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

NCT07226219 | Recruiting Phase 1 FDA Drug

• 1 other identifier: STARS
• Study Type: interventional
• Target: 72
• Locations: 1 country
• Sites: 1

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 parents.

Conditions

Sickle Cell Disease; Executive Dysfunction; Cognitive Impairment; Attention Deficit/Hyperactivity Disorder (ADHD)

Keywords

SCD; ADHD

Outcome Measures

Primary Outcomes (3)

• Assess feasibility of methylphenidate

  Feasibility is measured by the participation rate (i.e., ratio of those who agree to participate to those approached).

  Feasibility is measured during the initial recruitment process for each participant.

• Assess acceptability of methylphenidate

  Acceptability is captured by parent- and self-report ratings on the Acceptability of Intervention Measure (AIM).

  Acceptability ratings are captured at baseline and after 4 weeks of treatment with methylphenidate.

• Assess adherence to methylphenidate

  Adherence is measured through weekly pill counts. The primary adherence outcome is the ratio of the number of pills taken to those dispersed.

  Adherence is measured on a weekly basis through 4 weeks of treatment

Secondary Outcomes (9)

• Behavior Assessment System for Children, 3rd Edition (BASC-3), Parent Report

  Baseline and 4-6 weeks after treatment

• Behavior Rating Inventory of Executive Function, 2nd Edition (BRIEF-2), Parent Report

  Baseline and 4-6 weeks after treatment

• Pediatric Quality of Life Inventory (PedsQL) Sickle Cell Disease modules, Parent Report

  Baseline and 4-6 weeks after treatment

• Pediatric Quality of Life Inventory (PedsQL) Multidimensional Fatigue modules, Parent Report

  Baseline and 4-6 weeks after treatment

• Conners 4th Edition Short Form, Self Report

  Baseline and 4-6 weeks after treatment

Other Outcomes (1)

• Evaluate decision-making and determinants influencing methylphenidate utilization among parents

  Baseline and 4-6 weeks after treatment

Study Arms (1)

Methylphenidate Treatment Group

EXPERIMENTAL

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.

Drug: Extended-Release Methylphenidate

Interventions

Extended-Release Methylphenidate DRUG

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.

Also known as: MPH

Methylphenidate Treatment Group

Eligibility Criteria

• Age: 8 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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:

Sponsors & Collaborators

• St. Jude Children's Research Hospital: lead

Study Sites (1)

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

RECRUITING

Related Links

• St. Jude Children's Research Hospital
• Clinical Trials Open at St. Jude

MeSH Terms

Conditions

Anemia, Sickle Cell; Cognitive Dysfunction; Attention Deficit Disorder with Hyperactivity

Interventions

5,10-dihydro-5-methylphenazine

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Cognition Disorders; Neurocognitive Disorders; Mental Disorders; Attention Deficit and Disruptive Behavior Disorders; Neurodevelopmental Disorders

Study Officials

• Andrew W. Heitzer, PhD

  St. Jude Children's Research Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Andrew Heitzer, PhD

CONTACT

888-226-4343; referralinfo@stjude.org

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: 30 patients + 42 caregivers (including 12 caregivers who declined participation for interviews)

Study Record Dates

• First Submitted: October 22, 2025
• First Posted: November 10, 2025
• Study Start: November 25, 2025
• Primary Completion (Estimated): July 1, 2027
• Study Completion (Estimated): August 1, 2028
• Last Updated: April 23, 2026

Record last verified: 2026-04

Locations

US (1)