Methylphenidate for Sickle Cell Disease

Age: < 18

Sex: Any

Trial Phase: Phase 1

Sponsor: St. Jude Children's Research Hospital

Must not be taking: Anticonvulsants, Anticoagulants, Antipsychotics, others

Disqualifiers: Uncontrolled seizures, Heart arrhythmia, Hypertension, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 1

Duration: 4 weeks

72 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests whether Methylphenidate, a stimulant medication, can aid children and teens with sickle cell disease who experience difficulties with thinking, attention, or schoolwork. Participants will take the medication once daily for four weeks to assess its safety and adherence. The trial also examines parental decision-making regarding the use of this medication. It suits children aged 8 to 17 with sickle cell disease who have attention or executive function challenges, such as planning and organizing tasks. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants a chance to contribute to groundbreaking research.

Do I need to stop my current medications for the trial?

Yes, you will need to stop taking certain medications to participate in the trial. Specifically, you cannot be on stimulant medications within the past two weeks or currently take medications like anticonvulsants, warfarin, antipsychotics, SSRIs, TCAs, or vasopressors.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that methylphenidate is generally safe for children with sickle cell disease. One study found that children taking this medication demonstrated improved attention and thinking skills. However, like any medicine, it can have side effects. Some reports mention concerns about heart-related issues, such as changes in heart rhythm. While these are rare, they are important to know about. The FDA has already approved methylphenidate for treating attention deficit hyperactivity disorder (ADHD), indicating its safety is well-studied. This trial aims to provide more information about its safety for children with sickle cell disease over a four-week period. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for sickle cell disease?

Researchers are excited about using methylphenidate for treating sickle cell disease (SCD) because it targets executive functioning deficits, which are not typically addressed by current SCD treatments. Unlike the standard of care, which mainly focuses on managing pain and preventing complications through blood transfusions and hydroxyurea, methylphenidate is a stimulant commonly used for ADHD that has the potential to enhance cognitive functions. This unique approach could improve the quality of life for children and adolescents with SCD by addressing the cognitive challenges associated with the disease.

What evidence suggests that methylphenidate might be an effective treatment for sickle cell disease?

Research shows that methylphenidate (MPH) can improve focus and thinking skills in children with sickle cell disease (SCD). Studies have found that children and teens taking MPH had better attention compared to those on lower doses or a placebo. This suggests MPH might help with attention and thinking issues linked to SCD. While MPH is often used for attention deficit hyperactivity disorder (ADHD), researchers are studying its potential benefits for children with SCD due to these encouraging results. Monitoring for side effects remains important, as with any medication. In this trial, all participants will receive extended-release methylphenidate to evaluate its feasibility, acceptability, adherence, and safety in children and adolescents with SCD and executive functioning deficits. ^{1 2 3 4 6}

Who Is on the Research Team?

Andrew Heitzer, PhD

Principal Investigator

St. Jude Children's Research Hospital

Are You a Good Fit for This Trial?

This trial is for children with Sickle Cell Disease who may have attention or executive function issues, similar to ADHD. They should be able to take a daily dose of Methylphenidate for 4 weeks. The study will also consider the caregiver's perspective on medication use.

Inclusion Criteria

English as the primary language

Enrolled on the institutional protocol: Sickle Cell Clinical Research Intervention Program (SCCRIP)

Score 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)

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

Severe sensory loss

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive extended-release methylphenidate daily for 4 weeks to address attention and executive deficits

4 weeks

1 initial in-clinic visit, followed by weekly remote assessments

Follow-up

Participants are monitored for safety and effectiveness after treatment

4-6 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Methylphenidate

Trial Overview The trial tests if Extended-Release Methylphenidate (a drug used for ADHD) can help with cognitive and attention problems in kids with Sickle Cell Disease. It checks how well they stick to taking it daily and any changes in their thinking or schoolwork.

How Is the Trial Designed?

Group I: Methylphenidate Treatment Group Experimental Treatment 1 Intervention

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.

Who Is Running the Clinical Trial?

St. Jude Children's Research Hospital

Lead Sponsor

Trials

451

Recruited

5,326,000+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC3319853/

Ameliorating Attention Problems in Children with Sickle Cell ... This pilot study examined whether methylphenidate (MPH) was effective in enhancing cognitive performance and attention for children with sickle cell disease ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT07226219

Methylphenidate to Address Attention and Executive ... The purpose of this study is to determine if patients with sickle cell disease (SCD) can consistently take a drug called Methylphenidate ...

3. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/22343483/

Ameliorating attention problems in children with sickle cell ... Attention, as rated by parent and teachers, was improved for a greater number of children and adolescents on higher doses of MPH relative to LD MPH and placebo.

4. frontiersin.org frontiersin.org/journals/neurology/articles/10.3389/fneur.2022.867437/full

Sluggish Cognitive Tempo in Pediatric Sickle Cell Disease The K-SCT is a reliable and valid measure of SCT in SCD. SCT symptoms are associated with learning difficulties even after controlling for ADHD symptoms.

5. jamanetwork.com jamanetwork.com/journals/jama/fullarticle/1104778

ADHD Medications and Risk of Serious Cardiovascular ... In a summary from the FDA Adverse Event Reporting System, cardiac arrest, MI, and sudden unexplained death were among the top 50 adverse events ...

6. researchgate.net researchgate.net/publication/232698857_The_Use_of_Stimulant_Medication_to_Treat_Neurocognitive_Deficits_in_Patients_with_Pediatric_Cancer_Traumatic_Brain_Injury_and_Sickle_Cell_Disease_A_Review

The Use of Stimulant Medication to Treat Neurocognitive ... (PDF) The Use of Stimulant Medication to Treat Neurocognitive Deficits in Patients with Pediatric Cancer, Traumatic Brain Injury, and Sickle ...