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A Study of Balovaptan in Adults With Autism Spectrum Disorder With a 2-Year Open-Label Extension

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03504917
Recruitment Status : Terminated (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.)
First Posted : April 20, 2018
Results First Posted : May 7, 2021
Last Update Posted : May 7, 2021
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Treatment
Condition Autism Spectrum Disorder
Interventions Drug: Balovaptan
Drug: Placebo
Enrollment 322
Recruitment Details

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.

Pre-assignment Details 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.
Arm/Group Title Balovaptan Placebo
Hide Arm/Group Description Participants received 10 mg of oral administration balovaptan once a day (QD). Participants received matching placebo in Blinded Treatment Period and 10 mg of oral administration balovaptan once a day (QD) in OLE Treatment period
Period Title: Blinded Treatment Period
Started [1] 163 158
Completed [1] 103 102
Not Completed 60 56
Reason Not Completed
Protocol Violation             1             0
Withdrawal by Subject             12             9
Lack of Efficacy             1             1
Lost to Follow-up             1             4
Adverse Event             4             4
Physician Decision             1             0
Unable due to leaving the study site, non-interest, withdrawal, and a partner inability             1             3
Study terminated by sponsor             39             34
Non-compliance with study drug             0             1
[1]
ITT Population
Period Title: Open Label Extension Treatment Period
Started [1] 100 97
Completed [2] 0 0
Not Completed 100 97
Reason Not Completed
The participant left the study site             0             1
Withdrawal by Subject             8             2
Lost to Follow-up             1             4
Lack of Efficacy             3             1
Adverse Event             7             0
Non-compliance with study drug             0             1
Study terminated by sponsor             81             88
[1]
ITT Population, 63 and 61 Participants did not continue in open-label extension in Balovaptan and Placebo Arms respectively.
[2]
ITT Population
Arm/Group Title Balovaptan Placebo Total
Hide Arm/Group Description Participants received 10 mg of oral administration balovaptan once a day (QD). Participants received matching placebo. Total of all reporting groups
Overall Number of Baseline Participants 163 158 321
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 163 participants 158 participants 321 participants
27.6  (9.7) 27.6  (9.8) 27.6  (9.7)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 163 participants 158 participants 321 participants
Female
35
  21.5%
30
  19.0%
65
  20.2%
Male
128
  78.5%
128
  81.0%
256
  79.8%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 163 participants 158 participants 321 participants
Hispanic or Latino
15
   9.2%
13
   8.2%
28
   8.7%
Not Hispanic or Latino
145
  89.0%
142
  89.9%
287
  89.4%
Unknown or Not Reported
3
   1.8%
3
   1.9%
6
   1.9%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 163 participants 158 participants 321 participants
American Indian or Alaska Native
0
   0.0%
2
   1.3%
2
   0.6%
Asian
3
   1.8%
5
   3.2%
8
   2.5%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
9
   5.5%
6
   3.8%
15
   4.7%
White
143
  87.7%
140
  88.6%
283
  88.2%
More than one race
3
   1.8%
1
   0.6%
4
   1.2%
Unknown or Not Reported
5
   3.1%
4
   2.5%
9
   2.8%
1.Primary Outcome
Title Change From Baseline at Week 24 on the Vineland Adaptive Behavior Scales (Vineland-II) Two-domain Composite (2DC) Score.
Hide Description 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.
Time Frame Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population
Arm/Group Title Balovaptan Placebo
Hide Arm/Group Description:
Participants received 10 mg of oral administration balovaptan once a day (QD).
Participants received matching placebo.
Overall Number of Participants Analyzed 111 99
Mean (Standard Deviation)
Unit of Measure: Score
4.56  (10.85) 6.83  (12.18)
2.Secondary Outcome
Title Change From Baseline at Week 12 on the Vineland-II 2DC Score
Hide Description 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.
Time Frame Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population
Arm/Group Title Balovaptan Placebo
Hide Arm/Group Description:
Participants received 10 mg of oral administration balovaptan once a day (QD).
Participants received matching placebo.
Overall Number of Participants Analyzed 150 140
Mean (Standard Deviation)
Unit of Measure: Score
3.47  (10.00) 4.85  (12.64)
3.Secondary Outcome
Title 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
Hide Description 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.
Time Frame Weeks 12 and 24
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population
Arm/Group Title Balovaptan Placebo
Hide Arm/Group Description:
Participants received 10 mg of oral administration balovaptan once a day (QD).
Participants received matching placebo.
Overall Number of Participants Analyzed 163 158
Mean (Standard Deviation)
Unit of Measure: Score
Total Score at Week 12 Number Analyzed 144 participants 124 participants
4.1  (11.2) 5.0  (10.2)
Psychosocial Health Summary Score at Week 12 Number Analyzed 144 participants 124 participants
4.9  (13.2) 5.5  (12.7)
Physical Health Summary Score at Week 12 Number Analyzed 144 participants 125 participants
2.5  (13.3) 4.3  (12.3)
Total Score at Week 24 Number Analyzed 106 participants 92 participants
8.0  (13.7) 6.0  (11.6)
Psychosocial Health Summary Score at Week 24 Number Analyzed 106 participants 92 participants
10.0  (15.4) 6.9  (14.1)
Physical Health Summary Score at Week 24 Number Analyzed 106 participants 92 participants
4.3  (15.5) 4.4  (14.5)
4.Secondary Outcome
Title Change From Baseline at Weeks 12 and 24 in the Vineland-II Adaptive Behavior Composite Standard Score
Hide Description 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.
Time Frame Weeks 12 and 24
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population
Arm/Group Title Balovaptan Placebo
Hide Arm/Group Description:
Participants received 10 mg of oral administration balovaptan once a day (QD).
Participants received matching placebo.
Overall Number of Participants Analyzed 163 157
Mean (Standard Deviation)
Unit of Measure: Score
Week 12 Number Analyzed 150 participants 140 participants
2.87  (6.99) 3.99  (10.01)
Week 24 Number Analyzed 111 participants 99 participants
4.32  (8.43) 5.26  (9.69)
5.Secondary Outcome
Title Change From Baseline at Week 12 and 24 on the Vineland-II Socialization Domain Standard Score
Hide Description

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.

Time Frame Baseline, Weeks 12 and 24
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population
Arm/Group Title Balovaptan Placebo
Hide Arm/Group Description:
Participants received 10 mg of oral administration balovaptan once a day (QD).
Participants received matching placebo.
Overall Number of Participants Analyzed 163 158
Mean (Standard Deviation)
Unit of Measure: Score
12 Week Number Analyzed 150 participants 140 participants
3.63  (11.58) 5.26  (12.71)
24 Week Number Analyzed 111 participants 99 participants
5.54  (13.54) 6.86  (11.75)
6.Secondary Outcome
Title Change From Baseline at Weeks 12 and 24 on the Vineland-II Communication Domain Standard Score
Hide Description 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.
Time Frame Weeks 12 and 24
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population
Arm/Group Title Balovaptan Placebo
Hide Arm/Group Description:
Participants received 10 mg of oral administration balovaptan once a day (QD).
Participants received matching placebo.
Overall Number of Participants Analyzed 163 158
Mean (Standard Deviation)
Unit of Measure: Score
Week 12 Number Analyzed 150 participants 140 participants
3.30  (13.74) 4.44  (16.58)
Week 24 Number Analyzed 111 participants 99 participants
3.59  (16.30) 6.81  (17.50)
7.Secondary Outcome
Title Change From Baseline at Weeks 12 and 24 on the Vineland-II Daily Living Skills Domain Standard Score
Hide Description

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.

Time Frame Weeks 12 and 24
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population
Arm/Group Title Balovaptan Placebo
Hide Arm/Group Description:
Participants received 10 mg of oral administration balovaptan once a day (QD).
Participants received matching placebo.
Overall Number of Participants Analyzed 163 158
Mean (Standard Deviation)
Unit of Measure: Score
Week 12 Number Analyzed 150 participants 140 participants
2.93  (8.44) 2.74  (9.20)
Week 24 Number Analyzed 111 participants 99 participants
5.14  (9.34) 3.02  (9.04)
8.Secondary Outcome
Title Change From Baseline in Severity of Clinical Impressions as Measured by Clinical Global Impression-Severity (CGI-S)
Hide Description 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.
Time Frame Weeks 12 and 24
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population
Arm/Group Title Balovaptan Placebo
Hide Arm/Group Description:
Participants received 10 mg of oral administration balovaptan once a day (QD).
Participants received matching placebo.
Overall Number of Participants Analyzed 147 136
Measure Type: Number
Unit of Measure: Percentage of Participants
Week 12 -3 Number Analyzed 147 participants 136 participants
0 0
Week 12 -2 Number Analyzed 147 participants 136 participants
2.0 2.2
Week 12 -1 Number Analyzed 147 participants 136 participants
21.8 25.0
Week 12 0 Number Analyzed 147 participants 136 participants
74.8 72.1
Week 12 +1 Number Analyzed 147 participants 136 participants
0.7 0.7
Week 12 +2 Number Analyzed 147 participants 136 participants
0.7 0
Week 12 +3 Number Analyzed 147 participants 136 participants
0 0
Week 24 -3 Number Analyzed 109 participants 100 participants
0 1
Week 24 -2 Number Analyzed 109 participants 100 participants
5.5 10.0
Week 24 -1 Number Analyzed 109 participants 100 participants
27.5 20.0
Week 24 0 Number Analyzed 109 participants 100 participants
66.1 68.0
Week 24 +1 Number Analyzed 109 participants 100 participants
0.9 1.0
Week 24 +2 Number Analyzed 109 participants 100 participants
0 0
Week 24 +3 Number Analyzed 109 participants 100 participants
0 0
9.Secondary Outcome
Title Improvements in Clinical Impressions, as Measured by Clinical Global Impression-Improvement (CGI-I)
Hide Description 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.
Time Frame Weeks 12 and 24
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population
Arm/Group Title Balovaptan Placebo
Hide Arm/Group Description:
Participants received 10 mg of oral administration balovaptan once a day (QD).
Participants received matching placebo.
Overall Number of Participants Analyzed 148 136
Measure Type: Number
Unit of Measure: Percentage of Participants
Week 12 1 Number Analyzed 148 participants 136 participants
0 0
Week 12 2 Number Analyzed 148 participants 136 participants
10.1 14.7
Week 12 3 Number Analyzed 148 participants 136 participants
36.5 43.4
Week 12 4 Number Analyzed 148 participants 136 participants
50.0 41.2
Week 12 5 Number Analyzed 148 participants 136 participants
3.4 0.7
Week 12 6 Number Analyzed 148 participants 136 participants
0 0
Week 12 7 Number Analyzed 148 participants 136 participants
0 0
Week 24 1 Number Analyzed 109 participants 100 participants
0 2
Week 24 2 Number Analyzed 109 participants 100 participants
15.6 24.0
Week 24 3 Number Analyzed 109 participants 100 participants
44.0 40.0
Week 24 4 Number Analyzed 109 participants 100 participants
38.5 33.0
Week 24 5 Number Analyzed 109 participants 100 participants
0.9 1.0
Week 24 6 Number Analyzed 109 participants 100 participants
0.9 0
Week 24 7 Number Analyzed 109 participants 100 participants
0 0
10.Secondary Outcome
Title Change From Baseline at Weeks 12 and 24 in the Hamilton Anxiety Rating Scale (HAM-A) Total and Domain Scores
Hide Description 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.
Time Frame Weeks 12 and 24
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population
Arm/Group Title Balovaptan Placebo
Hide Arm/Group Description:
Participants received 10 mg of oral administration balovaptan once a day (QD).
Participants received matching placebo.
Overall Number of Participants Analyzed 163 158
Mean (Standard Deviation)
Unit of Measure: Score
Week 12 Total Number Analyzed 153 participants 143 participants
-1.7  (4.9) -2.8  (4.4)
Week 12 Psychic Anxiety Subscale Number Analyzed 153 participants 143 participants
-1.3  (3.5) -1.8  (3.2)
Week 12 Somatic Anxiety Subscale Number Analyzed 153 participants 143 participants
-0.4  (2.4) -1.0  (2.5)
Week 24 Total Number Analyzed 115 participants 104 participants
-2.7  (4.5) -2.8  (5.7)
Week 24 Psychic Anxiety Subscale Number Analyzed 115 participants 104 participants
-2.1  (3.4) -1.8  (3.9)
Week 24 Somatic Anxiety Subscale Number Analyzed 115 participants 104 participants
-0.6  (2.2) -0.1  (2.9)
11.Secondary Outcome
Title Proportion of Subjects With a >=6-point Improvement in Vineland-II 2DC Score
Hide Description

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

Time Frame Weeks 12 and 24
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population
Arm/Group Title Balovaptan Placebo
Hide Arm/Group Description:
Participants received 10 mg of oral administration balovaptan once a day (QD).
Participants received matching placebo.
Overall Number of Participants Analyzed 128 114
Measure Type: Number
Unit of Measure: Percentage of Participants
Week 12 >=6 Number Analyzed 128 participants 114 participants
34.4 42.1
Week 24 >=6 Number Analyzed 100 participants 93 participants
43 48.4
12.Secondary Outcome
Title Percentage of Participants With Adverse Events
Hide Description

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.

Time Frame Week 24 and Up to Approximately 2 Years
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Hide Analysis Population Description
Safety Population
Arm/Group Title Balovaptan Treatment Placebo Treatment Balovaptan OLE Placebo OLE
Hide Arm/Group Description:
Participants received 10 mg of oral administration balovaptan once a day (QD). Blinded Treatment Period
Participants received matching placebo. Blinded Treatment Period
Participants received 10 mg of oral administration balovaptan once a day (QD). OLE Treatment Period
Participants received matching placebo in Blinded Treatment Period and 10 mg of oral administration balovaptan once a day (QD). OLE Treatment Period
Overall Number of Participants Analyzed 163 158 100 97
Measure Type: Number
Unit of Measure: Percentage of Participants
60.1 65.8 59.0 55.7
Time Frame From baseline to the end of Safety Period, up to 2 years
Adverse Event Reporting Description

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.

 
Arm/Group Title Balovaptan in Blinded Treatment Period Placebo Blinded Treatment Period Balovaptan in Open Label Extension Treatment Period Placebo in Open Label Extension Treatment Period
Hide Arm/Group Description Participants received 10 mg of oral administration balovaptan once a day (QD). Participants received matching placebo. Participants received 10 mg of oral administration balovaptan once a day (QD). Participants received matching placebo in Blinded Treatment Period and 10 mg of oral administration balovaptan once a day (QD). OLE Treatment Period
All-Cause Mortality
Balovaptan in Blinded Treatment Period Placebo Blinded Treatment Period Balovaptan in Open Label Extension Treatment Period Placebo in Open Label Extension Treatment Period
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/163 (0.00%)      1/158 (0.63%)      0/100 (0.00%)      0/97 (0.00%)    
Hide Serious Adverse Events
Balovaptan in Blinded Treatment Period Placebo Blinded Treatment Period Balovaptan in Open Label Extension Treatment Period Placebo in Open Label Extension Treatment Period
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   2/163 (1.23%)      5/158 (3.16%)      0/100 (0.00%)      2/97 (2.06%)    
Gastrointestinal disorders         
Colitis * 1  0/163 (0.00%)  0 1/158 (0.63%)  1 0/100 (0.00%)  0 0/97 (0.00%)  0
Infections and infestations         
Abscess limb * 1  0/163 (0.00%)  0 1/158 (0.63%)  1 0/100 (0.00%)  0 0/97 (0.00%)  0
Urosepsis * 1  0/163 (0.00%)  0 1/158 (0.63%)  1 0/100 (0.00%)  0 0/97 (0.00%)  0
Urinary tract infection * 1  0/163 (0.00%)  0 0/158 (0.00%)  0 0/100 (0.00%)  0 1/97 (1.03%)  1
Pharyngitis streptococcal * 1  0/163 (0.00%)  0 0/158 (0.00%)  0 0/100 (0.00%)  0 1/97 (1.03%)  1
Sepsis * 1  0/163 (0.00%)  0 0/158 (0.00%)  0 0/100 (0.00%)  0 1/97 (1.03%)  1
Injury, poisoning and procedural complications         
Femur fracture * 1  0/163 (0.00%)  0 0/158 (0.00%)  0 0/100 (0.00%)  0 1/97 (1.03%)  1
Psychiatric disorders         
Panic disorder * 1  0/163 (0.00%)  0 1/158 (0.63%)  1 0/100 (0.00%)  0 0/97 (0.00%)  0
Completed suicide * 1  0/163 (0.00%)  0 1/158 (0.63%)  1 0/100 (0.00%)  0 0/97 (0.00%)  0
Schizoaffective disorder * 1  1/163 (0.61%)  2 0/158 (0.00%)  0 0/100 (0.00%)  0 0/97 (0.00%)  0
Suicidal ideation * 1  1/163 (0.61%)  1 1/158 (0.63%)  1 0/100 (0.00%)  0 0/97 (0.00%)  0
1
Term from vocabulary, MedDRA v23.0
*
Indicates events were collected by non-systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Balovaptan in Blinded Treatment Period Placebo Blinded Treatment Period Balovaptan in Open Label Extension Treatment Period Placebo in Open Label Extension Treatment Period
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   49/163 (30.06%)      59/158 (37.34%)      28/100 (28.00%)      30/97 (30.93%)    
Gastrointestinal disorders         
Diarrhoea * 1  11/163 (6.75%)  15 14/158 (8.86%)  15 5/100 (5.00%)  5 4/97 (4.12%)  4
Nausea * 1  4/163 (2.45%)  4 7/158 (4.43%)  8 1/100 (1.00%)  1 5/97 (5.15%)  5
Infections and infestations         
Nasopharyngitis * 1  14/163 (8.59%)  14 19/158 (12.03%)  22 7/100 (7.00%)  10 4/97 (4.12%)  4
Upper respiratory tract infection * 1  10/163 (6.13%)  13 9/158 (5.70%)  11 6/100 (6.00%)  7 7/97 (7.22%)  11
Gastroenteritis * 1  2/163 (1.23%)  2 0/158 (0.00%)  0 1/100 (1.00%)  1 5/97 (5.15%)  5
Nervous system disorders         
Dizziness * 1  2/163 (1.23%)  2 10/158 (6.33%)  10 2/100 (2.00%)  2 1/97 (1.03%)  1
Headache * 1  8/163 (4.91%)  13 7/158 (4.43%)  9 5/100 (5.00%)  5 10/97 (10.31%)  17
Psychiatric disorders         
Anxiety * 1  8/163 (4.91%)  11 7/158 (4.43%)  7 8/100 (8.00%)  8 3/97 (3.09%)  4
Insomnia * 1  5/163 (3.07%)  5 8/158 (5.06%)  8 3/100 (3.00%)  3 3/97 (3.09%)  3
Respiratory, thoracic and mediastinal disorders         
Oropharyngeal pain * 1  5/163 (3.07%)  5 8/158 (5.06%)  8 0/100 (0.00%)  0 3/97 (3.09%)  3
1
Term from vocabulary, MedDRA v23.0
*
Indicates events were collected by non-systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Reference Study ID Number: WN39434
Organization: www.roche.com/about_roche/roche_worldwide.htm
Phone: 888-662-6728 (U.S. Only)
EMail: global-roche-genentech-trials@gene.com
Layout table for additonal information
Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT03504917    
Other Study ID Numbers: WN39434
First Submitted: April 12, 2018
First Posted: April 20, 2018
Results First Submitted: March 2, 2021
Results First Posted: May 7, 2021
Last Update Posted: May 7, 2021