Recruiting
Phase 1

Methylphenidate

Sponsor:

St. Jude Children's Research Hospital

Code:

NCT07226219

Conditions

Sickle Cell Disease

Executive Dysfunction

Cognitive Impairment

Attention Deficit/Hyperactivity Disorder (ADHD)

Eligibility Criteria

Sex: All

Age: 8 - 17

Healthy Volunteers: Not accepted

Interventions

Extended-Release Methylphenidate

Study Details

Brief summary:

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 caregivers.

Conditions

Sickle Cell Disease

Executive Dysfunction

Cognitive Impairment

Attention Deficit/Hyperactivity Disorder (ADHD)

Study ID

NCT07226219

Start date

Nov 25, 2025

Status verified date

Sep, 2026

Completion date

Aug 1, 2028

Anticipated

Primary completion date

Jul 1, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 8 - 17

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • Diagnosed with SCD
  • 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 a Standard Score of 85 (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 a T Score of 60 (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 caregiver willing to participate and provide consent/assent according to institutional guidelines
  • Negative pregnancy test in female participant of childbearing age

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 (ever)
  • 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.
  • Diagnosed with sickle cell-hereditary persistence of fetal hemoglobin (HbSHPFH), sickle cell homozygous C (CC), Hb S/N-Baltimore, or other rare genotypes that would preclude the participant from having the classic symptoms of sickle cell disease
  • 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

Study Design

Enrollment

72 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Methylphenidate Treatment Group

All participants in this single-arm pilot study will receive extended-release methylphenidate for 4 weeks. The intervention is designed to evaluate feasibility, acceptability, adherence, and safety of stimulant treatment in children and adolescents with sickle cell disease (SCD) and executive functioning deficits.

Interventions

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 measure

  • Assess feasibility of methylphenidate [ Time Frame: Feasibility is measured during the initial recruitment process for each participant. ]
  • Assess acceptability of methylphenidate [ Time Frame: Acceptability ratings are captured at baseline and after 4 weeks of treatment with methylphenidate. ]
  • Assess adherence to methylphenidate [ Time Frame: Adherence is measured on a weekly basis through 4 weeks of treatment ]

Central Contacts and Locations

Central contacts

Andrew Heitzer, PhD

888-226-4343referralinfo@stjude.org

Locations

St. Jude Children's Research Hospital

Recruiting

Memphis, Tennessee, United States, 38105

Contacts

Andrew Heitzer, PhD

888-226-4343referralinfo@stjude.org

Principal Investigator:

Andrew K Heitzer, PhD

More Information

Sponsor

St. Jude Children's Research Hospital

Last update posted

Sep 9, 2026

Last verified

Sep, 2026

Keywords

  • SCD
  • ADHD

Trial information was received from ClinicalTrials.gov and was last updated on 2026-10-25. This information was provided to ClinicalTrials.gov by St. Jude Children's Research Hospital on 2026-09-09. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.