Methylphenidate for attention and executive problems in children with sickle cell disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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

Where it's running

• 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

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.