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NCT06042257 | RECRUITING | Hyperactivity in Children with Down Syndrome

Guanfacine for Hyperactivity in Children with Down Syndrome (HYPEbeGONE_DS)
Sponsor:

Rachel G. Greenberg, MD, MB, MHS

Information provided by (Responsible Party):

Rachel G. Greenberg, MD, MB, MHS

Brief Summary:

The purpose of this study is to determine efficacy of guanfacine immediate release (GIR) for the treatment of hyperactivity/impulsivity and inattention in children 6-12 years of age with Down syndrome (DS) after 8 weeks of treatment.

Condition or disease

Hyperactivity in Children with Down Syndrome

Impulsivity in Children with Down Syndrome

Intervention/treatment

Guanfacine Hydrochloride Immediate Release

Placebo

Phase

PHASE2

Detailed Description:

This is a randomized, double-blind, placebo-controlled flexibly dosed trial of guanfacine immediate release (GIR) in children with Down syndrome (DS) and symptoms of hyperactivity, inattention, and impulsivity. Participants will undergo a screening period of up to 29 days. Eligible participants meeting study criteria will be randomized 2:1 GIR or placebo. There are a total of up to 4 in person visits (screening, baseline, at Week 4, and at Week 8). Participants will receive GIR or placebo for up to 8 weeks. Weekly dose escalation will be determined via a telephone assessment at Weeks 1-3 and Weeks 4-7. Unmasking of participant and site staff will occur at the week 8, in-person visit. After unmasking, participants who were randomized to receive GIR will be given the option to 1) remain on GIR and to transition to open-label GIR per standard of care or 2) taper off of GIR. A Telephone Safety Assessment will be conducted for all participants, at 5 (+2) days after final study product administration. Blood specimens will be collected at the Week 4 and Week 8 visits for Pharmacokinetic (PK) analyses and lab assessments. Participants will be asked to keep a daily study diary and will complete study measures at screening/baseline, Week 4 and Week 8. Parents/Caregivers will need to complete the Study Diary during the bridge/taper period for those who are in the GIR arm.

Study Type : INTERVENTIONAL
Estimated Enrollment : 60 participants
Masking : TRIPLE
Masking Description : A masked investigational pharmacist or designee will dispense study product according to randomization treatment assignments. All other site staff as well as participants and parents/legal guardians will also be masked for up to 8 weeks while the study participant is receiving study product. Participants, parents/legal guardians, site staff, and study administrators will be unmasked at the 8 week study visit. Emergency unmasking may occur at any time throughout the study in the event that knowledge of the actual treatment is absolutely essential for further management of the participant.
Primary Purpose : TREATMENT
Official Title : Guanfacine for Hyperactivity in Children with Down Syndrome (HYPEbeGONE_DS)
Actual Study Start Date : 2024-12
Estimated Primary Completion Date : 2025-11
Estimated Study Completion Date : 2026-02

Information not available for Arms and Intervention/treatment

Ages Eligible for Study: 6 Years to 12 Years
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers: 1
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.
Guanfacine for Hyperactivity in Children with Down Syndrome (HYPEbeGONE_DS)

Location Details

NCT06042257

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How to Participate

Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.

Locations

NOT YET RECRUITING

United States, Arizona

Phoenix Childrens Hospital

Phoenix, arizona, United States, 85016

NOT YET RECRUITING

United States, Connecticut

Yale University School of Medicine

New Haven, Connecticut, United States, 06250

NOT YET RECRUITING

United States, Georgia

Emory University

Atlanta, Georgia, United States, 30322

NOT YET RECRUITING

United States, Illinois

Ann and Robert H. Lurie Hospital of Chicago

Chicago, Illinois, United States, 60611

NOT YET RECRUITING

United States, Iowa

University of Iowa

Iowa City, Iowa, United States, 52242

RECRUITING

United States, Maryland

Kennedy Krieger Institute

Baltimore, Maryland, United States, 21205

NOT YET RECRUITING

United States, Massachusetts

Boston Children's Hospital

Boston, Massachusetts, United States, 02115

NOT YET RECRUITING

United States, Massachusetts

Massachusetts General Hospital

Lexington, Massachusetts, United States, 02421

RECRUITING

United States, North Carolina

Atrium Health-Wake Forest School of Medicine

Charlotte, North Carolina, United States, 28204

NOT YET RECRUITING

United States, North Carolina

Duke University Hospital

Durham, North Carolina, United States, 27705

NOT YET RECRUITING

United States, Ohio

Akron Children's Hospital

Akron, Ohio, United States, 44308

RECRUITING

United States, Ohio

Cincinnati Children's Hospital

Cincinnati, Ohio, United States, 45229

NOT YET RECRUITING

United States, Virginia

Virginia Center for Children

Richmond, Virginia, United States, 23220

NOT YET RECRUITING

United States, Washington

University of Washington

Seattle, Washington, United States, 98195

NOT YET RECRUITING

United States, Wisconsin

University of Wisconsin Madison

Madison, Wisconsin, United States, 53792