The Impact of Oxidative Stress on Erythrocyte Biology

NCT04028700 | Terminated Phase 2 Results DMC FDA Drug

• 2 other identifiers: 21-0587R01HL148151-01
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 1

Brief Summary

This study will address if red blood cells transfused to a sickle cell patient from a donor with a glucose-6-phosphate-dehydrogenase (G6PD) enzyme deficiency have a different lifespan as measured by the percentage of red blood cells that survive post-transfusion compared to red blood cells transfused to a sickle cell patient from a donor without a G6PD enzyme deficiency.

Conditions

Sickle Cell Disease Without Crisis

Outcome Measures

Primary Outcomes (1)

• Percentage of Red Blood Cells Surviving

  Post-Transfusion Recovery

  24 hours post-transfusion

Secondary Outcomes (1)

• Mean Percent Change in Hemoglobin A

  1 hour post-transfusion, 4 weeks post-transfusion

Study Arms (2)

G6PD Deficient Red Blood Cell Transfusion, then Non-G6PD deficient Red Blood Cell Transfusion

EXPERIMENTAL

Transfusion of a red blood cell unit that has been identified by local laboratory procedures to be deficient in G6PD enzyme activity followed after 4 months by transfusion of a red blood cell unit that has been identified by local laboratory procedures to not be deficient in G6PD enzyme activity.

Biological: G6PD Deficient Red Blood Cell Transfusion; Biological: Non-G6PD deficient Red Blood Cell Transfusion

Non-G6PD deficient Red Blood Cell Transfusion, then G6PD Deficient Red Blood Cell Transfusion,

ACTIVE COMPARATOR

Transfusion of a red blood cell unit that has been identified by local laboratory procedures to not be deficient in G6PD enzyme activity followed after 4 months by transfusion of a red blood cell unit that has been identified by local laboratory procedures to be deficient in G6PD enzyme activity

Biological: G6PD Deficient Red Blood Cell Transfusion; Biological: Non-G6PD deficient Red Blood Cell Transfusion

Interventions

G6PD Deficient Red Blood Cell Transfusion BIOLOGICAL

Patients will receive a red blood cell transfusion. The last 50 mL of the transfusion will be labeled with chromium to allow investigators to study the lifespan of the red blood cells transfused into each patient.

G6PD Deficient Red Blood Cell Transfusion, then Non-G6PD deficient Red Blood Cell Transfusion; Non-G6PD deficient Red Blood Cell Transfusion, then G6PD Deficient Red Blood Cell Transfusion,

Non-G6PD deficient Red Blood Cell Transfusion BIOLOGICAL

Patients will receive a red blood cell transfusion. The last 50 mL of the transfusion will be labeled with chromium to allow investigators to study the lifespan of the red blood cells transfused into each patient.

G6PD Deficient Red Blood Cell Transfusion, then Non-G6PD deficient Red Blood Cell Transfusion; Non-G6PD deficient Red Blood Cell Transfusion, then G6PD Deficient Red Blood Cell Transfusion,

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Age 18-60 years
• Has diagnosis of sickle cell disease
• Steady state (no pain or baseline pain and ≥1 month from any hospital admission)
• Receiving chronic transfusions (i.e., regular transfusion every 4-8 weeks).

You may not qualify if:

• History of transfusion reactions not adequately managed by antihistamines
• Does not have crossmatch compatible red cells
• Known G6PD deficiency
• Hepato- or splenomegaly
• Participation in another therapeutic trial
• Pregnant or nursing
• HIV positive
• At investigator discretion for uncontrolled inter-current illness or social situation limiting compliance with study requirements.
• Inability to speak and/or read English

Sponsors & Collaborators

• University of North Carolina, Chapel Hill: lead
• Columbia University: collaborator
• National Heart, Lung, and Blood Institute (NHLBI): collaborator

Study Sites (1)

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27599, United States

Location

Results Point of Contact

• Title: Matthew S. Karafin MD MS
• Organization: University of North Carollina at Chapel Hill

matthew.karafin@unchealth.unc.edu

984-974-1583

Study Officials

• Matthew Karafin, MD, MS

  University of North Carolina, Chapel Hill

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: PARTICIPANT
• Masking Details: Patients will not be told which type of blood they are receiving first. There is no way to tell if blood has enzyme deficiencies by looking at it.
• Purpose: BASIC SCIENCE
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 19, 2019
• First Posted: July 23, 2019
• Study Start: January 2, 2022
• Primary Completion: July 18, 2024
• Study Completion: August 16, 2024
• Last Updated: June 3, 2025
• Results First Posted: June 3, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: 9 to 36 months following publication
• Access Criteria: Investigator proposing to use the data has approval from an IRB, IEC, or REB and an executed data use/sharing agreement with UNC.

Locations

US (1)