ClinicalTrials.gov record NCT05301361
Sensitivity of the NIH Toolbox to Stimulant Treatment in Intellectual Disabilities
Sensitivity of the NIH Toolbox Cognition Battery to Stimulant Treatment in Intellectual Disabilities
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
A randomized, double‑blind, placebo‑controlled crossover trial testing extended‑release liquid methylphenidate (Quillivant XR) in young people with intellectual disability and ADHD. About 68 participants aged 6 to 24 will receive both drug and placebo in random order, with dose titration, monitoring, and assessments after each treatment period. The study uses the NIH Toolbox Cognition Battery and ADHD rating scales to measure change.
What it is studying. The study is trying to learn if the NIH Toolbox Cognition Battery (a set of computer tasks) can detect cognitive changes when people with intellectual disability and ADHD take extended‑release methylphenidate compared with placebo. It also collects ADHD symptom ratings (Conners) and related measures to compare with the Toolbox results.
What the researchers hope to learn. To determine whether the NIH Toolbox Cognition Battery is sensitive to cognitive changes from stimulant treatment in people with intellectual disability and ADHD. To compare Toolbox results with standard ADHD rating scales and provide data on measurement methods for future research.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- Methylphenidate Oral Solution
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- IQ below 80
- Mental age 3.0 or higher (Stanford Binet)
- Diagnosis of Intellectual Disability
- Diagnosis of ADHD
- Ability to complete valid NIHTB-CB tests at screening/baseline
Exclusion criteria
- stimulant use within 2 weeks prior to randomization
- history of a sensitivity reaction to stimulants
- presence of significant comorbid psychiatric or medical disorder/illness deemed by the site physician as inappropriate for stimulant use (uncontrolled epilepsy, bipolar disorder, psychosis, severe OCD, hypertension, tachycardia hypertension, failure to thrive, psychosis for example)
- a household resident with a current substance abuse disorder
Study description and what participation involves
The registry record has no detailed description beyond the summary above.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants will be randomly assigned (double-blind) to receive either an extended‑release methylphenidate oral solution or matching placebo, with individual dose titration over about two weeks and phone check-ins at the end of weeks 1, 2 and 3. They will complete baseline and post-treatment testing including the NIH Toolbox Cognition Battery, parent and teacher Conners ADHD rating scales, a Go/No‑Go task, and the PedsQL quality-of-life measure. After one treatment period (about 5 weeks on the chosen dose) there is a one-week washout, repeat baseline assessments, then crossover to the other treatment with the same titration and testing schedule. No especially invasive procedures are listed in the record.
How the study is structured. This is an interventional, randomized, quadruple‑masked crossover trial. Each participant is randomly assigned to start with either extended‑release methylphenidate (oral suspension) or matching placebo, goes through a 2–3 week dose‑titration and a 5‑week treatment period, then has a 1‑week washout and crosses over to the other treatment with the same titration and treatment period. Study visits include baseline testing and repeat assessments after each treatment period; phone calls are used to monitor side effects and response during titration.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
- The registry says participants are enrolled by invitation. Who decides who is invited, and how?
- The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on fragile X syndrome
Important dates
- Study start
- Feb 1, 2023
- Primary completion
- Sep 29, 2026 (estimated)
- Study completion
- Sep 29, 2026 (estimated)
- Target enrollment
- 68 participants (estimated)
- Last updated on registry
- Dec 15, 2025
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- University of California, Davis
- Collaborators
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Participating sites (3)
- UC Davis MIND Institute
Sacramento, California, 95817 · United States
- Rush University
Chicago, Illinois, 60612 · United States
- Cincinnati Children's Hospital
Cincinnati, Ohio, 45229 · United States
Terms used on this page · explanations by MedicalUpdates.ai
- randomized
- Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
- double-blind
- Two types of people involved in the study do not know which intervention a participant is receiving. The ClinicalTrials.gov record specifies who is masked, for example participants, care providers, investigators or outcome assessors.
- placebo-controlled
- A study design in which one group receives a placebo so that the study treatment's effects can be compared with no active treatment.
- crossover
- Each participant receives more than one intervention, one after the other, usually with a gap (a washout) in between. Participants act as their own comparison.
- inclusion criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- baseline
- Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
- placebo
- A treatment that looks like the study treatment but contains no active ingredient. Some participants receive it instead of the study treatment, usually without knowing which they got.
- 1:1
- How many participants go to each group. In a 2:1 ratio, two are assigned to the first group for every one assigned to the second.
- adverse event
- Any unwanted medical problem that happens during a study, whether or not it was caused by the treatment. A 'serious' adverse event is one that meets specific criteria, such as causing death, being life-threatening, requiring or prolonging hospital admission, causing significant disability, or another medically important outcome.
- follow-up
- The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
- washout period
- A period during which a participant stops a previous treatment before starting the study treatment, so the earlier treatment does not affect the results.
- treatment arm
- One of the groups in a study. Each arm receives a particular intervention, comparator or no intervention, so the groups can be compared.
- at screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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