Guanfacine for Hyperactivity in Children With Down Syndrome (HYPEbeGONE_DS)

Phase 2

Active, not recruiting

• Conditions: Hyperactivity in Children With Down Syndrome; Impulsivity in Children With Down Syndrome
• Interventions: Drug: Guanfacine Hydrochloride Immediate Release; Drug: Placebo

• Registration Number: NCT06042257
• Lead Sponsor: 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.

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 Timeline

• Study First Submitted: 2023-07-31
• Study First Submitted QC: 2023-09-11
• Study First Posted: 2023-09-18
• Study Start: 2024-12-09
• Status Verified: 2025-08
• Last Update Submitted: 2025-08-26
• Last Update Posted: 2025-09-02
• Primary Completion: 2026-07
• Study Completion: 2026-07-01

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 60

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Guanfacine Hydrochloride Immediate Release	Guanfacine Hydrochloride Immediate Release	Eligible participants will receive GIR for up to 8 weeks. The treatment period will consist of study product administration from day 0 through day 56 with a masked dose-escalation period from day 0 through day 49.
Placebo	Placebo	Eligible participants will receive Placebo for up to 8 weeks.The treatment period will consist of study product administration from day 0 through day 56 with a masked dose-escalation period from day 0 through day 49.

Primary Outcome Measures

Name	Time	Method
Change in parent-rated ABC-H (Aberrant Behavior Checklist-Hyperactivity) subscale core	Baseline to Week 8	Change from baseline to Week 8 of the ABC-H subscale score. The ABC-H is a subscale of the ABC. Each of the 16 items is scored as 0 (never a problem), 1 (slight problem), 2 (moderately serious problem), or 3 (severe problem). The total score range is 0 to 48, where a higher score indicates endorsement of greater hyperactivity. Rating based on patient's behavior in last 4 weeks.

Secondary Outcome Measures

Name	Time	Method
Change in parent-rated ABC-H (Aberrant Behavior Checklist-Hyperactivity) subscale core	Baseline to Week 4	Change from baseline to Week 4 of the ABC-H subscale score. The ABC-H is a subscale of the ABC. Each of the 16 items is scored as 0 (never a problem), 1 (slight problem), 2 (moderately serious problem), or 3 (severe problem). The total score range is 0 to 48, where a higher score indicates endorsement of greater hyperactivity. Rating based on patient's behavior in last 4 weeks.
Proportion of participants with a CGI-I (Clinical Global Impression-Improvement) score of 2 or better at Week 4	Baseline to Week 4	CGI-I specific to hyperactivity, inattention and impulsivity behaviors. The CGI-I is a seven-point scale that requires the clinician to assess how much the patient's illness has improved or worsened relative to a baseline state at the beginning of the intervention. 1 = Very much improved, 2 = Much improved, 3 = Minimally improved, 4 = No change, 5 = Minimally worse, 6 = Much worse, 7 = Very much worse.
Safety of GIR (guanfacine immediate release)	Baseline through Week 8	Number of participants with adverse events (AEs), serious adverse events (SAEs), or events of special interest (ESIs).
Proportion of participants with a CGI-I (Clinical Global Impression-Improvement) score of 2 or better at Week 8	Baseline to Week 8	CGI-I specific to hyperactivity, inattention and impulsivity behaviors. The CGI-I is a seven-point scale that requires the clinician to assess how much the patient's illness has improved or worsened relative to a baseline state at the beginning of the intervention. 1 = Very much improved, 2 = Much improved, 3 = Minimally improved, 4 = No change, 5 = Minimally worse, 6 = Much worse, 7 = Very much worse.

Trial Locations

Locations (15)

Phoenix Childrens Hospital; 🇺🇸 Phoenix, Arizona, United States

Yale University School of Medicine; 🇺🇸 New Haven, Connecticut, United States

Emory University; 🇺🇸 Atlanta, Georgia, United States

Ann and Robert H. Lurie Hospital of Chicago; 🇺🇸 Chicago, Illinois, United States

University of Iowa; 🇺🇸 Iowa City, Iowa, United States

Kennedy Krieger Institute; 🇺🇸 Baltimore, Maryland, United States

Boston Children's Hospital; 🇺🇸 Boston, Massachusetts, United States

Massachusetts General Hospital; 🇺🇸 Lexington, Massachusetts, United States

Atrium Health-Wake Forest School of Medicine; 🇺🇸 Charlotte, North Carolina, United States

Duke University Hospital; 🇺🇸 Durham, North Carolina, United States

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