Condition category
Haematological Disorders
Date applied
24/08/2004
Date assigned
11/10/2004
Last edited
21/08/2014
Prospective/Retrospective
Prospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

Plain English Summary

Not provided at time of registration

Trial website

http://sitstudy.wustl.edu

Contact information

Type

Scientific

Primary contact

Dr Michael R. DeBaun

ORCID ID

Contact details

WUSM CB 8519
4444 Forest Park Blvd.
Saint Louis
MO
63108
United States of America
+1 314 286 1186
DeBaun_M@kids.wustl.edu

Additional identifiers

EudraCT number

ClinicalTrials.gov number

Protocol/serial number

U01NS42804

Study information

Scientific title

Acronym

SIT Trial

Study hypothesis

On 12/03/2008 this trial record was updated. The start date and anticipated end date of this trial were also updated from 01/04/2000 and 30/04/2005 to 01/12/2005 and 30/11/2012, respectively.

Added as of 12/03/2008:
Primary hypothesis:
1. Prophylactic blood transfusion therapy in children with silent cerebral infarcts will result in at least 86% reduction in the proportion of patients with clinically evident strokes or new or progressive silent cerebral infarcts.

Secondary hypotheses:
1. Prophylactic blood transfusion therapy will limit the further decline in general intellectual abilities when compared to the observation arm.
2. The overall benefit of blood transfusion therapy for silent cerebral infarctions outweighs the risks associated with this therapy.

Ethics approval

Not provided at time of registration

Study design

Randomised controlled trial

Primary study design

Interventional

Secondary study design

Randomised controlled trial

Trial setting

Hospitals

Trial type

Treatment

Patient information sheet

Condition

Sickle Cell Anemia and Stroke

Intervention

Participants will be randomly assigned to one of two groups - the blood transfusion group or the observation group. The blood transfusion group will receive regular blood transfusion therapy. All participants will receive history and physical exams every three months, cognitive testing and neurological exam annually, a TCD 12-18 months following randomization, and a final MRI of the brain at study exit. Active participation on the study is 3 years.

Intervention type

Other

Phase

Not Specified

Drug names

Primary outcome measures

Not provided at time of registration

Secondary outcome measures

Not provided at time of registration

Overall trial start date

01/12/2005

Overall trial end date

30/11/2012

Reason abandoned

Eligibility

Participant inclusion criteria

Inclusion criteria amended as of 12/03/2008:
Inclusion Criteria for Screening:
1. Patient must have sickle cell anemia (hemoglobin SS or sickle B) as confirmed by the local institution by hemoglobin analysis after six months of age
2. Patient must be 5 through 14 years of age (i.e., must have attained their 5th, but not their 15th birthday when the screening consent is signed)
3. Informed consent with assent in accordance with the institutional policies

Inclusion criteria provided at time of registration:
1. Patients ages 6-12 with sickle cell anemia (Hemoglobin SS or SB)
2. Patient must have a silent cerebral infarct documented by magnetic resonance imaging (MRI) of the brain as read by the neuroradiology panel

Participant type

Patient

Age group

Adult

Gender

Both

Target number of participants

Added as of 12/03/2008: Screen 1880; Randomize at least 204 and up to 250

Participant exclusion criteria

Exclusion criteria amended as of 12/03/2008:
Exclusion Criteria for Screening at initial screening evaluation and after informed consent has been signed as well as during the interval up to randomization:
1. Patient with a history of a focal neurologic event lasting more than 24 hours with medical documentation or a history of prior overt stroke
2. Patient with other neurological problems, such as neurofibromatosis, lead poisoning, non-febrile seizure disorder, or tuberous sclerosis
3. Patient with HIV infection
4. Pregnancy or lactating females
5. Patient who received treatment with anti-sickling drugs or hydroxyurea within 3 months or anticipate receiving anti-sickling drugs or hydroxyurea during the course of the study
6. Patients on chronic blood transfusion therapy for other reasons
7. Patient judged not likely to be compliant by his/her hematologist and study coordinator based on previous compliance in clinic appointments and following advice. Specifically, families that have missed at least two appointments without notification within 12 months prior to the trial or parents/guardians of potential patients that have been reported for medical or education neglect are not eligible for this trial
8. Patient unable to receive blood transfusion because of alloimmunization
9. Patients who have or anticipate receiving permanent (or semi-permanent) metallic structures attached to their body. (e.g., braces on teeth, body piercings), which their physicians believe will interfere with the MRI of the head to assess the presence of silent cerebral infarct
10. Patients with any person living in the same household who have been randomly assigned in the SIT Trial

Exclusion criteria provided at time of registration:
1. Patients with a history of overt stroke or focal neurological event lasting more than 24 hours
2. Patients with other neurological problems, such as neurofibromatosis, lead poisoning, or tuberous sclerosis
3. Patients with a transcranial doppler (TCD) study with a time-averaged mean velocity greater than 200 cm/sec verified by the study radiologist

Recruitment start date

01/12/2005

Recruitment end date

30/11/2012

Locations

Countries of recruitment

United States of America

Trial participating centre

WUSM CB 8519
Saint Louis, MO
63108
United States of America

Sponsor information

Organisation

National Institute of Neurological Disorders and Stroke (NINDS) (USA)

Sponsor details

Neuroscience Center
Room 2212
6001 Executive Blvd
Rockville
MD
20892
United States of America

Sponsor type

Government

Website

http://www.ninds.nih.gov/

Funders

Funder type

Government

Funder name

National Institute of Neurological Disorders and Stroke (NINDS) (USA) (ref: U01NS42804)

Alternative name(s)

NINDS

Funding Body Type

government organisation

Funding Body Subtype

federal/national government

Location

United States of America

Results and Publications

Publication and dissemination plan

Not provided at time of registration

Intention to publish date

Participant level data

Not provided at time of registration

Results - basic reporting

Publication summary

2014 results in: http://www.ncbi.nlm.nih.gov/pubmed/25140956

Publication citations

  1. Results

    DeBaun MR, Gordon M, McKinstry RC, Noetzel MJ, White DA, Sarnaik SA, Meier ER, Howard TH, Majumdar S, Inusa BP, Telfer PT, Kirby-Allen M, McCavit TL, Kamdem A, Airewele G, Woods GM, Berman B, Panepinto JA, Fuh BR, Kwiatkowski JL, King AA, Fixler JM, Rhodes MM, Thompson AA, Heiny ME, Redding-Lallinger RC, Kirkham FJ, Dixon N, Gonzalez CE, Kalinyak KA, Quinn CT, Strouse JJ, Miller JP, Lehmann H, Kraut MA, Ball WS, Hirtz D, Casella JF, Controlled trial of transfusions for silent cerebral infarcts in sickle cell anemia., N. Engl. J. Med., 2014, 371, 8, 699-710, doi: 10.1056/NEJMoa1401731.

Additional files

Editorial Notes