💬In plain words
What the trial is, and whether it could suit you
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.
Sign in to get the eligibility rules as simple questions to take to your doctor.
📋 Eligibility rules (as published)
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
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