Rejuvesol® Washed RBC in Sickle Cell Patients Requiring Frequent Transfusions

NCT02731157 | Completed Phase 4 Results

• 1 other identifier: Pro00069955
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 2

Brief Summary

The objective of this proposal is to test the feasibility of red blood cell (RBC) rejuvenation to chronic transfusion in sickle cell disease (SCD) and the potential benefit of RBC rejuvenation in this population to determine if a larger clinical trial powered to definitively characterize the benefits of rejuvenation is warranted. This is a small pilot study is to see if restoring important energy molecules (ATP and 2,3,DPG) in stored red blood cells before they are transfused, with a rejuvenating solution (Rejuvesol), offers any advantages to individuals over standard blood transfusion. Subjects will receive either rejuvenated (R) or standard (S) RBCs with each transfusion for 6 transfusions (over approximately a 6-month period) in a pre-defined order to maximize detection of any signal.

Conditions

Sickle Cell Disease

Outcome Measures

Primary Outcomes (1)

• Average Percent Hemoglobin (HbA) Decrement Per Day

  The %HbA decrement is the current pre-treatment HbA - previous post treatment HbA in %. The average %HbA decrement per day was calculated using matched pairs.

  6 months

Secondary Outcomes (5)

• Actual HbA Decrement (g/dl) With Indexing to Calculated Circulating Blood Volume

  6 months

• Actual HbA Decrement (g/dl) Without Indexing to Calculated Circulating Blood Volume

  6 months

• Change in RBC Microparticles (MP) Counts

  6 months

• Change in RBC/RBC-MP-mediated Thrombin Generation

  6 months

• Change in p50 Pre- and Post-transfusion

  6 months

Study Arms (2)

Transfusion with rejuvenated red blood cells (RBCs)

EXPERIMENTAL

Subjects with sickle cell disease will receive RBCs treated with Rejuvesol®. Scheduled red cell exchanges performed with the last 4 units of the exchange having been incubated with Rejuvesol® solution. Only transfusions necessary for the treatment of the sickle cell disease will be given for the purpose of the study.

Drug: Rejuvesol; Procedure: Blood transfusion

Transfusion with standard red blood cells

ACTIVE COMPARATOR

Subjects with sickle cell disease will receive standard RBCs. Only transfusions necessary for the treatment of the sickle cell disease will be given for the purpose of the study.

Procedure: Blood transfusion

Interventions

Rejuvesol DRUG

Transfusion with rejuvenated red blood cells (RBCs)

Blood transfusion PROCEDURE

Only transfusions necessary for the treatment of the sickle cell disease will be given for the purpose of the study.

Transfusion with rejuvenated red blood cells (RBCs); Transfusion with standard red blood cells

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Stable, compliant, chronically transfused sickle cell disease (SCD) patients
• Currently maintained crisis-free with repeated RBC therapy for at least 3 consecutive sessions
• ≥18 years old
• Have Hb SS disease
• Have the capacity to give informed consent

You may not qualify if:

• Baseline need for washed RBCs
• Pre-treatment SaO2 \< 92%.

Sponsors & Collaborators

• Duke University: lead

Study Sites (2)

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States

Location

Duke Univeristy Medical Center

Durham, North Carolina, 27710, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cell

Interventions

Blood Transfusion

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Biological Therapy; Therapeutics

Results Point of Contact

• Title: Ian Welsby, M.D.
• Organization: Duke University

ian.welsby@duke.edu

919-681-6752

Study Officials

• Ian J Welsby, BSc MBBS

  Duke University

  PRINCIPAL INVESTIGATOR

• Jay Raval, MD

  University of North Carolina

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NON RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: OTHER
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 2, 2016
• First Posted: April 7, 2016
• Study Start: December 1, 2016
• Primary Completion: December 31, 2017
• Study Completion: December 31, 2017
• Last Updated: January 25, 2019
• Results First Posted: January 25, 2019

Record last verified: 2018-12

Locations

US (2)