In Vivo Study to Assess the Recovery and Survival of Radiolabeled Autologous INTERCEPT Apheresis Platelet Components Suspended in 100% Plasma Stored for up to 7 Days

NCT04022889 | Completed Phase 2 Results FDA Device

• 1 other identifier: CLI 00127
• Study Type: interventional
• Target: 37
• Locations: 1 country
• Sites: 2

Brief Summary

The principle objective of this study is to evaluate the hypothesis that INTERCEPT Platelets in 100% plasma stored for 5 or more days (up to 7 days) after apheresis collection retain sufficient viability for therapeutic transfusion efficacy. The post-infusion recovery and survival of autologous radiolabeled 7 day INTERCEPT platelets (Test) stored in 100% plasma will be measured in comparison to "fresh" autologous radiolabeled platelets (Control) according to FDA guidance for platelet testing (FDA 1999) in Stage 2 of this study protocol. A secondary objective is to compare the recovery and survival results for Test platelets prepared for radiolabeling using the procedures outlined by the Biomedical Excellence for Safer Transfusion Collaboration (BEST) or a variation of the BEST procedure (referred to as Variant 1) in Stage 1 of this study protocol. Cerus has demonstrated that the Variant 1 method, which does not incorporate an initial soft spin in the presence of ACD A, results in improved in vitro platelet recovery and quality during preparation for radiolabeling compared to the BEST procedure. This comparison will evaluate the hypothesis that preparation methods prior to radiolabeling may influence in vitro quality of the radiolabeled platelets and post-infusion viability outcomes.

Conditions

Healthy

Keywords

INTERCEPT; Recovery; Survival; 7 days; Radiolabeled; Autologous

Outcome Measures

Primary Outcomes (2)

• Post Infusion Recovery of Test Platelets at End of Storage (Day 7)

  Recovery of Test platelets stored for 7 Days as compared to fresh controls. In vivo recovery was expressed as proportion of infused in days and was estimated using a multiple-hit model. The FDA acceptance criteria for survival is \>66% of control with the lower bound of a two-sided 95% CI for the mean treatment difference (Test-0.66\*Control) in survival is greater than or equal to zero.

  11 days (+/- 1 day) post infusion of radiolabeled Test platelets stored for 7 days and fresh Control platelets

• Post Infusion Survival of Test Platelets at End of Storage

  Survival of Test platelets stored for 7 Days as compared to fresh controls. In vivo recovery was expressed as proportion of infused in days and was estimated using a multiple-hit model. The FDA acceptance criteria for survival is \>58% of control with the lower bound of a two-sided 95% CI for the mean treatment difference (Test - 0.58 \* Control) in survival is greater than or equal to zero.

  11 days (+/- 1 day) post infusion of radiolabeled Test platelets stored for 7 days and fresh Control platelets

Secondary Outcomes (3)

• Platelet Dose in Test Component

  At the end of INTERCEPT treatment on Day 1 or Day 2

• Platelet Yield Retention

  At the end of INTERCEPT treatment on Day 1 or Day 2

• pH 22°C

  At the end of storage on Day 7

Other Outcomes (34)

• Assessment of the Stored Test (INTERCEPT) Platelet Components: Component Volume

  At the end of storage on Day 7

• Assessment of the Stored Test (INTERCEPT) Platelet Components: Platelet Count

  At the end of storage on Day 7

• Assessment of the Stored Test (INTERCEPT) Platelet Components: Platelet Dose

  At the end of storage on Day 7

Study Arms (2)

Stage 1

EXPERIMENTAL

The study will be performed in two stages. Stage 1 is a randomized, 2-period crossover design. Test platelets stored for 7 days will be radiolabeled based on either the BEST or Variant 1 methods (depending on the period and randomization scheme for the Test platelets) for 12 healthy subjects. The recovery and survival for Test platelets prepared with the BEST and Variant 1 methods will be compared with each other and against the fresh platelet Control. With agreement from the FDA (BQ200481, July 8, 2020), completion of Stage 1 is not required.

Device: INTERCEPT Treated Platelets

Stage 2

EXPERIMENTAL

Stage 2 is a single arm design. Test platelets from 24 healthy subjects, stored for 7 days, will be prepared for radiolabeling following the Variant 1 methodology. The recovery and survival for Test platelets will be compared against the fresh platelet Control. Stage 1 subjects with evaluable Variant 1 method data will contribute to the requirement of the 24 subjects for Stage 2.

Device: INTERCEPT Treated Platelets

Interventions

INTERCEPT Treated Platelets DEVICE

Apheresis platelet components in 100% plasma collected using the Trima separator, prepared with the INTERCEPT Blood System for Platelets (Test Platelets) and stored for 7days at 20-24°C with continuous agitation. Samples from the Test component will be processed with either the BEST or the Variant 1 procedure prior to radiolabeling. The radiolabeled autologous Test and Control platelets (approximately 10-30 mL) will be simultaneously administered intravenously into the subject.

Stage 1; Stage 2

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age greater than or equal to 18 years, of either gender.
• Normal health status (as determined by the Investigator review of medical history and blood donor physical exam).
• Meet FDA, AABB, and site guidelines for blood donation and apheresis platelet donation. Travel, tattoos/piercings and/or male to male sexual contact deferrals do not apply.
• Complete blood count (CBC) and serum chemistry values within established reference ranges or within guidelines as above.
• Pre-donation platelet count of more than 150×10\^9 platelets/ L.
• Negative blood donor screening test panel for HIV, HBV, HCV, HTLV, syphilis, and WNV.
• Subjects of childbearing potential must agree to use a medically acceptable method of contraception throughout the study. A barrier method of contraception must be included, regardless of other methods.
• Signed and dated informed consent form.

You may not qualify if:

• For participation in Stage 2, received any previous infusion in this study.
• Clinically significant acute or chronic disease (as determined by the Investigator).
• Pregnant or nursing females.
• Subjects of childbearing potential not using effective contraception.
• Disease states or conditions that preclude apheresis platelet donation per AABB reference standards.
• Treatment with aspirin or aspirin-containing medications within 7 days of apheresis or treatment with non-steroidal anti-inflammatory drugs (NSAID), anti-platelet agents (or other drugs affecting platelet viability within 3 days of apheresis (e.g., ibuprofen or other NSAIDs).
• Subject received platelet inhibitors within 14 days of donation (e.g., clopidogrel, ticlopidine, amphetamines (e.g., Adderall, Dexedrine)).
• Subjects with positive cocaine and/or amphetamine results from urine drug screen.
• Splenectomized subjects.
• History of known hypersensitivity to indium or chromium.
• Has received an investigational drug within the past 28 days

Sponsors & Collaborators

• Cerus Corporation: lead

Study Sites (2)

Hoxworth Blood Center

Cincinnati, Ohio, 45221, United States

Location

Bloodworks Northwest Research Institute

Seattle, Washington, 98102, United States

Location

Results Point of Contact

• Title: Carol Moore
• Organization: Cerus Corporation

cmoore@cerus.com

9258766819

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: OTHER
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 9, 2019
• First Posted: July 17, 2019
• Study Start: November 5, 2019
• Primary Completion: April 16, 2021
• Study Completion: April 17, 2021
• Last Updated: October 27, 2022
• Results First Posted: October 27, 2022

Record last verified: 2022-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)