NCT04156893

Brief Summary

To determine the feasibility and efficacy of matching donor red cells by RH genotype for a cohort of chronically transfused patients with SCD.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
35

participants targeted

Target at P50-P75 for phase_1

Timeline
42mo left

Started Jan 2020

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Progress65%
Jan 2020Oct 2029

First Submitted

Initial submission to the registry

November 6, 2019

Completed
2 days until next milestone

First Posted

Study publicly available on registry

November 8, 2019

Completed
3 months until next milestone

Study Start

First participant enrolled

January 30, 2020

Completed
9.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2029

Expected
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2029

Last Updated

April 27, 2026

Status Verified

April 1, 2026

Enrollment Period

9.2 years

First QC Date

November 6, 2019

Last Update Submit

April 24, 2026

Conditions

Sickle Cells Disease

Keywords

Chronic Transfusion

Outcome Measures

Primary Outcomes (2)

  • Determine the treatment efficacy by monitoring the rate of Rh alloimmunization

    A primary objective is to determine whether providing RH genotype matched red cell units can reduce or prevent Rh alloimmunization.

    3.5 years

  • Determine the feasibility of identifying sufficient RH genotype matched units

    A primary objective is to determine the feasibility of identifying sufficient RH genotype matched red cells for chronically transfused patients with SCD with varied RH genotypes. Approximately 20 RHD (Rhesus D) and 20 RHCE (Rhesus CE) variants have been observed in patients with SCD and will determine whether sufficient RH genotyped units can be matched to the patient's own RH genotype.

    3.5 years

Secondary Outcomes (1)

  • Determine the rate of non-Rh alloimmunization

    3.5 years

Study Arms (1)

RH genotype matched red cell transfusions

EXPERIMENTAL

Subjects will receive RH genotyped matched red cell units for transfusion in addition to standard serologic C, E, and K antigen matching and being hemoglobin S negative, which is our institutional standard of care for patients with Sickle Cell Disease.

Biological: Red cell units that are genotype matched at the RHD and RHCE loci

Interventions

Red cell units that are genotype matched at the RHD and RHCE lociBIOLOGICAL

Patients will be provided with red cell units that are C, E, and K antigen matched (standard of care for patients with SCD) and genotype matched at the RHD and RHCE loci.

RH genotype matched red cell transfusions

Eligibility Criteria

Age6 Months+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Subjects age \>6 months
  • Diagnosis of SCD, all genotypes
  • Require a period of chronic red cell transfusion therapy
  • Subject/parental/guardian permission (informed consent) and if appropriate, child assent

You may not qualify if:

  • Rare RH genotype that would preclude identification of sufficient RBC units
  • Antigen negative requirements due to alloimmunization that would preclude identification of sufficient RBC units
  • Alloimmunized to D antigen
  • Rh alloimmunized patients for whom providing RH genotype matched blood would expose the patient to an antigen that would not be consistent with standard of care and blood bank protocols
  • Parents/guardians or subjects who, in the opinion of the Investigator, may be non-compliant with study schedules or procedures

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Related Publications (5)

  • Chou ST, Jackson T, Vege S, Smith-Whitley K, Friedman DF, Westhoff CM. High prevalence of red blood cell alloimmunization in sickle cell disease despite transfusion from Rh-matched minority donors. Blood. 2013 Aug 8;122(6):1062-71. doi: 10.1182/blood-2013-03-490623. Epub 2013 May 30.

    PMID: 23723452BACKGROUND

  • Vichinsky EP, Earles A, Johnson RA, Hoag MS, Williams A, Lubin B. Alloimmunization in sickle cell anemia and transfusion of racially unmatched blood. N Engl J Med. 1990 Jun 7;322(23):1617-21. doi: 10.1056/NEJM199006073222301.

    PMID: 2342522BACKGROUND

  • Yawn BP, Buchanan GR, Afenyi-Annan AN, Ballas SK, Hassell KL, James AH, Jordan L, Lanzkron SM, Lottenberg R, Savage WJ, Tanabe PJ, Ware RE, Murad MH, Goldsmith JC, Ortiz E, Fulwood R, Horton A, John-Sowah J. Management of sickle cell disease: summary of the 2014 evidence-based report by expert panel members. JAMA. 2014 Sep 10;312(10):1033-48. doi: 10.1001/jama.2014.10517. Erratum In: JAMA. 2014 Nov 12;312(18):1932. JAMA. 2015 Feb 17;313(7):729.

    PMID: 25203083BACKGROUND

  • Chou ST, Evans P, Vege S, Coleman SL, Friedman DF, Keller M, Westhoff CM. RH genotype matching for transfusion support in sickle cell disease. Blood. 2018 Sep 13;132(11):1198-1207. doi: 10.1182/blood-2018-05-851360. Epub 2018 Jul 19.

    PMID: 30026182BACKGROUND

  • Coleman S, Westhoff CM, Friedman DF, Chou ST. Alloimmunization in patients with sickle cell disease and underrecognition of accompanying delayed hemolytic transfusion reactions. Transfusion. 2019 Jul;59(7):2282-2291. doi: 10.1111/trf.15328. Epub 2019 Apr 25.

    PMID: 31021439BACKGROUND

Study Officials

  • Stella Chou, MD

    Children's Hospital of Philadelphia

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Stella Chou, MD

CONTACT

215-590-0947chous@chop.edu

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Model Details: Subjects will receive RH genotype matched red cell units for transfusion. For subjects with a history of stroke/recurrent transient ischemic attack or other indication who require tight control of Hb S, and RH genotyped blood is not available, standard of care serologic matched blood would be administered rather than delaying transfusion and risking higher Hb S level. For all subjects, standard of care serologic matched blood would be administered rather than delaying transfusion beyond 7 days
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 6, 2019

First Posted

November 8, 2019

Study Start

January 30, 2020

Primary Completion (Estimated)

April 1, 2029

Study Completion (Estimated)

October 1, 2029

Last Updated

April 27, 2026

Record last verified: 2026-04

Data Sharing

IPD Sharing
Will share

Protected Health Information (PHI) will be shared with the New York Blood Center (NYBC) to order blood for the subjects. This will be done using a secure online blood ordering system. The NYBC uses an FDA-approved HIPAA secure system called Blood Enterprise Computer System (BECS) to store patient data and results, including PHI. 3rd party computers at New York Blood Center will also store study data using study identification (ID) numbers. The purpose of having study data coded at NYBC is for specialized testing for red antigens or antibodies for which we will provide some clinical data to assist in the laboratory evaluation. To assure that the transmission of data and samples maintains confidentiality we will used study ID numbers for these samples. The master list will be maintained at the Children's Hospital of Philadelphia (CHOP). The study databases are password protected and on password protected computers at CHOP and at NYBC, that are backed up on the research servers.

Shared Documents
STUDY PROTOCOL
Time Frame
For the duration of the study
Access Criteria
Only study team members will have access to the data.

Locations

US(1)