Not provided
Not provided
Not provided
Not provided
Not provided
A futility analysis assessed that the study is highly unlikely to meet the pre-defined primary objective of the study. No new safety concerns were identified.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
This study will evaluate the efficacy, safety, and pharmacokinetics of 10 mg of oral administration balovaptan once a day (QD) compared with matching placebo in adults (18 years and older) with autism spectrum disorder (ASD).
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Balovaptan | Experimental |
| |
| Placebo | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Balovaptan | Drug | Participants will receive 10 mg of oral administration balovaptan once a day (QD). |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline at Week 24 on the Vineland Adaptive Behavior Scales (Vineland-II) Two-domain Composite (2DC) Score. | Vineland™-II Adaptive Behavior Scales 2-Domain Composite (2DC) Score is defined as mean of the Communication domain standard score & Socialization domain standard score. If any of the 2 individual domain standard scores is missing 2DC score is not computed. Vineland™-II is an instrument that measures communication, daily living skills, socialization, motor skills and maladaptive behavior of individuals with developmental disabilities. Survey Interview Form will be administered to a subject's reliable study partner in this study, during which the rater or clinician will ask to the study partner open ended questions relating to the subject's activities and behavior. Standardized scores on the Adaptive behavior composite range from 20-160 with higher scores indicating better functioning. | Week 24 |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline at Week 12 on the Vineland-II 2DC Score | Vineland™-II Adaptive Behavior Scales 2-Domain Composite (2DC) Score is defined as mean of the Communication domain standard score & Socialization domain standard score. If any of the 2 individual domain standard scores is missing 2DC score is not computed. Vineland™-II is an instrument that measures communication, daily living skills, socialization, motor skills and maladaptive behavior of individuals with developmental disabilities. Survey Interview Form will be administered to a subject's reliable study partner in this study, during which the rater or clinician will ask to the study partner open ended questions relating to the subject's activities and behavior. Standardized scores on the Adaptive behavior composite range from 20-160 with higher scores indicating better functioning. |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Clinical Trials | Hoffmann-La Roche | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Harmonex Neuroscience Research | Dothan | Alabama | 36303 | United States | ||
| Southwest Autism Research & Resource Center |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41198718 | Derived | Aponte EA, Tillmann J, Gleissl T, Del Valle Rubido M, Murtagh L, Sanders K, Chatham CH, Wiese T, Suter EE. Anxiety, repetitive and restricted behaviors and interests, and social communication in autistic adults: an exploratory analysis of a phase 3, randomized clinical trial. Sci Rep. 2025 Nov 6;15(1):38912. doi: 10.1038/s41598-025-22659-y. | |
| 35151410 |
Not provided
Not provided
Participants received matching placebo in Blinded Treatment Period for 24 Weeks and 10 mg of oral administration balovaptan once a day (QD) during the Open Label Extension Treatment Period.
322 Participants were randomized. 1 Participants did not receive the treatment and in the ITT Population, 321 participants received at least one dose of the study treatment.
The Study was discontinued early before the planned sample size was reached.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Balovaptan | Participants received 10 mg of oral administration balovaptan once a day (QD). |
| FG001 | Placebo | Participants received matching placebo in Blinded Treatment Period and 10 mg of oral administration balovaptan once a day (QD) in OLE Treatment period |
| Title | Milestones | Reasons Not Completed | ||||
|---|---|---|---|---|---|---|
| Blinded Treatment Period |
|
Not provided
Not provided
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Feb 1, 2019 |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Placebo | Drug | Participants will receive matching placebo. |
|
| Week 12 |
| Change From Baseline at Weeks 12 and 24 in the Pediatric Quality of Life (PedsQL) Inventory Generic Core Scales, Version 4.0, on Summary and Total Scores | The Pediatric Quality of Life Inventory PedsQL™4.0 Generic Core Scale assessment consists of a 23 item questionnaire encompassing 4 core scale domains: Physical Functioning (8 items); Emotional Functioning (5 items); Social Functioning (5 items); and School Functioning (5 items). Items are scored on a 5 point Likert-type response scale (0=never a problem; 1=almost never a problem; 2=sometimes a problem; 3=often a problem; and 4=almost always a problem). Once scored, items will be reverse scored and linearly transformed to a 0-100 scale (0=100, 1=75, 2=50, 3=25, 4=0), so that higher scores indicate better health-related quality of life. | Weeks 12 and 24 |
| Change From Baseline at Weeks 12 and 24 in the Vineland-II Adaptive Behavior Composite Standard Score | The Vineland-II is an instrument that measures communication, daily living skills, socialization, motor skills (only in children up to 6 years) and maladaptive (not assessed in this study) behavior of individuals with developmental disabilities. The Survey Interview Form (i.e., semi -structured interview) will be administered to a subject's reliable study partner in this study, during which the rater or clinician will ask to the study partner open ended questions relating to the subject's activities and behavior. Domain scores will be obtained for the individual domains of Socialization, Communication, Daily Living Skills, and motor skills (up to 6 years only) and used to calculate the Vineland-II Adaptive Behavior Composite score. Standardized scores on the Adaptive behavior composite range from 20-160 with higher scores indicating better functioning. Only descriptive statistics presented instead of the planned estimated due to the early discontinuation of the study due to futility. | Weeks 12 and 24 |
| Change From Baseline at Week 12 and 24 on the Vineland-II Socialization Domain Standard Score | The Vineland-II is an instrument that measures communication, daily living skills, socialization, motor skills (only in children up to 6 years) and maladaptive (not assessed in this study) behavior of individuals with developmental disabilities. The Survey Interview Form (i.e., semi -structured interview) will be administered to a subject's reliable study partner in this study, during which the rater or clinician will ask to the study partner open ended questions relating to the subject's activities and behavior. Domain scores will be obtained for the individual domains of Socialization, Communication, Daily Living Skills, and motor skills (up to 6 years only) and used to calculate the Vineland-II Adaptive Behavior Composite score. Standardized scores on the Adaptive behavior composite range from 20-160 with higher scores indicating better functioning. Only descriptive statistics presented instead of the planned estimand due to the early discontinuation of the study due to futility. | Baseline, Weeks 12 and 24 |
| Change From Baseline at Weeks 12 and 24 on the Vineland-II Communication Domain Standard Score | The Vineland-II is an instrument that measures communication, daily living skills, socialization, motor skills (only in children up to 6 years) and maladaptive (not assessed in this study) behavior of individuals with developmental disabilities. The Survey Interview Form (i.e., semi -structured interview) will be administered to a subject's reliable study partner in this study, during which the rater or clinician will ask to the study partner open ended questions relating to the subject's activities and behavior. Domain scores will be obtained for the individual domains of Socialization, Communication, Daily Living Skills, and motor skills (up to 6 years only) and used to calculate the Vineland-II Adaptive Behavior Composite score. Standardized scores on the Adaptive behavior composite range from 20-160 with higher scores indicating better functioning. | Weeks 12 and 24 |
| Change From Baseline at Weeks 12 and 24 on the Vineland-II Daily Living Skills Domain Standard Score | The Vineland-II is an instrument that measures communication, daily living skills, socialization, motor skills (only in children up to 6 years) and maladaptive (not assessed in this study) behavior of individuals with developmental disabilities. The Survey Interview Form (i.e., semi -structured interview) will be administered to a subject's reliable study partner in this study, during which the rater or clinician will ask to the study partner open ended questions relating to the subject's activities and behavior. Domain scores will be obtained for the individual domains of Socialization, Communication, Daily Living Skills, and motor skills (up to 6 years only) and used to calculate the Vineland-II Adaptive Behavior Composite score. Standardized scores on the Adaptive behavior composite range from 20-160 with higher scores indicating better functioning. Only descriptive statistics presented instead of the planned estimated due to the early discontinuation of the study due to futility. | Weeks 12 and 24 |
| Change From Baseline in Severity of Clinical Impressions as Measured by Clinical Global Impression-Severity (CGI-S) | The CGI-S reflects the rater's impression of the subject's current autism severity on a 7-point scale ranging from no symptoms (1) to very severe symptoms (7). Changes in CGI-S score were calculated as increase or decrease in absolute CGI-S scores between Baseline and Weeks 12 and 24. Percentage of participants reported for each change in score from baseline. | Weeks 12 and 24 |
| Improvements in Clinical Impressions, as Measured by Clinical Global Impression-Improvement (CGI-I) | This is a 7-point Likert scale that assesses improvement of the patient's condition. Scores range from the worst score of 7 (Very much worse) to the best score of 1 (Very much improved). Lower scores are better on this scale, and indicate greater improvement. Percentage of participants reported for each score. | Weeks 12 and 24 |
| Change From Baseline at Weeks 12 and 24 in the Hamilton Anxiety Rating Scale (HAM-A) Total and Domain Scores | The HAM-A is a 14-item, rater administered interview, assessing the severity of anxiety symptoms during the past 7 days. Seven items assess psychic anxiety and seven assess somatic anxiety. Each item utilizes a 5-point symptom severity response scale, ranging from none (0) to very severe (4). A total score is calculated that ranges from 0 to 56; higher scores are indicative of more severe anxiety. | Weeks 12 and 24 |
| Proportion of Subjects With a >=6-point Improvement in Vineland-II 2DC Score | The Vineland-II is an instrument that measures communication, daily living skills, socialization, motor skills (only in children up to 6 years) and maladaptive (not assessed in this study) behavior of individuals with developmental disabilities. The Survey Interview Form (i.e., semi -structured interview) will be administered to a subject's reliable study partner in this study, during which the rater or clinician will ask to the study partner open ended questions relating to the subject's activities and behavior. Domain scores will be obtained for the individual domains of Socialization, Communication, Daily Living Skills, and motor skills (up to 6 years only) and used to calculate the Vineland-II Adaptive Behavior Composite score. Standardized scores on the Adaptive behavior composite range from 20-160 with higher scores indicating better functioning All participants who have an improvement of at least 6 points are included in the >=6 score threshold | Weeks 12 and 24 |
| Percentage of Participants With Adverse Events | According to the ICH guideline for Good Clinical Practice, an adverse event is any untoward medical occurrence in a clinical investigation subject administered a pharmaceutical product, regardless of causal attribution. The Blinded Treatment Period continued for 24 weeks, Open Label Extension (OLE) Treatment Period continued up to 2 years. The study was pre-maturely terminated, therefore did not reach the planned end date. | Week 24 and Up to Approximately 2 Years |
| Phoenix |
| Arizona |
| 85006 |
| United States |
| Woodland Research Northwest, LLC | Rogers | Arkansas | 72758 | United States |
| University of California , Los Angeles (UCLA); Child, Adolescent Psychiatry | Los Angeles | California | 90095 | United States |
| PCSD Feighner Research | San Diego | California | 92108 | United States |
| University of California at San Francisco | San Francisco | California | 94115 | United States |
| MCB Clinical Research Centers | Colorado Springs | Colorado | 80910 | United States |
| Yale University / Yale-New Haven Hospital | New Haven | Connecticut | 06519-1124 | United States |
| Sarkis Clinical Trials | Gainesville | Florida | 32607 | United States |
| APG- Advanced Psychiatric Group | Orlando | Florida | 32803 | United States |
| IMIC Inc. | Palmetto Bay | Florida | 33157 | United States |
| Rush University Medical Center | Chicago | Illinois | 60612 | United States |
| Uni of Chicago; Centre For Advanced Medicine | Chicago | Illinois | 60637 | United States |
| Lake Charles Clinical Trials, LLC | Lake Charles | Louisiana | 70601 | United States |
| The Johns Hopkins Hospital | Baltimore | Maryland | 21287 | United States |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
| University of Minnesota Medical Center-Fairview | Minneapolis | Minnesota | 55414 | United States |
| Millennium Psychiatric Associates, LLC | St Louis | Missouri | 63132 | United States |
| Hapworth Research Inc. | New York | New York | 10019 | United States |
| Center for Autism and the Developing Brain | New York | New York | 10032 | United States |
| Nathan S. Kline Institute for Psychiatric Research | Orangeburg | New York | 10962 | United States |
| Richmond Behavioral Associates | Staten Island | New York | 10312 | United States |
| University Hospitals | Cleveland | Ohio | 44106 | United States |
| Ohio State University | Columbus | Ohio | 43210 | United States |
| Cutting Edge Research Group | Oklahoma City | Oklahoma | 73116 | United States |
| UPMC Western Psychiatric Institute and Clinic | Pittsburgh | Pennsylvania | 15203 | United States |
| Vanderbilt University Medical Center; Department of Psychiatry | Nashville | Tennessee | 37212 | United States |
| BioBehavioral Research of Austin, PC | Austin | Texas | 78759 | United States |
| Red Oak Psychiatry Associates, PA | Houston | Texas | 77090 | United States |
| Aspen Clinical Research | Orem | Utah | 84058 | United States |
| Northwest Clinical Research Center | Bellevue | Washington | 98007 | United States |
| Seattle Children's Hospital | Seattle | Washington | 98105 | United States |
| Okanagan Clinical Trials | Kelowna | British Columbia | V1Y 1Z9 | Canada |
| Holland Bloorview Kids Rehabilitation Hospital; Autism Research Centre | East York | Ontario | M4G 1R8 | Canada |
| University of Western Ontario | London | Ontario | N6A 4G5 | Canada |
| McGill University Health Centre - Glen Site | Montreal | Quebec | H4A 3J1 | Canada |
| Hopital Charles Perrens; Centre de Ressources Autisme Aquitaine | Bordeaux | 33076 | France |
| Hospices Civils de Lyon; Centre d'Investigation Clinique Pédiatrique | Lyon | 69003 | France |
| Centre hospitalier du Rouvray; CRAHN Centre de Ressources Autisme Haute-Normandie | Sotteville-lès-Rouen | 76300 | France |
| ASST di Pavia; Dip. di Scienze del Sistema Nervoso e del Comportamento | Pavia | Lombardy | 27100 | Italy |
| AUSL di Piacenza; Psichiatria di Collegamento | Piacenza | Lombardy | 29121 | Italy |
| ASL TO2; Centro Pilota Regione Piemonte - Dip. Salute Mentale | Turin | Piedmont | 10138 | Italy |
| A.O.U. Policlinico - V. Emanuele - P.O. Gaspare Rodolico; Dip. Terapia integrata disturbi resistenti | Catania | Sicily | 95123 | Italy |
| Hospital Mutua de Terrassa; Departamento de Psiquiatria | Terrassa | Barcelona | 08221 | Spain |
| Hospital Universitari Vall d'Hebron; Sevicio de Psiquiatría | Barcelona | 08035 | Spain |
| Hospital General Universitario Gregorio Marañon; Servicio de Psiquiatria del niño y del adolescente | Madrid | 28009 | Spain |
| Hospital Universitario Rio Hortega; Departamento de Psiquiatria | Valladolid | 47012 | Spain |
| Western General Hospital; Wellcome Trust CRF | Edinburgh | EH4 2XU | United Kingdom |
| Queen Elizabeth University Hospital; Clinical Research Facility | Glasgow | G51 4TF | United Kingdom |
| Kings College Hospital; Kings Clinical Research Facility | London | SE5 9RS | United Kingdom |
| RE:Cognition Health; RE:Cognition Health | London | W1G 9JF | United Kingdom |
| Jacob S, Veenstra-VanderWeele J, Murphy D, McCracken J, Smith J, Sanders K, Meyenberg C, Wiese T, Deol-Bhullar G, Wandel C, Ashford E, Anagnostou E. Efficacy and safety of balovaptan for socialisation and communication difficulties in autistic adults in North America and Europe: a phase 3, randomised, placebo-controlled trial. Lancet Psychiatry. 2022 Mar;9(3):199-210. doi: 10.1016/S2215-0366(21)00429-6. Epub 2022 Feb 10. |
|
| COMPLETED | ITT Population |
|
| NOT COMPLETED |
|
|
| Open Label Extension Treatment Period |
|
|
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Balovaptan | Participants received 10 mg of oral administration balovaptan once a day (QD). |
| BG001 | Placebo | Participants received matching placebo. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change From Baseline at Week 24 on the Vineland Adaptive Behavior Scales (Vineland-II) Two-domain Composite (2DC) Score. | Vineland™-II Adaptive Behavior Scales 2-Domain Composite (2DC) Score is defined as mean of the Communication domain standard score & Socialization domain standard score. If any of the 2 individual domain standard scores is missing 2DC score is not computed. Vineland™-II is an instrument that measures communication, daily living skills, socialization, motor skills and maladaptive behavior of individuals with developmental disabilities. Survey Interview Form will be administered to a subject's reliable study partner in this study, during which the rater or clinician will ask to the study partner open ended questions relating to the subject's activities and behavior. Standardized scores on the Adaptive behavior composite range from 20-160 with higher scores indicating better functioning. | ITT Population | Posted | Mean | Standard Deviation | Score | Week 24 |
|
|
| ||||||||||||||||||||||||||||
| Secondary | Change From Baseline at Week 12 on the Vineland-II 2DC Score | Vineland™-II Adaptive Behavior Scales 2-Domain Composite (2DC) Score is defined as mean of the Communication domain standard score & Socialization domain standard score. If any of the 2 individual domain standard scores is missing 2DC score is not computed. Vineland™-II is an instrument that measures communication, daily living skills, socialization, motor skills and maladaptive behavior of individuals with developmental disabilities. Survey Interview Form will be administered to a subject's reliable study partner in this study, during which the rater or clinician will ask to the study partner open ended questions relating to the subject's activities and behavior. Standardized scores on the Adaptive behavior composite range from 20-160 with higher scores indicating better functioning. | ITT Population | Posted | Mean | Standard Deviation | Score | Week 12 |
|
| |||||||||||||||||||||||||||||
| Secondary | Change From Baseline at Weeks 12 and 24 in the Pediatric Quality of Life (PedsQL) Inventory Generic Core Scales, Version 4.0, on Summary and Total Scores | The Pediatric Quality of Life Inventory PedsQL™4.0 Generic Core Scale assessment consists of a 23 item questionnaire encompassing 4 core scale domains: Physical Functioning (8 items); Emotional Functioning (5 items); Social Functioning (5 items); and School Functioning (5 items). Items are scored on a 5 point Likert-type response scale (0=never a problem; 1=almost never a problem; 2=sometimes a problem; 3=often a problem; and 4=almost always a problem). Once scored, items will be reverse scored and linearly transformed to a 0-100 scale (0=100, 1=75, 2=50, 3=25, 4=0), so that higher scores indicate better health-related quality of life. | ITT Population | Posted | Mean | Standard Deviation | Score | Weeks 12 and 24 |
|
| |||||||||||||||||||||||||||||
| Secondary | Change From Baseline at Weeks 12 and 24 in the Vineland-II Adaptive Behavior Composite Standard Score | The Vineland-II is an instrument that measures communication, daily living skills, socialization, motor skills (only in children up to 6 years) and maladaptive (not assessed in this study) behavior of individuals with developmental disabilities. The Survey Interview Form (i.e., semi -structured interview) will be administered to a subject's reliable study partner in this study, during which the rater or clinician will ask to the study partner open ended questions relating to the subject's activities and behavior. Domain scores will be obtained for the individual domains of Socialization, Communication, Daily Living Skills, and motor skills (up to 6 years only) and used to calculate the Vineland-II Adaptive Behavior Composite score. Standardized scores on the Adaptive behavior composite range from 20-160 with higher scores indicating better functioning. Only descriptive statistics presented instead of the planned estimated due to the early discontinuation of the study due to futility. | ITT Population | Posted | Mean | Standard Deviation | Score | Weeks 12 and 24 |
| ||||||||||||||||||||||||||||||
| Secondary | Change From Baseline at Week 12 and 24 on the Vineland-II Socialization Domain Standard Score | The Vineland-II is an instrument that measures communication, daily living skills, socialization, motor skills (only in children up to 6 years) and maladaptive (not assessed in this study) behavior of individuals with developmental disabilities. The Survey Interview Form (i.e., semi -structured interview) will be administered to a subject's reliable study partner in this study, during which the rater or clinician will ask to the study partner open ended questions relating to the subject's activities and behavior. Domain scores will be obtained for the individual domains of Socialization, Communication, Daily Living Skills, and motor skills (up to 6 years only) and used to calculate the Vineland-II Adaptive Behavior Composite score. Standardized scores on the Adaptive behavior composite range from 20-160 with higher scores indicating better functioning. Only descriptive statistics presented instead of the planned estimand due to the early discontinuation of the study due to futility. | ITT Population | Posted | Mean | Standard Deviation | Score | Baseline, Weeks 12 and 24 |
| ||||||||||||||||||||||||||||||
| Secondary | Change From Baseline at Weeks 12 and 24 on the Vineland-II Communication Domain Standard Score | The Vineland-II is an instrument that measures communication, daily living skills, socialization, motor skills (only in children up to 6 years) and maladaptive (not assessed in this study) behavior of individuals with developmental disabilities. The Survey Interview Form (i.e., semi -structured interview) will be administered to a subject's reliable study partner in this study, during which the rater or clinician will ask to the study partner open ended questions relating to the subject's activities and behavior. Domain scores will be obtained for the individual domains of Socialization, Communication, Daily Living Skills, and motor skills (up to 6 years only) and used to calculate the Vineland-II Adaptive Behavior Composite score. Standardized scores on the Adaptive behavior composite range from 20-160 with higher scores indicating better functioning. | ITT Population | Posted | Mean | Standard Deviation | Score | Weeks 12 and 24 |
| ||||||||||||||||||||||||||||||
| Secondary | Change From Baseline at Weeks 12 and 24 on the Vineland-II Daily Living Skills Domain Standard Score | The Vineland-II is an instrument that measures communication, daily living skills, socialization, motor skills (only in children up to 6 years) and maladaptive (not assessed in this study) behavior of individuals with developmental disabilities. The Survey Interview Form (i.e., semi -structured interview) will be administered to a subject's reliable study partner in this study, during which the rater or clinician will ask to the study partner open ended questions relating to the subject's activities and behavior. Domain scores will be obtained for the individual domains of Socialization, Communication, Daily Living Skills, and motor skills (up to 6 years only) and used to calculate the Vineland-II Adaptive Behavior Composite score. Standardized scores on the Adaptive behavior composite range from 20-160 with higher scores indicating better functioning. Only descriptive statistics presented instead of the planned estimated due to the early discontinuation of the study due to futility. | ITT Population | Posted | Mean | Standard Deviation | Score | Weeks 12 and 24 |
| ||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Severity of Clinical Impressions as Measured by Clinical Global Impression-Severity (CGI-S) | The CGI-S reflects the rater's impression of the subject's current autism severity on a 7-point scale ranging from no symptoms (1) to very severe symptoms (7). Changes in CGI-S score were calculated as increase or decrease in absolute CGI-S scores between Baseline and Weeks 12 and 24. Percentage of participants reported for each change in score from baseline. | ITT Population | Posted | Number | Percentage of Participants | Weeks 12 and 24 |
|
| ||||||||||||||||||||||||||||||
| Secondary | Improvements in Clinical Impressions, as Measured by Clinical Global Impression-Improvement (CGI-I) | This is a 7-point Likert scale that assesses improvement of the patient's condition. Scores range from the worst score of 7 (Very much worse) to the best score of 1 (Very much improved). Lower scores are better on this scale, and indicate greater improvement. Percentage of participants reported for each score. | ITT Population | Posted | Number | Percentage of Participants | Weeks 12 and 24 |
|
| ||||||||||||||||||||||||||||||
| Secondary | Change From Baseline at Weeks 12 and 24 in the Hamilton Anxiety Rating Scale (HAM-A) Total and Domain Scores | The HAM-A is a 14-item, rater administered interview, assessing the severity of anxiety symptoms during the past 7 days. Seven items assess psychic anxiety and seven assess somatic anxiety. Each item utilizes a 5-point symptom severity response scale, ranging from none (0) to very severe (4). A total score is calculated that ranges from 0 to 56; higher scores are indicative of more severe anxiety. | ITT Population | Posted | Mean | Standard Deviation | Score | Weeks 12 and 24 |
|
| |||||||||||||||||||||||||||||
| Secondary | Proportion of Subjects With a >=6-point Improvement in Vineland-II 2DC Score | The Vineland-II is an instrument that measures communication, daily living skills, socialization, motor skills (only in children up to 6 years) and maladaptive (not assessed in this study) behavior of individuals with developmental disabilities. The Survey Interview Form (i.e., semi -structured interview) will be administered to a subject's reliable study partner in this study, during which the rater or clinician will ask to the study partner open ended questions relating to the subject's activities and behavior. Domain scores will be obtained for the individual domains of Socialization, Communication, Daily Living Skills, and motor skills (up to 6 years only) and used to calculate the Vineland-II Adaptive Behavior Composite score. Standardized scores on the Adaptive behavior composite range from 20-160 with higher scores indicating better functioning All participants who have an improvement of at least 6 points are included in the >=6 score threshold | ITT Population | Posted | Number | Percentage of Participants | Weeks 12 and 24 |
| |||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants With Adverse Events | According to the ICH guideline for Good Clinical Practice, an adverse event is any untoward medical occurrence in a clinical investigation subject administered a pharmaceutical product, regardless of causal attribution. The Blinded Treatment Period continued for 24 weeks, Open Label Extension (OLE) Treatment Period continued up to 2 years. The study was pre-maturely terminated, therefore did not reach the planned end date. | Safety Population | Posted | Number | Percentage of Participants | Week 24 and Up to Approximately 2 Years |
|
From baseline to the end of Safety Period, up to 2 years
The proportion of patients with at least one AE in the balovaptan and placebo treatment arms in the Blinded Treatment period and Open Label Extension (OLE)Treatment period.
Other Adverse Events are reported at 5% frequency threshold. In the Open Label Extension (OLE) Treatment Period, the participants received active treatment, i.e. 10mg balovaptan QD. Placebo was only given during the blinded treatment period but not during OLE Treatment Period.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Balovaptan in Blinded Treatment Period | Participants received 10 mg of oral administration balovaptan once a day (QD). | 0 | 163 | 2 | 163 | 49 | 163 |
| EG001 | Placebo Blinded Treatment Period | Participants received matching placebo. | 1 | 158 | 5 | 158 | 59 | 158 |
| EG002 | Balovaptan in Open Label Extension Treatment Period | Participants received 10 mg of oral administration balovaptan once a day (QD). | 0 | 100 | 0 | 100 | 28 | 100 |
| EG003 | Placebo in Open Label Extension Treatment Period | Participants received matching placebo in Blinded Treatment Period and 10 mg of oral administration balovaptan once a day (QD). OLE Treatment Period | 0 | 97 | 2 | 97 | 30 | 97 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Panic disorder | Psychiatric disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Colitis | Gastrointestinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Abscess limb | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Urosepsis | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Completed suicide | Psychiatric disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Schizoaffective disorder | Psychiatric disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Suicidal ideation | Psychiatric disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Pharyngitis streptococcal | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Femur fracture | Injury, poisoning and procedural complications | MedDRA v23.0 | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
|
The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Reference Study ID Number: WN39434 | www.roche.com/about_roche/roche_worldwide.htm | 888-662-6728 (U.S. Only) | global-roche-genentech-trials@gene.com |
| Mar 2, 2021 |
| Prot_SAP_000.pdf |
Not provided
| ID | Term |
|---|---|
| D000067877 | Autism Spectrum Disorder |
| ID | Term |
|---|---|
| D002659 | Child Development Disorders, Pervasive |
| D065886 | Neurodevelopmental Disorders |
| D001523 | Mental Disorders |
Not provided
Not provided
| ID | Term |
|---|---|
| C000708839 | balovaptan |
Not provided
Not provided
Not provided
| Lost to Follow-up |
|
| Lack of Efficacy |
|
| Adverse Event |
|
| Non-compliance with study drug |
|
| Study terminated by sponsor |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
|
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Units |
|---|
| Counts |
|---|
| Participants |
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
Participants received matching placebo in Blinded Treatment Period and 10 mg of oral administration balovaptan once a day (QD). OLE Treatment Period
|
|