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Methylphenidate to Address Attention and Executive Deficits Among Children With Sickle Cell Disease

Pilot Trial of Stimulant Treatment to Address Attention and Executive Deficits Among Children With Sickle Cell Disease

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
8 Years to 17 Years
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

  • Time periods mentioned: 4 weeks
  • Procedures mentioned: Pills, Interviews

Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.

Show the study's own words
  • "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."
  • "Extended-release methylphenidate will be administered once daily for 4 weeks."
  • "Adherence will be monitored via weekly video pill counts."
  • "Caregivers will complete rating scales and interviews to assess treatment acceptability and decision-making."
Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests whether children ages 8 to 17 with sickle cell disease and thinking or attention problems can take methylphenidate once a day for 4 weeks. It checks for side effects and changes in thinking and attention. It also looks at how caregivers decide whether to use this medicine.

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.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Have sickle cell disease and be 8 to 17 years old.
  • Be enrolled in the Sickle Cell Clinical Research Intervention Program (SCCRIP).
  • Meet at least one of the next three conditions within the past 2 years.
  • Score 85 or lower on at least 2 of the 4 listed thinking and attention tests.
  • Have a parent rating score of 60 or higher on either the BRIEF-2 Global Executive or BASC-3 Attention scale.
  • Have a recorded diagnosis of ADHD, a condition that affects attention or activity level.
  • Use English as the main language. The child and one caregiver must be willing to take part and give the required permission.
  • Have a negative pregnancy test if the participant is female and able to become pregnant.

Who can't join

  • Use a main language other than English, or the child or legal guardian cannot or will not give written permission.
  • Have ever scored below the 2nd percentile on the WASI-2 IQ test, which measures thinking skills; have severe loss of senses; or have had a seizure in the past 6 months.
  • Have heart muscle disease, a heart structure problem present at birth, narrowed heart valves or left coronary artery, past inflammation in or around the heart, or a history of abnormal heart rhythms.
  • Have blood pressure above the 95th percentile at all 3 most recent St. Jude hospital visits, or have had at least 2 episodes of priapism (an erection that lasts too long).
  • Have used a stimulant medicine in the past 2 weeks, had a bad reaction to methylphenidate before, or currently take another experimental medicine.
  • Have HbSHPFH, CC, Hb S/N-Baltimore, or another rare gene type that prevents the usual symptoms of sickle cell disease.
  • Currently take phenobarbital, phenytoin, or primidone (seizure medicines); warfarin (a blood thinner); or vasopressors (medicines that raise blood pressure).
  • Currently take antipsychotic medicines, SSRI or tricyclic antidepressants, or medicines linked to QT prolongation (a change in the heart's electrical timing).

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

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 sites (1)

TN