Adults 5 to 17, any sex, with Autism Spectrum Disorder. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Change From Baseline in Vineland™-II Adaptive Behavior Scale Two Domain Composite (2DC) Score at Week 24 for Balovaptan (R05285119) 10 mg Equivalent Compared to PlaceboPrimary· Baseline, 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 caregiver in this study, during which the rater or clinician will ask to the caregiver open e
Group
Value
95% CI
Placebo
2.34
± 1.15
Balovaptan (RO5285119) 10 mg/d Equivalent
2.17
± 1.11
Change From Baseline in Vineland™-II Composite Standard Score After 12 Weeks and 24 Weeks of Treatment for Balovaptan (R05285119) 10 mg Equivalent Compared to PlaceboSecondary· 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 caregiver in this study, during which the rater or clinician will ask to the caregiver open ended questions relating to the subject's activities and behavior. Domain scores will be obtained for the individual domains of Socialization, Communicatio
Week 12
Group
Value
95% CI
Placebo
1.45
± 1.07
Balovaptan (RO5285119) 10 mg/d Equivalent
1.74
± 1.04
Week 24
Group
Value
95% CI
Placebo
2.20
± 1.19
Balovaptan (RO5285119) 10 mg/d Equivalent
1.97
± 1.15
Change From Baseline in Vineland™-II Adaptive Behavior Scale Communication, Socialization, and Daily Living Skills Domain Standard Scores at Weeks 12 and 24 for Balovaptan (R05285119) 10 mg Equivalent Compared to PlaceboSecondary· 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 caregiver in this study, during which the rater or clinician will ask to the caregiver open ended questions relating to the subject's activities and behavior. Domain scores will be obtained for the individual domains of Socialization, Communicatio
Communication Domain Standard Score, Week 12
Group
Value
95% CI
Placebo
1.86
± 1.06
Balovaptan (RO5285119) 10 mg/d Equivalent
1.64
± 1.03
Communication Domain Standard Score, Week 24
Group
Value
95% CI
Placebo
1.51
± 1.13
Balovaptan (RO5285119) 10 mg/d Equivalent
2.21
± 1.09
Socialization Domain Standard Score, Week 12
Group
Value
95% CI
Placebo
1.69
± 1.31
Balovaptan (RO5285119) 10 mg/d Equivalent
2.20
± 1.26
Socialization Domain Standard Score, Week 24
Group
Value
95% CI
Placebo
2.87
± 1.50
Balovaptan (RO5285119) 10 mg/d Equivalent
2.26
± 1.45
Daily Living Skills Domain Standard Score, Week 12
Group
Value
95% CI
Placebo
-0.01
± 1.38
Balovaptan (RO5285119) 10 mg/d Equivalent
2.13
± 1.35
Daily Living Skills Domain Standard Score, Week 24
Group
Value
95% CI
Placebo
1.44
± 1.49
Balovaptan (RO5285119) 10 mg/d Equivalent
1.61
± 1.45
Proportion of Subjects With >=6 Points Improvement in the Vineland-II 2DC Score for Balovaptan (R05285119) 10 mg Equivalent Compared to PlaceboSecondary· Baseline, Weeks 12 and 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 caregiver in this study, during which the rater or clinician will ask to the caregiver open e
Week 12
Group
Value
95% CI
Placebo
30.3
Balovaptan (RO5285119) 10 mg/d Equivalent
27.1
Week 24
Group
Value
95% CI
Placebo
34.4
Balovaptan (RO5285119) 10 mg/d Equivalent
32.8
Change From Baseline in Clinical Global Impressions-Severity (CGI-S) Score at Weeks 12 and 24 for Balovaptan (R05285119) 10 mg Equivalent Compared to PlaceboSecondary· Baseline, 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.
-3 (Very much improved), Week 12
Group
Value
95% CI
Placebo
1.5
Balovaptan (RO5285119) 10 mg/d Equivalent
0
-2 (Much improved), Week 12
Group
Value
95% CI
Placebo
1.5
Balovaptan (RO5285119) 10 mg/d Equivalent
2.7
-1 (Minimally improved), Week 12
Group
Value
95% CI
Placebo
31.3
Balovaptan (RO5285119) 10 mg/d Equivalent
31.1
0 (No change), Week 12
Group
Value
95% CI
Placebo
65.7
Balovaptan (RO5285119) 10 mg/d Equivalent
63.5
+1 (Minimally worse), Week 12
Group
Value
95% CI
Placebo
0
Balovaptan (RO5285119) 10 mg/d Equivalent
2.7
+2 (Much worse), Week 12
Group
Value
95% CI
Placebo
0
Balovaptan (RO5285119) 10 mg/d Equivalent
0
+3 (Very Much worse), Week 12
Group
Value
95% CI
Placebo
0
Balovaptan (RO5285119) 10 mg/d Equivalent
0
-3 (Very much improved), Week 24
Group
Value
95% CI
Placebo
1.6
Balovaptan (RO5285119) 10 mg/d Equivalent
0
Change From Baseline in Ohio Autism Clinical Impressions Scale-Severity (OACIS-S) Score at Weeks 12 and 24 for Balovaptan (R05285119) 10 mg Equivalent Compared to PlaceboSecondary· Baseline, Weeks 12 and 24
The OACIS-S is a 10-item, clinician-completed measures based upon interview and/or observation. The OACIS-S assess severity and improvement, respectively, in social interaction, aberrant behavior, repetitive or ritualistic behavior, verbal communication, nonverbal communication skills, hyperactivity and inattention, anxiety and fearfulness, sensory sensitivities, restricted and narrow interests, and a global rating of autism. Each item of the OACIS-S is rated 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 decre
-3 (Very much improved), Week 12
Group
Value
95% CI
Placebo
3.0
Balovaptan (RO5285119) 10 mg/d Equivalent
0
-2 (Much improved), Week 12
Group
Value
95% CI
Placebo
6.0
Balovaptan (RO5285119) 10 mg/d Equivalent
6.8
-1 (Minimally improved), Week 12
Group
Value
95% CI
Placebo
28.4
Balovaptan (RO5285119) 10 mg/d Equivalent
34.2
0 (No change), Week 12
Group
Value
95% CI
Placebo
59.7
Balovaptan (RO5285119) 10 mg/d Equivalent
57.5
+1 (Minimally worse), Week 12
Group
Value
95% CI
Placebo
3.0
Balovaptan (RO5285119) 10 mg/d Equivalent
1.4
+2 (Much worse), Week 12
Group
Value
95% CI
Placebo
0
Balovaptan (RO5285119) 10 mg/d Equivalent
0
+3 (Very much worse), Week 12
Group
Value
95% CI
Placebo
0
Balovaptan (RO5285119) 10 mg/d Equivalent
0
-3 (Very much improved), Week 24
Group
Value
95% CI
Placebo
6.5
Balovaptan (RO5285119) 10 mg/d Equivalent
0
Clinical Global Impressions- Improvement (CGI-I) Score at Weeks 12 and 24 for Balovaptan (R05285119) 10 mg Equivalent Compared to PlaceboSecondary· Weeks 12 and 24
The CGI rating scales are tools used to evaluate both the severity of illness and change from baseline. The CGI-I is used to assess the clinical change as compared to symptoms at baseline using a 7-point scale, ranging from very much improved (1) to very much worse (7). For this study modified versions will be used.
1 - Very much improved, Week 12
Group
Value
95% CI
Placebo
0
Balovaptan (RO5285119) 10 mg/d Equivalent
0
2 - Much improved, Week 12
Group
Value
95% CI
Placebo
22.4
Balovaptan (RO5285119) 10 mg/d Equivalent
21.6
3 - Minimally improved, Week 12
Group
Value
95% CI
Placebo
49.3
Balovaptan (RO5285119) 10 mg/d Equivalent
35.1
4 - No change, Week 12
Group
Value
95% CI
Placebo
22.4
Balovaptan (RO5285119) 10 mg/d Equivalent
43.2
5 - Minimally worse, Week 12
Group
Value
95% CI
Placebo
6.0
Balovaptan (RO5285119) 10 mg/d Equivalent
0
6 - Much worse, Week 12
Group
Value
95% CI
Placebo
0
Balovaptan (RO5285119) 10 mg/d Equivalent
0
7 - Very much worse, Week 12
Group
Value
95% CI
Placebo
0
Balovaptan (RO5285119) 10 mg/d Equivalent
0
1 - Very much improved, Week 24
Group
Value
95% CI
Placebo
0
Balovaptan (RO5285119) 10 mg/d Equivalent
4.4
Ohio Autism Clinical Impressions Scale- Improvement (OACIS-I) Score at Weeks 12 and 24 for Balovaptan (R05285119) 10 mg Equivalent Compared to PlaceboSecondary· Weeks 12 and 24
The OACIS-I is a 10-item, clinician-completed measures based upon interview and/or observation. The OACIS-I assess severity and improvement, respectively, in social interaction, aberrant behavior, repetitive or ritualistic behavior, verbal communication, nonverbal communication skills, hyperactivity and inattention, anxiety and fearfulness, sensory sensitivities, restricted and narrow interests, and a global rating of autism. The OACIS-I is used to assess the clinical change as compared to symptoms at baseline using a 7-point scale, ranging from very much improved (1) to very much worse (7).
1 - Very much improved, Week 12
Group
Value
95% CI
Placebo
1.5
Balovaptan (RO5285119) 10 mg/d Equivalent
0
2 - Much improved, Week 12
Group
Value
95% CI
Placebo
16.4
Balovaptan (RO5285119) 10 mg/d Equivalent
20.3
3 - Minimally improved, Week 12
Group
Value
95% CI
Placebo
38.8
Balovaptan (RO5285119) 10 mg/d Equivalent
25.7
4 - No change, Week 12
Group
Value
95% CI
Placebo
40.3
Balovaptan (RO5285119) 10 mg/d Equivalent
54.1
5 - Minimally worse, Week 12
Group
Value
95% CI
Placebo
3.0
Balovaptan (RO5285119) 10 mg/d Equivalent
0
6 - Much worse, Week 12
Group
Value
95% CI
Placebo
0
Balovaptan (RO5285119) 10 mg/d Equivalent
0
7 - Very much worse, Week 12
Group
Value
95% CI
Placebo
0
Balovaptan (RO5285119) 10 mg/d Equivalent
0
1 - Very much improved, Week 24
Group
Value
95% CI
Placebo
0
Balovaptan (RO5285119) 10 mg/d Equivalent
2.9
Change From Baseline in Patient-Reported Pediatric Quality of Life (PedsQL) v4.0 Generic Core Scale After 12 Weeks and 24 Weeks of Treatment for Balovaptan (R05285119) 10 mg Equivalent Compared to PlaceboSecondary· Baseline, 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). For children aged 8 years and above, the PedsQL 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). For children aged 5-7 years, scoring is based on a three-point scale (0=Not at all, 2=Sometime
Change from Baseline at Week 12
Group
Value
95% CI
Placebo
2.42
± 1.49
Balovaptan (RO5285119) 10 mg/d Equivalent
6.16
± 1.41
Change from Baseline at Week 24
Group
Value
95% CI
Placebo
3.70
± 1.50
Balovaptan (RO5285119) 10 mg/d Equivalent
5.98
± 1.44
Change From Baseline in Vineland-II Composite Standard Score in Adolescents and Children Independently at Weeks 12 and 24 for Balovaptan (R05285119) 10 mg Equivalent Compared to PlaceboSecondary· 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 caregiver in this study, during which the rater or clinician will ask to the caregiver open ended questions relating to the subject's activities and behavior. Domain scores will be obtained for the individual domains of Socialization, Communication
5-12 Years Age Group, Absolute Value at Baseline
Group
Value
95% CI
Placebo
73.9
± 9.7
Balovaptan (RO5285119) 10 mg/d Equivalent
77.3
± 10.9
5-12 Years Age Group, Change From Baseline at Week 12
Group
Value
95% CI
Placebo
3.6
± 8.4
Balovaptan (RO5285119) 10 mg/d Equivalent
1.2
± 7.8
5-12 Years Age Group, Change From Baseline at Week 24
Group
Value
95% CI
Placebo
4.8
± 9.1
Balovaptan (RO5285119) 10 mg/d Equivalent
1.0
± 8.4
13-17 Years Age Group, Absolute Baseline Value
Group
Value
95% CI
Placebo
71.0
± 10.3
Balovaptan (RO5285119) 10 mg/d Equivalent
73.5
± 8.9
13-17 Years Age Group, Change From Baseline at Week 12
Group
Value
95% CI
Placebo
1.8
± 4.8
Balovaptan (RO5285119) 10 mg/d Equivalent
3.7
± 9.4
13-17 Years Age Group, Change From Baseline at Week 24
Group
Value
95% CI
Placebo
2.7
± 8.6
Balovaptan (RO5285119) 10 mg/d Equivalent
3.3
± 8.7
Change From Baseline in Vineland-II Adaptive Behavior Scale 2DC Score at Week 12 for Balovaptan (R05285119) 10 mg Equivalent Compared to PlaceboSecondary· Baseline, Week 12
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 caregiver in this study, during which the rater or clinician will ask to the caregiver open ended questions relating to the subject's activities and behavior. Domain scores will be obtained for the individual domains of Socialization, Communication
Group
Value
95% CI
Placebo
1.87
± 1.00
Balovaptan (RO5285119) 10 mg/d Equivalent
1.88
± 0.97
Percentage of Participants With Adverse Events for Treatment With BalovaptanSecondary· Baseline to Week 24 and up to Week 76
PK review revealed that age-adjusted doses of 4 and 10 mg in 5-17 year olds were not equivalent to target exposure. For the Main Study Part, data was summarised by exposure ranges (tertiles) based on individual participants PK exposure at Week 12, estimated as the average plasma concentration since treatment start. To allow clear analysis by exposure tertiles, participants with dose adjustment were excluded from analysis by tertiles.
Group
Value
95% CI
Main Study Part, Low Exposure Tertile
77.2
Main Study Part, Medium Exposure Tertile
71.2
Main Study Part, High Exposure Tertile
66.7
Main Study Part (Study Part 2) - All Treated
71.4
Open Label Extension Part, Low Exposure Tertile
68.6
Open Label Extension Part, Medium Exposure Tertile
70.0
Open Label Extension Part, High Exposure Tertile
78.3
Open Label Extension Part, All Treated
72.2
Adverse events — posted to ClinicalTrials.gov
Time frame: From the first study drug to the data cutoff date: 30 June 2020 (up to 43 months).
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
PK Part (Study Part 1) - Placebo
Serious: 0/12 (0%)
Deaths: 0/12
PK Part (Study Part 1) - Balovaptan (RO5285119) 4 mg/d Equivalent
Serious: 1/11 (9%)
Deaths: 0/11
PK Part (Study Part 1) - Balovaptan (RO5285119) 10 mg/d Equivalent
Serious: 0/15 (0%)
Deaths: 0/15
Main Study Part (Study Part 2) - Placebo
Serious: 4/112 (4%)
Deaths: 0/112
Main Study Part (Study Part 2) - Low Exposure Tertile
Serious: 1/57 (2%)
Deaths: 0/57
Main Study Part (Study Part 2) - Medium Exposure Tertile
Serious: 0/66 (0%)
Deaths: 0/66
Main Study Part (Study Part 2) - High Exposure Tertile
Serious: 1/66 (2%)
Deaths: 0/66
Main Study Part (Study Part 2) - Dose-Adjusters
Serious: 0/7 (0%)
Deaths: 0/7
Open Label Extension Part (Study Part 3) - Placebo
Serious: 2/68 (3%)
Deaths: 0/68
Open Label Extension Part (Study Part 3) - Low Exposure Tertile
Serious: 0/35 (0%)
Deaths: 0/35
Open Label Extension Part (Study Part 3) - Medium Exposure Tertile
Serious: 0/40 (0%)
Deaths: 0/40
Open Label Extension Part (Study Part 3) - High Exposure Tertile
Serious: 2/46 (4%)
Deaths: 0/46
Open Label Extension Part (Study Part 3) - Dose-Adjusters
Serious: 0/5 (0%)
Deaths: 0/5
Serious adverse events (11 terms)
Reaction
System
PK Part (Study Part 1) - P…
PK Part (Study Part 1) - B…
PK Part (Study Part 1) - B…
Main Study Part (Study Par…
Main Study Part (Study Par…
Main Study Part (Study Par…
Main Study Part (Study Par…
Main Study Part (Study Par…
Open Label Extension Part …
Open Label Extension Part …
Open Label Extension Part …
Open Label Extension Part …
Open Label Extension Part …
Pneumonia
Infections and infestations
—
—
—
—
—
—
—
—
—
—
—
—
—
Gastroenteritis viral
Infections and infestations
—
—
—
—
—
—
—
—
—
—
—
—
—
Dehydration
Metabolism and nutrition disorders
—
—
—
—
—
—
—
—
—
—
—
—
—
Acute lymphocytic leukaemia
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
For participants enrolled prior to Version 6 of the protocol: This was a Phase II multi-center, randomized, double-blind, 24-week, 3-arm, parallel group, placebo-controlled study to investigate the efficacy, safety, and pharmacokinetics of balovaptan in children and adolescents aged 5-17 years with ASD who are high functioning (intelligence quotient \[IQ\] greater than or equal to \[\>=\] 70).
For participants enrolled according to Version 6 of the protocol: This was a Phase II multi-center, randomized, double-blind, 24-week, parallel group, placebo-controlled, 2-arm study with participants assigned either to a 10 milligram (mg) or equivalent dose of balovaptan, or placebo. All other study parameters remained as stated above.
There are three parts to this study: PK Part (Study part 1) included up to 8 weeks of treatment, Main Treatment Part (Study part 2) included 24 week of treatment, and the Open Label Extension Part (Study part 3) included Week 24 to Week 76 of treatment.
All participants that completed the 24-week treatment period were eligible to participate in an optional 52-week open-label extension (OLE) during which they received balovaptan treatment.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Hoffmann-La Roche
Last refreshed: 8 February 2021
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02901431.