Stroke Prevention in Sickle Cell Anemia (STOP 2)

NCT ID: NCT00006182

Last Updated: 2016-02-18

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Study Classification

INTERVENTIONAL

Study Start Date

2000-07-31

Study Completion Date

2006-02-28

Brief Summary

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To determine how long blood transfusions are needed for primary stroke prevention. Also, to determine the duration of risk associated with abnormal transcranial Doppler ultrasound (TCD) and to determine the specificity of the stroke risk model developed in STOP 1 in patients with abnormal TCD measurements.

Detailed Description

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BACKGROUND:

Without intervention, 11 percent of children with sickle cell anemia will sustain a stroke before the age of 20. The STOP 2 follows on the recently completed NHLBI trial, Stroke Prevention Trial in Sickle Cell Anemia (STOP 1), which found that patients at high risk for stroke could be identified using transcranial Doppler (TCD) ultrasound, and that the incidence of stroke could be reduced by 90 percent in those children by periodic blood transfusion for at least 36 months. However, chronic intermittent blood transfusions are cumbersome, expensive and associated with morbidity from iron overload. Thus, this study to determine if transfusion can be safely halted after 30 months of treatment is critically important to the continued clinical care of patients with sickle cell disease at risk for stroke.

DESIGN NARRATIVE:

In this multicenter, randomized clinical trial, 100 children will be randomized to continue to receive periodic transfusion therapy and 50 to discontinue receiving periodic transfusion therapy. The primary endpoint will be the reversion of the transcranial Doppler velocity levels to greater than 200 cm/second, indicating return of increased risk for overt stroke. Recruitment will be in two phases. Phase I will include those patients who began transfusions before April 1, 1999. These will come mainly from the STOP 1 cohort. Those who begin transfusions after April 1, 1999 but before April 1, 2001 will be eligible for Phase II of recruitment. All patients will receive quarterly TCD examinations. Patients who revert to high risk will be offered return to transfusion. The overall design includes a three month start-up, the two phases of recruitment (established STOP patients and new patients) for a total of 36 months, 18 months of observations after recruitment ends and then 3 months of wrap-up.

The study completion date listed in this record was obtained from the "End Date" entered in the Protocol Registration and Results System (PRS) record.

Conditions

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Blood Disease Cerebrovascular Accident Anemia, Sickle Cell

Study Design

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Allocation Method

RANDOMIZED

Primary Study Purpose

PREVENTION

Interventions

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Blood Transfusion

Intervention Type PROCEDURE

Eligibility Criteria

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Inclusion Criteria

No eligibility criteria
Minimum Eligible Age

2 Years

Maximum Eligible Age

20 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Heart, Lung, and Blood Institute (NHLBI)

NIH

Sponsor Role lead

Principal Investigators

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Robert Adams

Role:

Augusta University

Donald Brambilla

Role:

New England Research Institutes, Inc.

References

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Vichinsky EP, Luban NL, Wright E, Olivieri N, Driscoll C, Pegelow CH, Adams RJ; Stroke Prevention Trail in Sickle Cell Anemia. Prospective RBC phenotype matching in a stroke-prevention trial in sickle cell anemia: a multicenter transfusion trial. Transfusion. 2001 Sep;41(9):1086-92. doi: 10.1046/j.1537-2995.2001.41091086.x.

Reference Type BACKGROUND
PMID: 11552063 (View on PubMed)

Kral MC, Brown RT. Transcranial Doppler ultrasonography and executive dysfunction in children with sickle cell disease. J Pediatr Psychol. 2004 Apr-May;29(3):185-95. doi: 10.1093/jpepsy/jsh020.

Reference Type BACKGROUND
PMID: 15131136 (View on PubMed)

Wang WC, Morales KH, Scher CD, Styles L, Olivieri N, Adams R, Brambilla D; STOP Investigators. Effect of long-term transfusion on growth in children with sickle cell anemia: results of the STOP trial. J Pediatr. 2005 Aug;147(2):244-7. doi: 10.1016/j.jpeds.2005.02.030.

Reference Type BACKGROUND
PMID: 16126058 (View on PubMed)

Adams RJ, Brambilla D; Optimizing Primary Stroke Prevention in Sickle Cell Anemia (STOP 2) Trial Investigators. Discontinuing prophylactic transfusions used to prevent stroke in sickle cell disease. N Engl J Med. 2005 Dec 29;353(26):2769-78. doi: 10.1056/NEJMoa050460.

Reference Type RESULT
PMID: 16382063 (View on PubMed)

Adamkiewicz TV, Abboud MR, Paley C, Olivieri N, Kirby-Allen M, Vichinsky E, Casella JF, Alvarez OA, Barredo JC, Lee MT, Iyer RV, Kutlar A, McKie KM, McKie V, Odo N, Gee B, Kwiatkowski JL, Woods GM, Coates T, Wang W, Adams RJ. Serum ferritin level changes in children with sickle cell disease on chronic blood transfusion are nonlinear and are associated with iron load and liver injury. Blood. 2009 Nov 19;114(21):4632-8. doi: 10.1182/blood-2009-02-203323. Epub 2009 Aug 31.

Reference Type DERIVED
PMID: 19721013 (View on PubMed)

Other Identifiers

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U01HL052016

Identifier Type: NIH

Identifier Source: secondary_id

View Link

68

Identifier Type: -

Identifier Source: org_study_id

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