NCT00850018

Brief Summary

Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited blood disease that can cause intense pain episodes and may lead to organ failure. Preliminary studies have shown that adults with SCD may have brain abnormalities that contribute to problems with cognitive functioning, including attention and memory difficulties. This study will use brain magnetic resonance imaging (MRI) and neuropsychological testing to examine the differences in cognitive functioning in adults with SCD who are treated for anemia with monthly blood transfusions for 6 months versus adults with SCD who receive usual care for 6 months.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
36

participants targeted

Target at P50-P75 for phase_1

Timeline
Completed

Started Dec 2004

Longer than P75 for phase_1

Geographic Reach
1 country

14 active sites

Status
completed

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

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

December 1, 2004

Completed
4.2 years until next milestone

First Submitted

Initial submission to the registry

February 20, 2009

Completed
4 days until next milestone

First Posted

Study publicly available on registry

February 24, 2009

Completed
1.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2010

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2010

Completed
Last Updated

February 15, 2017

Status Verified

February 1, 2017

Enrollment Period

6 years

First QC Date

February 20, 2009

Last Update Submit

February 14, 2017

Conditions

Keywords

Sickle Cell DiseaseSickle Cell AnemiaHemoglobin SSHemoglobin SB0

Outcome Measures

Primary Outcomes (1)

  • Cognitive function

    Measured at Month 6

Study Arms (2)

1

EXPERIMENTAL

Participants will receive monthly blood transfusions.

Procedure: Monthly blood transfusion regimen

2

ACTIVE COMPARATOR

Participants will receive usual care.

Behavioral: Usual care

Interventions

Participants will receive blood transfusions at 3- to 4-week intervals for 6 months for the treatment of SCD-related anemia; the total number of transfusions that participants will receive will vary between six and eight.

Also known as: Chronic transfusion therapy
1
Usual careBEHAVIORAL

Participants will receive usual care for the treatment of SCD.

Also known as: Standard care alone, guided by disease symptoms.
2

Eligibility Criteria

Age21 Years - 55 Years
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64)

You may qualify if:

  • Completion of all components of the Phase 1 study (NCT00528801)
  • Wechsler Adult Intelligence Scale (WAIS) III-Performance IQ (PIQ) score less than or equal to 90
  • Hemoglobin less than or equal to 9.0 g/dL
  • People who did not complete Phase I of the study are eligible for enrollment in this study if they meet all of the following criteria:
  • Capable of giving informed consent for the study
  • Willing to undergo transfusion therapy for 6 months
  • African descent
  • Proficient/fluent in English
  • Hemoglobin electrophoresis confirming hemoglobin SS or SB0 (less than or equal to 15%)
  • WAIS III-PIQ score less than or equal to 90
  • Hemoglobin less than or equal to 9.0 g/dL
  • Mini-Mental Status Examination (MMSE) score of greater than or equal to 20
  • Profile of Mood States (POMS) score on the Depression-Dejection Subscale less than or equal to 40

You may not qualify if:

  • People who meet any of the following criteria are disqualified from enrollment in this study:
  • History of life threatening or serious transfusion complications
  • Lack of venous access
  • Current enrollment in the Arginine study (NCT00513617)
  • Pregnant
  • Refusal of transfusion
  • History of unexplained severe hemolytic transfusion reaction
  • History of serious allergic, pulmonary transfusion reaction requiring hospitalization
  • Positive auto-immune hemolytic anemia (direct Coombs test with IgG and complement)
  • Multiple (three or more) clinically significant allo-antibodies, due to common antigens (e.g., EC, Kel)
  • Uncommon, clinically significant antibody that results in difficulty in finding matched units (e.g., anti-JKB)
  • Currently taking Hydroxyurea and not on a stable dose in the 6 months before study entry
  • Creatinine level greater than 1.7 mg/dL
  • Major infarct identified on Phase I MRI
  • Currently on Procrit or related drug that stimulates red blood cell production
  • +26 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (14)

Children's Hospital & Research Center at Oakland

Oakland, California, 94609, United States

Location

Howard University

Washington D.C., District of Columbia, 20001, United States

Location

Memorial Cancer Institute

Hollywood, Florida, 33021, United States

Location

University of Miami Miller School of Medicine

Miami, Florida, 33136, United States

Location

Medical College of Georgia

Augusta, Georgia, 30912, United States

Location

Johns Hopkins

Baltimore, Maryland, 94117, United States

Location

Boston Medical Center

Boston, Massachusetts, 02118, United States

Location

Karmanos Cancer Institute at Wayne State University

Detroit, Michigan, 48201, United States

Location

Washington University

St Louis, Missouri, 63110, United States

Location

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27514, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Cincinnati Children's Hospital

Cincinnati, Ohio, 45229-3039, United States

Location

University of Cincinnati Medical Center

Cincinnati, Ohio, 45267, United States

Location

University of Texas Medical Branch

Galveston, Texas, 77555, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cell

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Elliott Vichinsky, MD

    UCSF Benioff Children's Hospital Oakland

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER

Study Record Dates

First Submitted

February 20, 2009

First Posted

February 24, 2009

Study Start

December 1, 2004

Primary Completion

December 1, 2010

Study Completion

December 1, 2010

Last Updated

February 15, 2017

Record last verified: 2017-02

Locations