Trial · NCT03504917
A Study of Balovaptan in Adults With Autism Spectrum Disorder With a 2-Year Open-Label Extension
NCT03504917, Phase 3, Terminated, Hoffmann-La Roche; results first posted 2021-05-07
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study of Balovaptan in Adults With Autism Spectrum Disorder With a 2-Year Open-Label Extension. Trials, record NCT03504917. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03504917
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of Balovaptan in Adults With Autism Spectrum Disorder With a 2-Year Open-Label Extension
- NCT number
- NCT03504917
- Status
- Terminated
- Phases
- Phase 3
- Enrollment
- 322 participants, actual
- Lead sponsor
- Hoffmann-La Roche
- Conditions
- Autism Spectrum Disorder
- Interventions
- Balovaptan, Placebo
- First posted
- 2018-04-20
- Last updated
- 2021-10-27
- Results first posted
- 2021-05-07
Source ClinicalTrials.gov
Registered outcomes12
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline at Week 24 on the Vineland Adaptive Behavior Scales (Vineland-II) Two-domain Composite (2DC) Score. | Week 24 | 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. |
| Secondary | Change From Baseline at Week 12 on the Vineland-II 2DC Score | Week 12 | 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. |
| 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 | Weeks 12 and 24 | 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. |
| Secondary | Change From Baseline at Weeks 12 and 24 in the Vineland-II Adaptive Behavior Composite Standard Score | Weeks 12 and 24 | 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. |
| Secondary | Change From Baseline at Week 12 and 24 on the Vineland-II Socialization Domain Standard Score | Baseline, Weeks 12 and 24 | 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. |
| Secondary | Change From Baseline at Weeks 12 and 24 on the Vineland-II Communication Domain Standard Score | Weeks 12 and 24 | 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. |
| Secondary | Change From Baseline at Weeks 12 and 24 on the Vineland-II Daily Living Skills Domain Standard Score | Weeks 12 and 24 | 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. |
| Secondary | Change From Baseline in Severity of Clinical Impressions as Measured by Clinical Global Impression-Severity (CGI-S) | Weeks 12 and 24 | 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. |
| Secondary | Improvements in Clinical Impressions, as Measured by Clinical Global Impression-Improvement (CGI-I) | Weeks 12 and 24 | 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. |
| Secondary | Change From Baseline at Weeks 12 and 24 in the Hamilton Anxiety Rating Scale (HAM-A) Total and Domain Scores | Weeks 12 and 24 | 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. |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 registered outcomesOutcomes12
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline at Week 24 on the Vineland Adaptive Behavior Scales (Vineland-II) Two-domain Composite (2DC) Score. | Week 24 | ITT Population | Score | MEAN |
| SECONDARY | Change From Baseline at Week 12 on the Vineland-II 2DC Score | Week 12 | ITT Population | Score | MEAN |
| 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 | Weeks 12 and 24 | ITT Population | Score | MEAN |
| SECONDARY | Change From Baseline at Weeks 12 and 24 in the Vineland-II Adaptive Behavior Composite Standard Score | Weeks 12 and 24 | ITT Population | Score | MEAN |
| SECONDARY | Change From Baseline at Week 12 and 24 on the Vineland-II Socialization Domain Standard Score | Baseline, Weeks 12 and 24 | ITT Population | Score | MEAN |
| SECONDARY | Change From Baseline at Weeks 12 and 24 on the Vineland-II Communication Domain Standard Score | Weeks 12 and 24 | ITT Population | Score | MEAN |
| SECONDARY | Change From Baseline at Weeks 12 and 24 on the Vineland-II Daily Living Skills Domain Standard Score | Weeks 12 and 24 | ITT Population | Score | MEAN |
| SECONDARY | Change From Baseline in Severity of Clinical Impressions as Measured by Clinical Global Impression-Severity (CGI-S) | Weeks 12 and 24 | ITT Population | Percentage of Participants | NUMBER |
| SECONDARY | Improvements in Clinical Impressions, as Measured by Clinical Global Impression-Improvement (CGI-I) | Weeks 12 and 24 | ITT Population | Percentage of Participants | NUMBER |
| SECONDARY | Change From Baseline at Weeks 12 and 24 in the Hamilton Anxiety Rating Scale (HAM-A) Total and Domain Scores | Weeks 12 and 24 | ITT Population | Score | MEAN |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 outcomesResults by group108
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline at Week 24 on the Vineland Adaptive Behavior Scales (Vineland-II) Two-domain Composite (2DC) Score. | Balovaptan | 4.56 | Standard Deviation 10.85 | Score | |
| Change From Baseline at Week 24 on the Vineland Adaptive Behavior Scales (Vineland-II) Two-domain Composite (2DC) Score. | Placebo | 6.83 | Standard Deviation 12.18 | Score | |
| Change From Baseline at Week 12 on the Vineland-II 2DC Score | Balovaptan | 3.47 | Standard Deviation 10.00 | Score | |
| Change From Baseline at Week 12 on the Vineland-II 2DC Score | Placebo | 4.85 | Standard Deviation 12.64 | Score | |
| 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 | Balovaptan | Total Score at Week 12 | 4.1 | Standard Deviation 11.2 | Score |
| 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 | Placebo | Total Score at Week 12 | 5.0 | Standard Deviation 10.2 | Score |
| 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 | Balovaptan | Psychosocial Health Summary Score at Week 12 | 4.9 | Standard Deviation 13.2 | Score |
| 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 | Placebo | Psychosocial Health Summary Score at Week 12 | 5.5 | Standard Deviation 12.7 | Score |
| 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 | Balovaptan | Physical Health Summary Score at Week 12 | 2.5 | Standard Deviation 13.3 | Score |
| 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 | Placebo | Physical Health Summary Score at Week 12 | 4.3 | Standard Deviation 12.3 | Score |
1 to 10 of 108Source ClinicalTrials.gov
See all 108 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Blinded Treatment Period | STARTED | Balovaptan | 163 | |
| Blinded Treatment Period | STARTED | Placebo | 158 | |
| Blinded Treatment Period | COMPLETED | Balovaptan | 103 | |
| Blinded Treatment Period | COMPLETED | Placebo | 102 | |
| Blinded Treatment Period | NOT COMPLETED | Balovaptan | 60 | |
| Blinded Treatment Period | NOT COMPLETED | Placebo | 56 | |
| Open Label Extension Treatment Period | STARTED | Balovaptan | 100 | |
| Open Label Extension Treatment Period | STARTED | Placebo | 97 | |
| Open Label Extension Treatment Period | COMPLETED | Balovaptan | 0 | |
| Open Label Extension Treatment Period | COMPLETED | Placebo | 0 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events84
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Diarrhoea | Gastrointestinal disorders | Balovaptan in Blinded Treatment Period | 11 | 163 | 15 |
| other | Diarrhoea | Gastrointestinal disorders | Placebo Blinded Treatment Period | 14 | 158 | 15 |
| other | Diarrhoea | Gastrointestinal disorders | Balovaptan in Open Label Extension Treatment Period | 5 | 100 | 5 |
| other | Diarrhoea | Gastrointestinal disorders | Placebo in Open Label Extension Treatment Period | 4 | 97 | 4 |
| other | Nasopharyngitis | Infections and infestations | Balovaptan in Blinded Treatment Period | 14 | 163 | 14 |
| other | Nasopharyngitis | Infections and infestations | Placebo Blinded Treatment Period | 19 | 158 | 22 |
| other | Nasopharyngitis | Infections and infestations | Balovaptan in Open Label Extension Treatment Period | 7 | 100 | 10 |
| other | Nasopharyngitis | Infections and infestations | Placebo in Open Label Extension Treatment Period | 4 | 97 | 4 |
| other | Upper respiratory tract infection | Infections and infestations | Balovaptan in Blinded Treatment Period | 10 | 163 | 13 |
| other | Upper respiratory tract infection | Infections and infestations | Placebo Blinded Treatment Period | 9 | 158 | 11 |
1 to 10 of 84Source ClinicalTrials.gov
See all 84 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Balovaptan in Blinded Treatment Period | 0 | 163 |
| Total, all-cause mortality | Placebo Blinded Treatment Period | 1 | 158 |
| Total, all-cause mortality | Balovaptan in Open Label Extension Treatment Period | 0 | 100 |
| Total, all-cause mortality | Placebo in Open Label Extension Treatment Period | 0 | 97 |
| Total, other adverse events | Balovaptan in Blinded Treatment Period | 49 | 163 |
| Total, other adverse events | Placebo Blinded Treatment Period | 59 | 158 |
| Total, other adverse events | Balovaptan in Open Label Extension Treatment Period | 28 | 100 |
| Total, other adverse events | Placebo in Open Label Extension Treatment Period | 30 | 97 |
| Total, serious adverse events | Balovaptan in Blinded Treatment Period | 2 | 163 |
| Total, serious adverse events | Placebo Blinded Treatment Period | 5 | 158 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
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GET https://conductscience.org/api/trials/v1/NCT03504917/results?page=2