Ignite Creation Date:
2025-12-24 @ 6:43 PM
Ignite Modification Date:
2025-12-25 @ 4:14 PM
Study NCT ID:
NCT06042257
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-09-02
First Post:
2023-07-31
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Guanfacine for Hyperactivity in Children With Down Syndrome (HYPEbeGONE_DS)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D013035', 'term': 'Spasm'}, {'id': 'D007175', 'term': 'Impulsive Behavior'}], 'ancestors': [{'id': 'D020879', 'term': 'Neuromuscular Manifestations'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}, {'id': 'D001519', 'term': 'Behavior'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D016316', 'term': 'Guanfacine'}], 'ancestors': [{'id': 'D006146', 'term': 'Guanidines'}, {'id': 'D000578', 'term': 'Amidines'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D010648', 'term': 'Phenylacetates'}, {'id': 'D000146', 'term': 'Acids, Carbocyclic'}, {'id': 'D002264', 'term': 'Carboxylic Acids'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'TRIPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR'], 'maskingDescription': '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.\n\nParticipants, parents/legal guardians, site staff, and study administrators will be unmasked at the 8 week study visit.\n\nEmergency 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.'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'Participants will be randomized 2:1 to GIR or placebo.'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 60}}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2024-12-09', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-08', 'completionDateStruct': {'date': '2026-07', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-08-26', 'studyFirstSubmitDate': '2023-07-31', 'studyFirstSubmitQcDate': '2023-09-11', 'lastUpdatePostDateStruct': {'date': '2025-09-02', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2023-09-18', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2026-07', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change in parent-rated ABC-H (Aberrant Behavior Checklist-Hyperactivity) subscale core', 'timeFrame': 'Baseline to Week 8', 'description': "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."}], 'secondaryOutcomes': [{'measure': 'Change in parent-rated ABC-H (Aberrant Behavior Checklist-Hyperactivity) subscale core', 'timeFrame': 'Baseline to Week 4', 'description': "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."}, {'measure': 'Proportion of participants with a CGI-I (Clinical Global Impression-Improvement) score of 2 or better at Week 4', 'timeFrame': 'Baseline to Week 4', 'description': "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."}, {'measure': 'Proportion of participants with a CGI-I (Clinical Global Impression-Improvement) score of 2 or better at Week 8', 'timeFrame': 'Baseline to Week 8', 'description': "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."}, {'measure': 'Safety of GIR (guanfacine immediate release)', 'timeFrame': 'Baseline through Week 8', 'description': 'Number of participants with adverse events (AEs), serious adverse events (SAEs), or events of special interest (ESIs).'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['hyperactivity', 'impulsivity', 'inattention'], 'conditions': ['Hyperactivity in Children With Down Syndrome', 'Impulsivity in Children With Down Syndrome']}, 'descriptionModule': {'briefSummary': '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.', 'detailedDescription': '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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '12 Years', 'minimumAge': '6 Years', 'healthyVolunteers': True, 'eligibilityCriteria': "Inclusion:\n\n1. 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.\n2. Participant has clinical diagnosis of non-mosaic DS.\n3. Participant is between 6 and 12 years of age (inclusive) at time of consent.\n4. Participant weight is ≥ 25 kg.\n5. Participant has clinically significant symptoms of hyperactivity, inattention and impulsivity manifested as minimum scores of the following rating scales within 30 days of randomization:\n\n 1. A minimum score of 18 on the parent-reported ABC-H subscale, AND\n 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.\n6. Participant has co-morbid medical screening and clearance to proceed with a non-stimulant medication trial with GIR within 30 days of randomization.\n7. Participant is willing and able to comply with study procedures, including adherence to medication dosing schedule.\n\nExclusion:\n\n1. Participant has received guanfacine (any formulation) within 30 days of randomization.\n2. Participant has received any of the following concomitant medication classes within 30 days of randomization:\n\n 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)\n 2. Strong CYP3A4 inducers (e.g., avasimibe, carbamazepine, phenytoin, rifampin, and St. John's wort)\n3. 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\n4. 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.\n5. Participant is currently in or plans to participate in another interventional study.\n6. Participant has a known hypersensitivity to guanfacine.\n7. Participant has had a previous guanfacine treatment failure, as determined by their primary treating physician.\n8. Participant has had a change in another medication intended to treat symptoms of hyperactivity, inattention, and impulsivity within the last 2 weeks.\n9. Participant has had a seizure within the last 6 months.\n10. Participant has had a change in their anti-convulsant dose within the last 4 weeks.\n11. Participant has a cardiac-related condition including:\n\n 1. Significant symptomatic bradycardia;\n 2. 2nd degree or 3rd degree (complete) heart block;\n 3. Baseline heart rate (HR) or systolic blood pressure (BP) \\> 2 standard deviations (SD) below mean for age as determined by medical examination;\n 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;\n 5. Known history of congenital heart disease which requires ongoing care for monitoring or management will require clearance by cardiology prior to enrollment.\n12. 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).\n13. Participant has untreated thyroid disease.\n14. Participant has a known hepatic impairment defined as aspartate aminotransferase (AST) or alanine aminotransferase (ALT) \\> 2x the upper limit of normal (ULN) for age.\n15. Participant has known impending or renal failure defined as:\n\n 1. Anuria diagnosed within 12 hours prior to enrollment;\n 2. Requiring renal replacement therapy.\n16. Participant is pregnant.\n17. Participant has any condition which would make the participant, in the opinion of the investigator, unsuitable for the study."}, 'identificationModule': {'nctId': 'NCT06042257', 'acronym': 'HYP01', 'briefTitle': 'Guanfacine for Hyperactivity in Children With Down Syndrome (HYPEbeGONE_DS)', 'organization': {'class': 'OTHER', 'fullName': 'Duke University'}, 'officialTitle': 'Guanfacine for Hyperactivity in Children With Down Syndrome (HYPEbeGONE_DS)', 'orgStudyIdInfo': {'id': 'Pro00111256'}, 'secondaryIdInfos': [{'id': 'HHSN275201800003I', 'type': 'OTHER_GRANT', 'domain': 'National Institute of Child Health and Human Development'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'ACTIVE_COMPARATOR', 'label': 'Guanfacine Hydrochloride Immediate Release', 'description': '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.', 'interventionNames': ['Drug: Guanfacine Hydrochloride Immediate Release']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': '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.', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'Guanfacine Hydrochloride Immediate Release', 'type': 'DRUG', 'description': '0.5 mg capsules', 'armGroupLabels': ['Guanfacine Hydrochloride Immediate Release']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Matching placebo capsule', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '85016', 'city': 'Phoenix', 'state': 'Arizona', 'country': 'United States', 'facility': 'Phoenix Childrens Hospital', 'geoPoint': {'lat': 33.44838, 'lon': -112.07404}}, {'zip': '06250', 'city': 'New Haven', 'state': 'Connecticut', 'country': 'United States', 'facility': 'Yale University School of Medicine', 'geoPoint': {'lat': 41.30815, 'lon': -72.92816}}, {'zip': '30322', 'city': 'Atlanta', 'state': 'Georgia', 'country': 'United States', 'facility': 'Emory University', 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'zip': '60611', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': 'Ann and Robert H. Lurie Hospital of Chicago', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '52242', 'city': 'Iowa City', 'state': 'Iowa', 'country': 'United States', 'facility': 'University of Iowa', 'geoPoint': {'lat': 41.66113, 'lon': -91.53017}}, {'zip': '21205', 'city': 'Baltimore', 'state': 'Maryland', 'country': 'United States', 'facility': 'Kennedy Krieger Institute', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': "Boston Children's Hospital", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '02421', 'city': 'Lexington', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Massachusetts General Hospital', 'geoPoint': {'lat': 42.44732, 'lon': -71.2245}}, {'zip': '28204', 'city': 'Charlotte', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Atrium Health-Wake Forest School of Medicine', 'geoPoint': {'lat': 35.22709, 'lon': -80.84313}}, {'zip': '27705', 'city': 'Durham', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Duke University Hospital', 'geoPoint': {'lat': 35.99403, 'lon': -78.89862}}, {'zip': '44308', 'city': 'Akron', 'state': 'Ohio', 'country': 'United States', 'facility': "Akron Children's Hospital", 'geoPoint': {'lat': 41.08144, 'lon': -81.51901}}, {'zip': '45229', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'facility': "Cincinnati Children's Hospital", 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '23220', 'city': 'Richmond', 'state': 'Virginia', 'country': 'United States', 'facility': 'Virginia Center for Children', 'geoPoint': {'lat': 37.55376, 'lon': -77.46026}}, {'zip': '98195', 'city': 'Seattle', 'state': 'Washington', 'country': 'United States', 'facility': 'University of Washington', 'geoPoint': {'lat': 47.60621, 'lon': -122.33207}}, {'zip': '53792', 'city': 'Madison', 'state': 'Wisconsin', 'country': 'United States', 'facility': 'University of Wisconsin Madison', 'geoPoint': {'lat': 43.07305, 'lon': -89.40123}}], 'overallOfficials': [{'name': 'Rachel Greenberg', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'DCRI'}]}, 'ipdSharingStatementModule': {'url': 'https://dash.nichd.nih.gov', 'infoTypes': ['STUDY_PROTOCOL', 'ICF'], 'timeFrame': 'Data will be uploaded to the repository within 2 years of study completion. It will be maintained in the repository indefinitely.', 'ipdSharing': 'YES', 'description': 'All data collected is uploaded into the National Institute of Health Data Repository (DASH) at the end of the study (de-identified).', 'accessCriteria': 'In order to have access, researchers have to complete a Data access request. NICHD will review the request and either approve or deny it. IRB approval must be obtained by the researcher to access the data.\n\nhttps://dash.nichd.nih.gov/Resource/DataRequestChecklist'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Rachel G. Greenberg, MD, MB, MHS', 'class': 'OTHER'}, 'collaborators': [{'name': 'The Emmes Company, LLC', 'class': 'INDUSTRY'}, {'name': 'Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)', 'class': 'NIH'}], 'responsibleParty': {'type': 'SPONSOR_INVESTIGATOR', 'investigatorTitle': 'Associate Professor of Pediatrics', 'investigatorFullName': 'Rachel G. Greenberg, MD, MB, MHS', 'investigatorAffiliation': 'Duke University'}}}}