ratio (±10% per category) among the conditioning regimen strata, enrollment caps will be set for the Test cohorts, hence no Test patients will be enrolled to a stratum once the cap for the given stratum is met. Eligible patients will be enrolled in open Test strata sequentially as long as there is sufficient Test PC inventory available. Enrollment may be delayed for the Test cohort if sufficient inventory of Test PCs is not available.

Conditions

Transfusion Related Acute Lung Injury

Keywords

pathogen reduction; INTERCEPT; platelet; pulmonary; ARDS; hematology; oncology; assisted ventilation; transfusion of platelet components; hematology-oncology patients

Outcome Measures

Primary Outcomes (1)

• Proportion of patients requiring treatment-emergent assisted mechanical ventilation during the study observation period.

  For the purposes of this study, assisted mechanical ventilation defined as assisted ventilation started after the initiation of the first study PC and can be administered either by intubation or tight fitting mask with positive end-expiratory pressure (PEEP) or continuous positive airway pressure (CPAP or BiPAP) ≥ 5 cm H2O, if PEEP or CPAP information is available. Assisted mechanical ventilation is selected as the primary outcome as it provides an objective measure of severe, clinically significant pulmonary injury. Patients will be assessed for the initiation of assisted ventilation within 7 days after each study PC transfusion.

  Up to 28 days

Secondary Outcomes (7)

• Time from first study PC transfusion to onset of treatment-emergent assisted mechanical ventilation.

  Up to 28 days

• AEs occurring within 24 hours after the initiation of a study PC transfusion

  Up to 22 days

• TR occurring within 24 hours after the initiation of a study PC transfusion.

  Up to 22 days

• SAEs occurring within 7 days after each study PC transfusion

  Up to 28 days

• ARDS occurring within 7 days after each study PC transfusion

  Up to 28 days

Study Arms (2)

Control Cohort

During the Control cohort period patients will have study data collected following transfusion with only conventional PCs for up to 21 days of transfusion support, as clinically indicated in a manner that is consistent with the local standard of care.

Other: Standard of Care

INTERCEPT Cohort

During the INTERCEPT phase, patients will receive only INTERCEPT PCs and will have study data collected following transfusion for up to 21 days of transfusion support, as clinically indicated in a manner that is consistent with the local standard of care.

Other: Standard of Care

Interventions

Standard of Care OTHER

Control Cohort; INTERCEPT Cohort

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with hematology-oncology disorders, including those undergoing hematopoietic stem cell transplant (HSCT), expected to require one or more PC transfusions will be included in the study.

You may qualify if:

• Patients with a hematology-oncology disorder expected to require or requiring a transfusion of one or more PCs (time from last chemotherapy treatment to first study transfusion should be 30 days or less).
• Written signed informed consent (unless exemption of individual consent is granted by the center's IRB).

You may not qualify if:

• Documented allergy to psoralens

Sponsors & Collaborators

• Cerus Corporation: lead

Study Sites (15)

UCLA Medical Center

Los Angeles, California, 90095, United States

Location

Stanford University

Palo Alto, California, 94305, United States

Location

Yale-New Haven Hospital

New Haven, Connecticut, 06520, United States

Location

Emory University

Atlanta, Georgia, 30322, United States

Location

University of Maryland

Baltimore, Maryland, 21201, United States

Location

Beth Israel

Boston, Massachusetts, 02215, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

St. Louis Children's Hospital

St Louis, Missouri, 63110, United States

Location

Roswell Park Cancer Institute

Buffalo, New York, 14263, United States

Location

Memorial Sloan Kettering

New York, New York, 10065, United States

Location

Strong Memorial Hospital

Rochester, New York, 14642, United States

Location

Duke University Medical Center

Durham, North Carolina, 27705, United States

Location

East Carolina University Hospital

Greenville, North Carolina, 27834, United States

Location

TriHealth Cancer Institute

Cincinnati, Ohio, 45220, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37212, United States

Location

Related Publications (2)

• Wheeler AP, Snyder EL, Refaai M, Cohn CS, Poisson J, Fontaine M, Sehl M, Nooka AK, Uhl L, Spinella PC, Fenelus M, Liles D, Coyle T, Becker J, Jeng M, Gehrie EA, Spencer BR, Young P, Johnson A, O'Brien JJ, Schiller GJ, Roback JD, Malynn E, Jackups R, Avecilla ST, Liu K, Bentow S, Varrone J, Benjamin RJ, Corash LM. Acute pulmonary injury in hematology patients supported with pathogen-reduced and conventional platelet components. Blood Adv. 2024 May 14;8(9):2290-2299. doi: 10.1182/bloodadvances.2023012425.

  PMID: 38447116 DERIVED

• Delaney M, Karam O, Lieberman L, Steffen K, Muszynski JA, Goel R, Bateman ST, Parker RI, Nellis ME, Remy KE; Pediatric Critical Care Transfusion and Anemia EXpertise Initiative-Control/Avoidance of Bleeding (TAXI-CAB), in collaboration with the Pediatric Critical Care Blood Research Network (BloodNet), and the Pediatric Acute Lung Injury and Sepsis Investigators (PALISI) Network. What Laboratory Tests and Physiologic Triggers Should Guide the Decision to Administer a Platelet or Plasma Transfusion in Critically Ill Children and What Product Attributes Are Optimal to Guide Specific Product Selection? From the Transfusion and Anemia EXpertise Initiative-Control/Avoidance of Bleeding. Pediatr Crit Care Med. 2022 Jan 1;23(13 Suppl 1 1S):e1-e13. doi: 10.1097/PCC.0000000000002854.

  PMID: 34989701 DERIVED

MeSH Terms

Conditions

Transfusion-Related Acute Lung Injury; Neoplasms

Interventions

Standard of Care

Condition Hierarchy (Ancestors)

Acute Lung Injury; Lung Injury; Lung Diseases; Respiratory Tract Diseases; Transfusion Reaction; Hematologic Diseases; Hemic and Lymphatic Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Quality Indicators, Health Care; Quality of Health Care; Health Services Administration; Health Care Quality, Access, and Evaluation

Study Officials

• Edward L Snyder, MD

  Yale New Haven Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 11, 2015
• First Posted: September 15, 2015
• Study Start: December 1, 2015
• Primary Completion: September 1, 2020
• Study Completion: May 1, 2021
• Last Updated: February 18, 2022

Record last verified: 2022-02

Locations

US (15)