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Using guanfacine to treat hyperactivity in children with Down syndrome

Guanfacine for Hyperactivity in Children With Down Syndrome (HYPEbeGONE_DS)

Phase 2 Interventional Duke University · NCT06042257

This study is testing if a medication called guanfacine can help children with Down syndrome, aged 6-12, who struggle with hyperactivity, impulsivity, and inattention.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment60 (estimated)
Ages6 Years to 12 Years
SexAll
SponsorDuke University Academic / other
Locations15 sites (Phoenix, Arizona and 14 other locations)
Trial IDNCT06042257 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the effectiveness of guanfacine immediate release (GIR) in treating hyperactivity, impulsivity, and inattention in children aged 6-12 with Down syndrome. The study is designed as a randomized, double-blind, placebo-controlled trial, where participants will be assigned to receive either GIR or a placebo over an 8-week period. Participants will undergo a screening phase, followed by multiple in-person visits for assessments and dose adjustments based on their response. Blood samples will be collected for pharmacokinetic analysis, and caregivers will maintain a daily diary to track the child's symptoms.

Who should consider this trial

Good fit: Ideal candidates are children aged 6-12 with a clinical diagnosis of non-mosaic Down syndrome and significant symptoms of hyperactivity, impulsivity, and inattention.

Not a fit: Patients who do not have Down syndrome or those with mild symptoms of hyperactivity may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve attention and reduce hyperactive behaviors in children with Down syndrome.

How similar studies have performed: Other studies have explored treatments for hyperactivity in children with Down syndrome, but this specific approach using guanfacine is relatively novel.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion:

1. Parent/Legal Guardian can understand the consent process and is willing to provide informed consent/HIPAA authorization prior to the conduct of any study-related procedures. When applicable, the minor participant is willing to provide assent.
2. Participant has clinical diagnosis of non-mosaic DS.
3. Participant is between 6 and 12 years of age (inclusive) at time of consent.
4. Participant weight is ≥ 25 kg.
5. Participant has clinically significant symptoms of hyperactivity, inattention and impulsivity manifested as minimum scores of the following rating scales within 30 days of randomization:

   1. A minimum score of 18 on the parent-reported ABC-H subscale, AND
   2. A minimum score of moderate or greater (≥ 4) on the clinician reported Clinical Global Impression Severity (CGI-S) score specific to hyperactivity, inattention and impulsivity behaviors.
6. Participant has co-morbid medical screening and clearance to proceed with a non-stimulant medication trial with GIR within 30 days of randomization.
7. Participant is willing and able to comply with study procedures, including adherence to medication dosing schedule.

Exclusion:

1. Participant has received guanfacine (any formulation) within 30 days of randomization.
2. Participant has received any of the following concomitant medication classes within 30 days of randomization:

   1. Strong CYP3A4 inhibitors (e.g., boceprevir, clarithromycin, conivaptan, grapefruit juice, indinavir, itraconazole, ketoconazole, lopinavir/ritonavir, mibefradil, nefazodone, nelfinavir, posaconazole, ritonavir, saquinavir, telaprevir, telithromycin, and voriconazole)
   2. Strong CYP3A4 inducers (e.g., avasimibe, carbamazepine, phenytoin, rifampin, and St. John's wort)
3. Participant has a psychiatric comorbidity, such as major depressive disorder, bipolar disorder, obsessive-compulsive disorder, or a psychotic disorder, that requires a pharmacological treatment other than guanfacine
4. For participants ≥ 8 years old at the time of consent, participant has a history of suicidality or positive screen on Ask Suicide-Screening Questions (asQ) Tool.
5. Participant is currently in or plans to participate in another interventional study.
6. Participant has a known hypersensitivity to guanfacine.
7. Participant has had a previous guanfacine treatment failure, as determined by their primary treating physician.
8. Participant has had a change in another medication intended to treat symptoms of hyperactivity, inattention, and impulsivity within the last 2 weeks.
9. Participant has had a seizure within the last 6 months.
10. Participant has had a change in their anti-convulsant dose within the last 4 weeks.
11. Participant has a cardiac-related condition including:

    1. Significant symptomatic bradycardia;
    2. 2nd degree or 3rd degree (complete) heart block;
    3. Baseline heart rate (HR) or systolic blood pressure (BP) \> 2 standard deviations (SD) below mean for age as determined by medical examination;
    4. History of aborted sudden cardiac death, unexplained syncope or near syncope, or historical use of a pacemaker as determined by medical history will require clearance by cardiology prior to enrollment;
    5. Known history of congenital heart disease which requires ongoing care for monitoring or management will require clearance by cardiology prior to enrollment.
12. Participant has a history of untreated severe obstructive sleep apnea defined as obstructive apnea hypopnea index (OAHI) ≥ 10 events per hour or aortic regurgitation (AR). Participants with an OAHI index \> 10/hr are eligible if managed with continuous positive airway pressure (CPAP).
13. Participant has untreated thyroid disease.
14. Participant has a known hepatic impairment defined as aspartate aminotransferase (AST) or alanine aminotransferase (ALT) \> 2x the upper limit of normal (ULN) for age.
15. Participant has known impending or renal failure defined as:

    1. Anuria diagnosed within 12 hours prior to enrollment;
    2. Requiring renal replacement therapy.
16. Participant is pregnant.
17. Participant has any condition which would make the participant, in the opinion of the investigator, unsuitable for the study.

Where this trial is running

Phoenix, Arizona and 14 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Hyperactivity in Children With Down SyndromeImpulsivity in Children With Down Syndromehyperactivityimpulsivityinattention
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.