NCT07226219 · St. Jude Children's Research Hospital
Methylphenidate to Address Attention and Executive Deficits Among Children With Sickle Cell Disease
What this study is about
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. The study will assess any thinking or attention problems participants may have both before taking this drug and after.
View original scientific description
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. The 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. Primary Objective: • Assess the feasibility, acceptability, and adherence to MPH treatment in children with SCD and EF deficits. Secondary Objective: • Evaluate neurobehavioral and safety outcomes following MPH treatment. Exploratory Objective: • Evaluate decision-making and determinants influencing methylphenidate utilization among parents.
Interventions
DRUG
Extended-Release Methylphenidate
Participants will receive a weight-based dose of extended-release methylphenidate (0.6 mg/kg/day), rounded to either 10 mg or 20 mg, taken orally once daily for 4 weeks.
Primary outcome measures
Assess feasibility of methylphenidate
Time frame: Feasibility is measured during the initial recruitment process for each participant.
Feasibility is measured by the participation rate (i.e., ratio of those who agree to participate to those approached).
Assess acceptability of methylphenidate
Time frame: Acceptability ratings are captured at baseline and after 4 weeks of treatment with methylphenidate.
Acceptability is captured by parent- and self-report ratings on the Acceptability of Intervention Measure (AIM).
Assess adherence to methylphenidate
Time frame: Adherence is measured on a weekly basis through 4 weeks of treatment
Adherence is measured through weekly pill counts. The primary adherence outcome is the ratio of the number of pills taken to those dispersed.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- 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 \*Included if performance measure, rating scale or diagnostic criteria met (within the past 2 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/ 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 / hyperactivity disorder (any subtype)
- English as the primary language
- Research participant and one parent willing to participate and provide consent/assent according to institutional guidelines
- Negative pregnancy test
Exclusion criteria
- 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/representative 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
Where
- Memphis, Tennessee
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jul 6, 2026 · Source of record for eligibility and locations