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| ID | Type | Description | Link |
|---|---|---|---|
| 2012-005597-55 | EudraCT Number |
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This multi-center, randomized, double-blind, parallel group, placebo-controlled, proof of concept study will investigate the efficacy and safety of RG7314 in adult participants with ASD. In Stage I of the study, participants will be randomized in 2:1 to receive daily oral doses of 1.5 milligrams (mg) RG7314 or placebo for 12 weeks. After an independent safety review, the study may proceed to Stage II. In Stage II of the study, additional participants will be randomized in 2:1 to receive daily oral doses of 4 mg RG7314 or placebo for 12 weeks. After an independent safety review, Stage III will be started wherein additional participants will be randomized in 2:1 to receive daily oral doses of 10 mg RG7314 or placebo for 12 weeks. During Stage III, safety will be reviewed by independent safety review twice and if no safety signal is observed, then additional participants will be randomized in 1:1:1 either to receive 1.5 milligrams per day (mg/day) or 10 mg/day RG7314 orally or placebo for 12 weeks in Stage IV.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo | Placebo Comparator | Participants will receive placebo matching to RG7314 in each stage (Stage I, II, III and IV) for 12 weeks. |
|
| RG7314 | Experimental | Participants will receive RG7314 orally at a dose of 1.5 mg/day in Stage I, 4 mg/day in Stage II, 10 mg/day in Stage III and 1.5 mg/day or 10 mg/day in Stage IV for 12 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Placebo | Drug | Placebo will be administered in different stages of the study for 12 weeks. |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Social Communication Deficits as Measured by Social Responsiveness Scale 2 (SRS-2) | Baseline, Week 12 | |
| Percentage of Participants with Suicidality, as Measured by the Columbia-Suicide Severity Rating Scale (C-SSRS) | Baseline up to Week 12 | |
| Percentage of Participants With Adverse Events | From Baseline up to Week 18 |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Behavior/Symptoms as Measured by Aberrant Behavior Checklist (ABC) | Baseline, Week 12 | |
| Change From Baseline in Behavior/Symptoms as Measured by Repetitive Behavior Scale-Revised (RBS-R) | Baseline, Week 12 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Clinical Trials | Hoffmann-La Roche | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Southwest Autism Research & Resource Center | Phoenix | Arizona | 85006 | United States | ||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37811711 | Derived | Iffland M, Livingstone N, Jorgensen M, Hazell P, Gillies D. Pharmacological intervention for irritability, aggression, and self-injury in autism spectrum disorder (ASD). Cochrane Database Syst Rev. 2023 Oct 9;10(10):CD011769. doi: 10.1002/14651858.CD011769.pub2. |
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| RG7314 |
| Drug |
RG7314 will be administered orally at a dose of 1.5 mg/day in Stage I, 4 mg/day in Stage II, 10 mg/day in Stage III and 1.5 mg/day or 10 mg/day in Stage IV for 12 weeks. |
|
| Change From Baseline in Behavior/Symptoms as Measured by Anxiety, Depression and Mood Scale (ADAMS) | Baseline, Week 12 |
| Measure: Change From Baseline in Behavior/Symptoms as Measured by State-Trait Anxiety Inventory (STAI) | Baseline, Week 12 |
| Change From Baseline in Clinical Global Impressions as Measured by Clinical Global Impressions Improvement (CGI-I) Scale | Baseline, Week 12 |
| Change From Baseline in Adaptive Functioning and Skills as Measured by Vineland-II Adaptive Behavior Scale (VABS) | Baseline, Week 12 |
| Plasma Concentration of RG7314 | Pre-dose (0 hours [hr]), and 2, 4, 6 hrs post-dose on Day 1, 14, 84; pre-dose (0 hr) on Day 42; any time during early withdrawal visit (up to Week 12) |
| Plasma Concentration of RG7314 Metabolite M3 | Pre-dose (0 hr), and 2, 4, 6 hrs post-dose on Day 1, 14, 84; pre-dose (0 hr) on Day 42; any time during early withdrawal visit (up to Week 12) |
| Area Under the Plasma Concentration Versus Time Curve From Time Zero to Last Measurable Concentration (AUClast) of RG7314 | Pre-dose (0 hr), and 2, 4, 6 hrs post-dose on Day 1, 14, 84; pre-dose (0 hr) on Day 42; any time during early withdrawal visit (up to Week 12) |
| AUClast of RG7314 Metabolite M3 | Pre-dose (0 hr), and 2, 4, 6 hrs post-dose on Day 1, 14, 84; pre-dose (0 hr) on Day 42; any time during early withdrawal visit (up to Week 12) |
| Maximum Observed Plasma Concentration (Cmax) of RG7314 | Pre-dose (0 hr), and 2, 4, 6 hrs post-dose on Day 1, 14, 84; pre-dose (0 hr) on Day 42; any time during early withdrawal visit (up to Week 12) |
| Cmax of RG7314 Metabolite M3 | Pre-dose (0 hr), and 2, 4, 6 hrs post-dose on Day 1, 14, 84; pre-dose (0 hr) on Day 42; any time during early withdrawal visit (up to Week 12) |
| Percentage of Participants With Clinical Response, Defined as At Least 25% Improvement in the SRS-2 Score With a CGI-I Score of 1 or 2 | From Baseline to Week 12 |
| Phoenix |
| Arizona |
| 85006 |
| United States |
| University of California Davis | Sacramento | California | 95817 | United States |
| Sacramento | California | 95817 | United States |
| PCSD Feighner Research | San Diego | California | 92108 | United States |
| San Diego | California | 92108 | United States |
| University of California at San Francisco | San Francisco | California | 94115 | United States |
| San Francisco | California | 94115 | United States |
| Santa Ana | California | 92705 | United States |
| Stanford | California | 94305 | United States |
| DBA IMMUNOe Int'l Res Center | Centennial | Colorado | 80112 | United States |
| Centennial | Colorado | 80112 | United States |
| Yale University; Yale Child Study Center | New Haven | Connecticut | 06510 | United States |
| New Haven | Connecticut | 06510 | United States |
| Neurology offices of south florida | Delray Beach | Florida | 33445 | United States |
| Delray Beach | Florida | 33445 | United States |
| Research Centers of America, LLC | Oakland Park | Florida | 33334 | United States |
| Oakland Park | Florida | 33334 | United States |
| Compass Research | Orlando | Florida | 32806 | United States |
| Orlando | Florida | 32806 | United States |
| Emory University School of Medicine; Department of Human Genetics & Pediatrics | Decatur | Georgia | 30033 | United States |
| Decatur | Georgia | 30033 | United States |
| Rush University Medical Center | Chicago | Illinois | 60612 | United States |
| Chicago | Illinois | 60612 | United States |
| The Kennedy Krieger Institute | Baltimore | Maryland | 21205 | United States |
| Baltimore | Maryland | 21205 | United States |
| Neuroscientific Insights | Rockville | Maryland | 20852 | United States |
| Rockville | Maryland | 20852 | United States |
| Massachusetts General Hospital | Lexington | Massachusetts | 02421 | United States |
| Lexington | Massachusetts | 02421 | United States |
| University of Minnesota; Clin. Neuro Research Unit | Minneapolis | Minnesota | 55414 | United States |
| Minneapolis | Minnesota | 55414 | United States |
| Mount Sinai School of Medicine; Seaver Autism Center | New York | New York | 10029 | United States |
| New York | New York | 10029 | United States |
| Nathan S. Kline Institute for Psychiatric Research | Orangeburg | New York | 10962 | United States |
| Orangeburg | New York | 10962 | United States |
| Montefiore Medical Center; Departments of Psychiatry & Biobehavioral Science | The Bronx | New York | 10467 | United States |
| The Bronx | New York | 10467 | United States |
| DUKE SCHOOL OF MEDICINE;Duke Center for Autism and Brain Development | Durham | North Carolina | 27705 | United States |
| Durham | North Carolina | 27705 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| Cincinnati | Ohio | 45229 | United States |
| University Hospitals | Cleveland | Ohio | 44106 | United States |
| Cleveland | Ohio | 44106 | United States |
| UPMC Western Psychiatric Institute and Clinic | Pittsburgh | Pennsylvania | 15203 1101 | United States |
| Pittsburgh | Pennsylvania | 15203 1101 | United States |
| Vanderbilt Medical Center | Nashville | Tennessee | 37232 | United States |
| Nashville | Tennessee | 37232 | United States |
| Road Runner Research | San Antonio | Texas | 78258 | United States |
| San Antonio | Texas | 78258 | United States |
| Northwest Clinical Research Center | Bellevue | Washington | 98007 | United States |
| Bellevue | Washington | 98007 | United States |
| Pacific Institute of Medical Sciences | Bothell | Washington | 98011 | United States |
| Bothell | Washington | 98011 | United States |
| Seattle Children's Research Institute | Seattle | Washington | 98145 | United States |
| Seattle | Washington | 98145 | United States |
| ID | Term |
|---|---|
| D000067877 | Autism Spectrum Disorder |
| ID | Term |
|---|---|
| D002659 | Child Development Disorders, Pervasive |
| D065886 | Neurodevelopmental Disorders |
| D001523 | Mental Disorders |
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