Stroke Prevention in Sickle Cell Anemia (STOP 2)

• ClinicalTrials.gov Identifier: NCT00006182
• Recruitment Status: Completed
• First Posted: August 22, 2000
• Last Update Posted: February 18, 2016
• Sponsor: National Heart, Lung, and Blood Institute (NHLBI)
• Information provided by: National Heart, Lung, and Blood Institute (NHLBI)

Tracking Information

• First Submitted Date: August 21, 2000
• First Posted Date: August 22, 2000
• Last Update Posted Date: February 18, 2016
• Study Start Date: July 2000
• Primary Completion Date: Not Provided
• Current Primary Outcome Measures: Not Provided
• Original Primary Outcome Measures: Not Provided
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Stroke Prevention in Sickle Cell Anemia (STOP 2)
• Official Title: Not Provided
• Brief 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.

Detailed 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.

• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: Randomized; Primary Purpose: Prevention

Condition

• Blood Disease
• Cerebrovascular Accident
• Anemia, Sickle Cell

• Intervention: Procedure: Blood Transfusion
• Study Arms: Not Provided

Publications *

• 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.
• 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.
• 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.
• 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.
• 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.

Recruitment Information

• Recruitment Status: Completed
• Enrollment: Not Provided
• Original Enrollment: Not Provided
• Actual Study Completion Date: February 2006
• Primary Completion Date: Not Provided
• Eligibility Criteria: No eligibility criteria

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 2 Years to 20 Years (Child, Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Not Provided

Administrative Information

• NCT Number: NCT00006182
• Other Study ID Numbers: 68; U01HL052016 ( U.S. NIH Grant/Contract )
• Has Data Monitoring Committee: Not Provided
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: Not Provided
• Original Responsible Party: Same as current
• Current Study Sponsor: National Heart, Lung, and Blood Institute (NHLBI)
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Investigator: Robert Adams; Augusta University
• Investigator: Donald Brambilla; New England Research Institutes, Inc.

• PRS Account: National Heart, Lung, and Blood Institute (NHLBI)
• Verification Date: October 2005