Methylphenidate for attention and executive problems in children with sickle cell disease
Pilot Trial of Stimulant Treatment to Address Attention and Executive Deficits Among Children With Sickle Cell Disease
This study will test whether once-daily extended-release methylphenidate for 4 weeks helps attention and thinking skills in children 8–17 with sickle cell disease while monitoring side effects and adherence.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 72 (estimated) |
| Ages | 8 Years to 17 Years |
| Sex | All |
| Sponsor | St. Jude Children's Research Hospital Academic / other |
| Locations | 1 site (Memphis, Tennessee) |
| Trial ID | NCT07226219 on ClinicalTrials.gov |
What this trial studies
This single-arm, open-label pilot will give 30 children with sickle cell disease and measured executive function deficits a once-daily extended-release methylphenidate dose (10 mg or 20 mg based on weight) for 4 weeks. Baseline neurobehavioral testing and safety assessments are performed, the first dose is given in clinic, and weekly home dosing is monitored via video pill counts with weekly assessments and parent rating scales. Caregivers complete interviews about acceptability and decision-making, and up to 12 caregivers who decline medication will be interviewed to explore barriers. The trial will report feasibility, adherence, short-term neurobehavioral changes, and safety signals to inform larger studies.
Who should consider this trial
Good fit: Children aged 8.0–17.9 years with any-genotype sickle cell disease who meet objective or parent-report criteria for executive-function impairment, are enrolled in the SCCRIP protocol, speak English, and have a caregiver willing to participate are ideal candidates.
Not a fit: Children without measurable executive-function deficits, outside the 8.0–17.9 age range, not enrolled in SCCRIP, or with medical contraindications to stimulant therapy are unlikely to benefit from this protocol.
Why it matters
Potential benefit: If successful, participants may experience improved attention, working memory, classroom functioning, and overall quality of life.
How similar studies have performed: Stimulant medications like methylphenidate are well established to improve attention in pediatric ADHD, but their use and formal testing in children with sickle cell disease remains limited, making this approach partially evidence-based but novel in SCD.
Eligibility criteria
Show full inclusion / exclusion criteria
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 this trial is running
Memphis, Tennessee
- St. Jude Children's Research Hospital — Memphis, Tennessee, United States (Recruiting)
Study contacts
- Principal investigator: Andrew W. Heitzer, PhD — St. Jude Children's Research Hospital
- Study coordinator: Andrew Heitzer, PhD
- Email: referralinfo@stjude.org
- Phone: 888-226-4343
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.