Stroke Prevention in Sickle Cell Anemia (STOP 2)

Cerebrovascular Accident Clinical Trial

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00006182
• Other study ID #: 68
• Secondary ID: U01HL052016
• Status: Completed
• Phase: Phase 3
• First received: August 21, 2000
• Last updated: February 17, 2016
• Start date: July 2000
• Est. completion date: February 2006

Study information

• Verified date: October 2005
• Source: National Heart, Lung, and Blood Institute (NHLBI)
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Federal Government
• Study type: Interventional

Clinical Trial Summary

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.

Description:

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 0
• Est. completion date: February 2006
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 2 Years to 20 Years
• Eligibility: No eligibility criteria

Study Design

Allocation: Randomized, Primary Purpose: Prevention

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Blood Disease
• Cerebrovascular Accident
• Hematologic Diseases
• Stroke

Intervention

Procedure:
• Blood Transfusion

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
National Heart, Lung, and Blood Institute (NHLBI)

References & Publications (4)

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. — View Citation

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. — View Citation

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. — View Citation

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. — View Citation