[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT07226219":3,"trial-entities:NCT07226219":161,"trial-summary:NCT07226219":165},{"id":4,"nct_id":4,"org_study_id":5,"brief_title":6,"official_title":7,"overall_status":8,"completion_date":9,"status_verified_date":10,"last_update_date":11,"start_date":12,"sponsor_name":13,"lead_sponsor_class":14,"has_dmc":15,"brief_summary":16,"detailed_description":17,"conditions":18,"keywords":23,"study_type":26,"primary_purpose":27,"phases":28,"enrollment_info":30,"interventions":33,"primary_outcomes":42,"secondary_outcomes":55,"sex":84,"minimum_age":85,"maximum_age":86,"healthy_volunteers":15,"eligibility_criteria":87,"std_ages":129,"locations":131,"central_contacts":149,"overall_officials":151,"references":154,"see_also_links":155},"NCT07226219","STARS","Methylphenidate to Address Attention and Executive Deficits Among Children With Sickle Cell Disease","Pilot Trial of Stimulant Treatment to Address Attention and Executive Deficits Among Children With Sickle Cell Disease","RECRUITING","2028-08-01","2026-07","2026-07-06","2025-11-25","St. Jude Children's Research Hospital","OTHER",false,"The purpose of this study is to determine if patients with sickle cell disease (SCD) can consistently take a drug called Methylphenidate (MPH) daily, once a day for 4 weeks to help with any thinking, attention or schoolwork problems and if they have any side effects.\n\nThe study will assess any thinking or attention problems participants may have both before taking this drug and after. Additionally, the study will assess the decision-making process of the caregiver that may influence using this drug or not.\n\nPrimary Objective:\n\n• Assess the feasibility, acceptability, and adherence to MPH treatment in children with SCD and EF deficits.\n\nSecondary Objective:\n\n• Evaluate neurobehavioral and safety outcomes following MPH treatment.\n\nExploratory Objective:\n\n• Evaluate decision-making and determinants influencing methylphenidate utilization among parents.","Children with sickle cell disease (SCD) are at higher risk for executive functioning (EF) deficits, including attention, working memory, and inhibitory control. These deficits are associated with poor academic performance, reduced quality of life, and challenges transitioning to adult healthcare. Despite the effectiveness of stimulant medications like methylphenidate (MPH) in improving EF in the general population and other medical groups, their use in children with SCD is rare.\n\nThis is a single-arm, open-label pilot trial conducted at St. Jude Children's Research Hospital. Thirty children with SCD and EF deficits will receive a 4-week course of extended-release MPH (10 mg or 20 mg daily, based on weight). Extended-release methylphenidate will be administered once daily for 4 weeks. The initial dose will be given in clinic, followed by home administration. Adherence will be monitored via weekly video pill counts.\n\nThe study will enroll 30 patients aged 8.0 to 17.9 years with SCD and EF impairment, along with 30 caregivers. An additional 12 caregivers who decline participation will be interviewed to assess decision-making and treatment barriers.\n\nNeurobehavioral assessments and side effect evaluations will be conducted at baseline, immediately post-dose, and weekly during the home medication phase. Parents will complete rating scales and interviews to assess treatment acceptability and decision-making.",[19,20,21,22],"Sickle Cell Disease","Executive Dysfunction","Cognitive Impairment","Attention Deficit\u002FHyperactivity Disorder (ADHD)",[24,25],"SCD","ADHD","INTERVENTIONAL","TREATMENT",[29],"PHASE1",{"count":31,"type":32},72,"ESTIMATED",[34],{"type":35,"name":36,"description":37,"armGroupLabels":38,"otherNames":40},"DRUG","Extended-Release Methylphenidate","Participants will receive a weight-based dose of extended-release methylphenidate (0.6 mg\u002Fkg\u002Fday), rounded to either 10 mg or 20 mg, taken orally once daily for 4 weeks.",[39],"Methylphenidate Treatment Group",[41],"MPH",[43,47,51],{"measure":44,"description":45,"timeFrame":46},"Assess feasibility of methylphenidate","Feasibility is measured by the participation rate (i.e., ratio of those who agree to participate to those approached).","Feasibility is measured during the initial recruitment process for each participant.",{"measure":48,"description":49,"timeFrame":50},"Assess acceptability of methylphenidate","Acceptability is captured by parent- and self-report ratings on the Acceptability of Intervention Measure (AIM).","Acceptability ratings are captured at baseline and after 4 weeks of treatment with methylphenidate.",{"measure":52,"description":53,"timeFrame":54},"Assess adherence to methylphenidate","Adherence is measured through weekly pill counts. The primary adherence outcome is the ratio of the number of pills taken to those dispersed.","Adherence is measured on a weekly basis through 4 weeks of treatment",[56,60,63,66,68,70,74,77,81],{"measure":57,"description":58,"timeFrame":59},"Behavior Assessment System for Children, 3rd Edition (BASC-3), Parent Report","Attention, Hyperactivity, and Depression scales are calculated. The investigators will determine the amount and pattern of missing data. The relative change (defined as (post - pre)\u002Fpre)) will be calculated for each outcome and rank these changes to determine sensitivity of the measures. The goal of measuring neurobehavioral outcomes in this pilot trial is to determine how feasible the measures are for the target population and their relative sensitivity to the effects of methylphenidate treatment.","Baseline and 4-6 weeks after treatment",{"measure":61,"description":62,"timeFrame":59},"Behavior Rating Inventory of Executive Function, 2nd Edition (BRIEF-2), Parent Report","Global Executive Composite Scale is calculated. The investigator will determine the amount and pattern of missing data for neurobehavioral outcomes. The relative change (defined as (post - pre)\u002Fpre)) will be calculated for each outcome and rank these changes to determine sensitivity of the measures. The goal of measuring neurobehavioral outcomes in this pilot trial is to determine how feasible the measures are for the target population and their relative sensitivity to the effects of methylphenidate treatment.",{"measure":64,"description":65,"timeFrame":59},"Pediatric Quality of Life Inventory (PedsQL) Sickle Cell Disease modules, Parent Report","The investigator will determine the amount and pattern of missing data for neurobehavioral outcomes. The relative change (defined as (post - pre)\u002Fpre)) will be calculated for each outcome and rank these changes to determine sensitivity of the measures. The goal of measuring neurobehavioral outcomes in this pilot trial is to determine how feasible the measures are for the target population and their relative sensitivity to the effects of methylphenidate treatment.",{"measure":67,"description":65,"timeFrame":59},"Pediatric Quality of Life Inventory (PedsQL) Multidimensional Fatigue modules, Parent Report",{"measure":69,"description":65,"timeFrame":59},"Conners 4th Edition Short Form, Self Report",{"measure":71,"description":72,"timeFrame":73},"NIH Toolbox Cognition","Dimensional Change Card Cort, Flanker, Pattern Comparison, Picture Sequence Memory Test, Picture Vocabulary subtests are completed. The investigator will determine the amount and pattern of missing data for neurobehavioral outcomes. The relative change (defined as (post - pre)\u002Fpre)) will be calculated for each outcome and rank these changes to determine sensitivity of the measures. The goal of measuring neurobehavioral outcomes in this pilot trial is to determine how feasible the measures are for the target population and their relative sensitivity to the effects of methylphenidate treatment.","Baseline and ~90 minutes after first dose administered",{"measure":75,"description":76,"timeFrame":73},"Woodcock Johnson Tests of Academic Achievement, 4th Edition (WJ-IV)","Math Fluency subtest is completed. The investigator will determine the amount and pattern of missing data for neurobehavioral outcomes. The relative change (defined as (post - pre)\u002Fpre)) will be calculated for each outcome and rank these changes to determine sensitivity of the measures. The goal of measuring neurobehavioral outcomes in this pilot trial is to determine how feasible the measures are for the target population and their relative sensitivity to the effects of methylphenidate treatment.",{"measure":78,"description":79,"timeFrame":80},"Measure key safety outcomes using the Side Effects Rating Scale (SERS)","The investigator will record all adverse effects reported by participants and categorize adverse effects by type and severity based on the Side Effects Rating Scale. The investigator will present summary statistics, including the number of participants experiencing each type of adverse effect, the severity and the overall percentage of participants affected.","Baseline in-clinic assessments followed by remote weekly assessments for 4 weeks",{"measure":82,"description":83,"timeFrame":80},"Measure key safety outcomes using the Systematic Assessment for Treatment Emergent Effects","The investigator will record all adverse effects reported by participants and categorize adverse effects by type and severity based on the Systematic Assessment for Treatment Emergent Effects. The investigator will present summary statistics, including the number of participants experiencing each type of adverse effect, the severity, and the overall percentage of participants affected","ALL","8 Years","17 Years",{"inclusion":88,"exclusion":104,"raw_text":128},[89,90,91,92,93,94,95,96,97,98,99,100,101,102,103],"Diagnosed with SCD of any genotype","Enrolled on the institutional protocol: Sickle Cell Clinical Research Intervention Program (SCCRIP)","Between the ages of 8.0 and 17.9 years","\\*Score at or below the 16th percentile on any 2 out of 4 performance measures:","NIH Toolbox Flanker","NIH Toolbox List Sorting","NIH Toolbox Dimensional Change Card Sort Test (DCST)","Wechsler Intelligence Scale for Children (WISC) -5\u002F Wechsler Adult Intelligence Scale (WAIS)-4 Digit Span Forward (DSF)","\\*Score at or above the 84th percentile on any 1 out of 2 parent rating scales:","BRIEF-2 Global Executive","BASC-3 Attention","\\*Have a documented diagnosis of attention deficit \u002F hyperactivity disorder (any subtype)","English as the primary language","Research participant and one parent willing to participate and provide consent\u002Fassent according to institutional guidelines","Negative pregnancy test",[105,106,107,108,109,110,111,112,113,114,115,116,117,118,119,120,121,122,123,124,125,126,127],"Primary language other than English","Score below the 2nd percentile on the Wechsler Abbreviated Scale of Intelligence (WASI)-2 intelligence quotient (IQ) test","Uncontrolled seizures (seizure within the past 6 months)","Cardiomyopathy or known congenital structural cardiac defects","Stenotic valvular disease, left coronary artery stenosis, or history of myocarditis or pericarditis","History of heart arrhythmia including ventricular tachycardia, ventricular fibrillation, supraventricular tachycardia, QT prolongation or concomitant use of medications associated with QT prolongation","Two or more prior episodes of priapism","Blood pressure \\>95th percentile at the three most recent visits consecutively (i.e., \\>95th percentile reading at all three of the most recent hospital visits to St. Jude).","If blood pressure is \\> 95th %ile compared to age-norms on the day of the baseline visit, a repeat blood-pressure reading will be performed both electronically and manually to confirm findings.","Stimulant medication within the past two weeks","Severe sensory loss","Previous adverse reaction to methylphenidate","Inability or unwillingness of research participant or legal guardian\u002Frepresentative to give written informed consent.","Currently prescribed another investigational medication.","Currently prescribed any of the following:","Phenobarbital (anticonvulsant)","Phenytoin (anticonvulsant)","Primidone (anticonvulsant)","Warfarin (anticoagulant)","Antipsychotic medications","Selective Serotonin Reuptake Inhibitor (SSRI) medications","Tricyclic antidepressant (TCA) medications","Vasopressor medications","Inclusion Criteria:\n\n* Diagnosed with SCD of any genotype\n* Enrolled on the institutional protocol: Sickle Cell Clinical Research Intervention Program (SCCRIP)\n* Between the ages of 8.0 and 17.9 years\n\n  \\*Included if performance measure, rating scale or diagnostic criteria met (within the past 2 years):\n* \\*Score at or below the 16th percentile on any 2 out of 4 performance measures:\n\n  * NIH Toolbox Flanker\n  * NIH Toolbox List Sorting\n  * NIH Toolbox Dimensional Change Card Sort Test (DCST)\n  * Wechsler Intelligence Scale for Children (WISC) -5\u002F Wechsler Adult Intelligence Scale (WAIS)-4 Digit Span Forward (DSF)\n* \\*Score at or above the 84th percentile on any 1 out of 2 parent rating scales:\n\n  * BRIEF-2 Global Executive\n  * BASC-3 Attention\n* \\*Have a documented diagnosis of attention deficit \u002F hyperactivity disorder (any subtype)\n* English as the primary language\n* Research participant and one parent willing to participate and provide consent\u002Fassent according to institutional guidelines\n* Negative pregnancy test\n\nExclusion Criteria:\n\n* Primary language other than English\n* Score below the 2nd percentile on the Wechsler Abbreviated Scale of Intelligence (WASI)-2 intelligence quotient (IQ) test\n* Uncontrolled seizures (seizure within the past 6 months)\n* Cardiomyopathy or known congenital structural cardiac defects\n* Stenotic valvular disease, left coronary artery stenosis, or history of myocarditis or pericarditis\n* History of heart arrhythmia including ventricular tachycardia, ventricular fibrillation, supraventricular tachycardia, QT prolongation or concomitant use of medications associated with QT prolongation\n* Two or more prior episodes of priapism\n* Blood pressure \\>95th percentile at the three most recent visits consecutively (i.e., \\>95th percentile reading at all three of the most recent hospital visits to St. Jude).\n\n  * If blood pressure is \\> 95th %ile compared to age-norms on the day of the baseline visit, a repeat blood-pressure reading will be performed both electronically and manually to confirm findings.\n* Stimulant medication within the past two weeks\n* Severe sensory loss\n* Previous adverse reaction to methylphenidate\n* Inability or unwillingness of research participant or legal guardian\u002Frepresentative to give written informed consent.\n* Currently prescribed another investigational medication.\n* Currently prescribed any of the following:\n\n  * Phenobarbital (anticonvulsant)\n  * Phenytoin (anticonvulsant)\n  * Primidone (anticonvulsant)\n  * Warfarin (anticoagulant)\n  * Antipsychotic medications\n  * Selective Serotonin Reuptake Inhibitor (SSRI) medications\n  * Tricyclic antidepressant (TCA) medications\n  * Vasopressor medications",[130],"CHILD",[132],{"facility":13,"status":8,"city":133,"state":134,"zip":135,"country":136,"contacts":137,"geoPoint":146},"Memphis","Tennessee","38105","United States",[138,143],{"name":139,"role":140,"phone":141,"email":142},"Andrew Heitzer, PhD","CONTACT","888-226-4343","referralinfo@stjude.org",{"name":144,"role":145},"Andrew K Heitzer, PhD","PRINCIPAL_INVESTIGATOR",{"lat":147,"lon":148},35.14953,-90.04898,[150],{"name":139,"role":140,"phone":141,"email":142},[152],{"name":153,"affiliation":13,"role":145},"Andrew W. Heitzer, PhD",[],[156,158],{"label":13,"url":157},"http:\u002F\u002Fwww.stjude.org",{"label":159,"url":160},"Clinical Trials Open at St. Jude","http:\u002F\u002Fwww.stjude.org\u002Fprotocols",{"nct_id":4,"conditions":162,"biomarkers":164},[163,19],"Attention Deficit Hyperactivity Disorder",[],{"nct_id":4,"found":166,"summary":167,"prompt_version":177},true,{"design":168,"status":169,"heading":170,"summary":171,"follow_up":172,"word_count":173,"commitments":174,"compensation":175,"drugs_mentioned":176},"This is a single-arm, open-label pilot study involving 30 children with sickle cell disease and 30 caregivers. An additional 12 caregivers who decline participation will also be interviewed.","completed","Methylphenidate for Attention and Executive Deficits in Children with Sickle Cell Disease","This study is testing if children with sickle cell disease (SCD) who have problems with attention, thinking, or schoolwork can safely and consistently take a medication called extended-release methylphenidate. This medication is commonly used to help with attention and executive function (skills like planning and problem-solving). You would take a weight-based dose of extended-release methylphenidate once daily for 4 weeks. Researchers want to see if it's practical for children with SCD to take this medication, if they find it acceptable, and if they take it as prescribed. They will also look at how it affects thinking skills and any side effects. The study is currently recruiting 30 participants aged 8 to 17 years old who have SCD and specific difficulties with attention or executive function.","Acceptability ratings are captured after 4 weeks of treatment. Adherence is measured weekly through 4 weeks of treatment.",125,"You would take extended-release methylphenidate once daily for 4 weeks. You would have neurobehavioral assessments and side effect evaluations at the start, immediately after the first dose, and weekly during the home medication phase.","Not stated in the trial record.",[36],"v2"]