Methylphenidate for Intellectual Disability and ADHD
Trial Summary
What is the purpose of this trial?
This study is a randomized, double-blind, placebo-controlled, crossover trial of extended-release liquid methylphenidate (XRMPH) to evaluate the sensitivity of the NIH Toolbox Cognition Battery (NIHTB-CB) to changes in cognition in children and adolescents ages 6 to 17 with intellectual disability (D) and comorbid Attention Deficit Hyperactivity Disorder (ADHD). The sample will include 68 males or females (expected male: female ratio of 1.8:1 with ID and ADHD as determined by structured diagnostic interview and Conners 3 scores. Additional inclusion criteria will include Full Scale IQ above 50 and mental age greater than or equal to 3 years. In addition, participants must be able to complete NIHTB-CB testing and provide valid scores at baseline. After baseline testing, participants will then be randomized to drug or placebo in a 1:1 ratio (N=34 per group) at the end of the baseline visit. XRMPH in oral suspension supplied as Quillivant XR in 5 mg/ml (Tris Pharma, Monmouth Junction, NJ) will be the active treatment. The XRMPH or matching placebo will be started at a dose of 0.3 mg/kg/day and individually titrated over two weeks. Phone calls at the end of weeks 1, 2, and 3 will be used to collect adverse event and response data. If there is no evidence of side effects and ongoing symptoms of ADHD, the dose will be increased to 0.5 mg/kg/day at one week and 0.7 mg/kg/day at 2 weeks (maximum dose of 60 mg per day consistent with FDA labeled use in youth). The Clinical Global Impression (CGI) will be used as a guide to define optimal dose. If side effects occur the dose will be reduced to the dose level at which there were no side effects. Final optimal dose will be established by the end of week 3 and this will be maintained for 2 weeks until 5 weeks post randomization, at which time the follow-up parent and teacher Conners scales, NIHTB-CB, Go/No-Go, and PedsQL will be completed. Participants will have a washout period of 1 week, will then complete re-assessment at the second baseline, and then will cross over to the other treatment (Quillivant to placebo; placebo to Quillivant), also in a double-blind fashion. In the second treatment arm, patients will have the same titration, monitoring and treatment periods as in the first arm, again followed by repeated assessments at the conclusion of 5 weeks. The accrual of participants and number of visits is shown in the Timeline per 6-month period.
Will I have to stop taking my current medications?
The trial requires that you stop taking any stimulant medications at least 2 weeks before joining. If you're on other types of medications, the protocol doesn't specify, so it's best to discuss with the trial team.
What data supports the effectiveness of the drug Methylphenidate Oral Solution for treating Intellectual Disability and ADHD?
Research shows that methylphenidate is effective in reducing ADHD symptoms in children, adolescents, and adults. It has been studied in various forms, including controlled-delivery and osmotic-release systems, and has shown positive results in managing ADHD symptoms, which may suggest potential benefits for those with intellectual disabilities as well.12345
Is methylphenidate safe for humans?
How does the drug Methylphenidate Oral Solution differ from other treatments for ADHD and intellectual disability?
Methylphenidate Oral Solution is unique because it is an oral liquid form, which can be easier to administer for individuals who have difficulty swallowing pills. This form of methylphenidate is designed to help manage symptoms of ADHD by increasing levels of certain chemicals in the brain, similar to other forms of methylphenidate, but its liquid form offers a different administration option.311121314
Eligibility Criteria
This trial is for children and adolescents aged 6-17 with intellectual disabilities (ID) like Down Syndrome or Fragile X, who also have ADHD. They must have an IQ above 50 but below 80, a mental age of at least 4 years, and be able to complete certain cognitive tests. Those recently on stimulants or with conditions like uncontrolled epilepsy or severe psychiatric disorders cannot participate.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Baseline
Baseline testing and randomization to drug or placebo
Treatment
Participants receive XRMPH or placebo with dose titration over 3 weeks, followed by 2 weeks at optimal dose
Washout
Participants undergo a washout period before crossover
Crossover Treatment
Participants switch to the alternate treatment with the same titration and monitoring as the first arm
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- Methylphenidate Oral Solution
Find a Clinic Near You
Who Is Running the Clinical Trial?
University of California, Davis
Lead Sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Collaborator