Guanfacine for Hyperactivity in Children With Down Syndrome (HYPEbeGONE_DS)
- Conditions
- Hyperactivity in Children With Down SyndromeImpulsivity in Children With Down Syndrome
- Interventions
- 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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 60
Not provided
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
Akron Children's Hospital
🇺🇸Akron, Ohio, United States
Cincinnati Children's Hospital
🇺🇸Cincinnati, Ohio, United States
Virginia Center for Children
🇺🇸Richmond, Virginia, United States
University of Washington
🇺🇸Seattle, Washington, United States
University of Wisconsin Madison
🇺🇸Madison, Wisconsin, United States