Methylphenidate for Intellectual Disability and ADHD

Recruiting in Palo Alto (17 mi)

+2 other locations

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Waitlist Available

Sponsor: University of California, Davis

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.

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

My mental age is 4 years or older according to the Stanford Binet test.

You have been diagnosed with intellectual disability.

I can complete the required cognitive tests.

+2 more

Exclusion Criteria

You have had a bad reaction to stimulant medications in the past.

You have taken stimulant medications in the two weeks before the study starts.

Someone who lives with you has a current problem with drugs or alcohol.

+1 more

Participant Groups

The study tests if extended-release liquid methylphenidate (a medication commonly used for ADHD) affects cognition in participants with ID and ADHD. It's a double-blind trial where half the kids get the drug and half get a placebo without knowing which one they're taking. The dose starts low and can increase unless side effects appear.

2Treatment groups

Active Control

Placebo Group

Group I: MethylphenidateActive Control1 Intervention

Group II: PlaceboPlacebo Group1 Intervention