A Phase 2 interventional study of Placebo and RO7017773 in Autism Spectrum Disorder (ASD), sponsored by Hoffmann-La Roche. Completed at 21 sites in 4 countries. Open to participants aged 15 Years to 45 Years. Per ClinicalTrials.gov, last updated 2025-01-28.
Sponsored by Hoffmann-La Roche · Phase 2, Interventional, and Treatment
This study will investigate the efficacy, safety, tolerability, and pharmacokinetics of RO7017773 in participants aged 15-45 years who have been diagnosed with ASD with a score of >/=50 on the Wechsler Abreviated Scale of Intelligence (WASI-II).
1,344 studies on the registry are indexed under Autistic Disorder; 334 are open to participants now.
This study's enrollment of 104 is above the median of 45 across 1,044 interventional studies indexed under Autistic Disorder.
Browse Autistic Disorder studies →Hoffmann-La Roche is the lead sponsor of 2,061 studies on the registry; 85 are open to participants now.
Of its 319 completed or terminated interventional studies of FDA-regulated products, 239 (75%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria
Neurologic/Psychiatric Conditions:
Other Conditions:
Prior/Concurrent Clinical Study Experience:
Other Exculsions:
Participants will receive placebo matched to RO7017773 for approximately 12 weeks.
Drug: Placebo
Participants will receive a fixed low dose of RO7017773 for approximately 12 weeks.
Drug: RO7017773
Participants will receive a fixed high dose of RO7017773 for approximately 12 weeks.
Drug: RO7017773
Participants will receive oral placebo for approximately 12 weeks.
Participants will receive oral RO7017773 for approximately 12 weeks.
Change From Baseline to Week 12 in the Adaptive Behavior Composite (ABC) Score of the Vineland Adaptive Behavior Scales, Third Edition (Vineland-3)
Vineland-3 is a semi-structured interview that measures an individual's adaptive behavior across 3 domains: Communication, Socialization, and Daily Living skills. Each domain is composed of 3 subdomains. Subdomain raw score is based on item responses (3-point scale: 0=never present; 1=sometimes present; 2=usually present) and is calculated for each subdomain of the three main domains as the sum of the scores for each item within the subdomain. Raw scores of the 9 subdomains are used to derive Growth Scale Values (GSVs; range = 10-197). A conversion table for mapping raw scores to GSV scores is found in Appendix 3, Table B.2 in the Vineland-3 manual (Sparrow et al. 2016). GSV is a person-ability score used to track an individual's progress. Vineland-3 ABC Composite GSV score is calculated as the mean GSV score (summing the 9 GSV subdomain scores and dividing by 9; Vineland-3 ABC Composite GSV scores can range from 10-154). A higher score indicates better adaptive functioning.
Time frame: Baseline to Week 12
Number of Participants With at Least One Adverse Events (AEs)
An AE is an untoward medical occurrence in a participant administered a pharmaceutical product and regardless of the causal relationship with the treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom/disease temporally associated with the use of an investigational product, whether or not considered related to the investigational product.
Time frame: Up to Week 18
Number of Participants With at Least One Serious Adverse Events (SAEs)
An AE is an untoward medical occurrence in a participant administered a pharmaceutical product and regardless of causal relationship with the treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom/disease temporally associated with the use of an investigational product, whether or not considered related to investigational product. A SAE is any significant hazard, contraindication, side effect that is fatal or life-threatening, requires hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, is medically significant or requires intervention to prevent one or other of the outcomes listed above.
Time frame: Up to Week 18
Number of Participants Discontinuing Treatment Due to AEs
An AE is an untoward medical occurrence in a participant administered a pharmaceutical product and regardless of the causal relationship with the treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom/disease temporally associated with the use of an investigational product, whether or not considered related to the investigational product.
Time frame: Day 1 up to Week 12
Number of Participants With Post-baseline Suicidal Ideation or Suicidal Behaviour as Measured Using the Columbia-Suicide-Severity Rating Scale (C-SSRS)
C-SSRS=assessment tool used to assess lifetime suicidality of participant (at baseline) as well as any new instances of suicidality (C-SSRS since last visit). Structured interview prompts recollection of suicidal ideation, including intensity of ideation, behavior, and attempts with actual/potential lethality. Categories have binary responses (yes/no) and include Wish to be Dead; Non-specific Active Suicidal Thoughts; Active Suicidal Ideation with Any Methods (Not Plan) without Intent to Act; Active Suicidal Ideation with Some Intent to Act, without Specific Plan; Active Suicidal Ideation with Specific Plan and Intent, Preparatory Acts and Behavior; Aborted Attempt; Interrupted Attempt; Actual Attempt (non-fatal); Completed Suicide. Suicidal ideation/behavior is indicated by a "yes" answer to any of the listed categories. Score of 0 is assigned if no suicide risk is present. Score of 1 or higher= suicidal ideation or behavior. Categories with non-zero values are only reported here.
Time frame: Baseline up to Week 18
Change From Baseline in Karolinska Sleepiness Scale (KSS) Score for Assessing Daytime Sleepiness
The KSS measures the subjective level of sleepiness at a particular time during the day. On this scale, participants (or support persons for adolescents aged 15 to 17 years and low-functioning participants) indicate which level best reflects the psycho-physical state experienced in the last 5 minutes. The KSS is a 9-point scale (1=extremely alert, 9=very sleepy, great effort to keep awake, fighting sleep). A decrease in KSS score or negative change from baseline indicate an improvement in sleepiness.
Time frame: Baseline (Day 1 Predose), 3-4 hours post-dose on Day 1, Predose and 3-4 hours post-dose on Days 14, 42, and 84
Change From Baseline in Epworth Sleepiness Scale Score (ESS) for Assessing Daytime Sleepiness
The ESS is a brief, self-administered eight-item questionnaire that measures daytime sleepiness in adults. Participants were asked to rate on a scale of 0-3 the chances that, "over the past month" and "since last visit", he/she would have dozed in eight specific situations that are commonly met in daily life (0 = would never doze and 3 = high chance of dozing). The ESS score is the sum of eight item-scores and can range from 0 to 24. A lower ESS score or a negative change from baseline score indicates an improvement in daytime sleepiness.
Time frame: Baseline (Day 1), Days 14, 42, and 84
Change From Baseline ESS Score for Children and Adolescents (ESS-CHAD) for Assessing Daytime Sleepiness
The ESS-CHAD is a brief, support person-administered eight-item questionnaire that measures daytime sleepiness in children and adolescents. Each item asked the support persons of adolescents and participants with an IQ score \<70 to rate on a scale of 0-3 the chances that "Over the past month," and "since last visit", "your child" would have dozed in eight specific situations that are commonly met in daily life ( 0 to 3 where 0 = would never doze and 3 = high chance of dozing). The ESS score is the sum of eight item scores and can range from 0 to 24. A lower ESS score or negative change from baseline score indicates an improvement in daytime sleepiness.
Time frame: Baseline (Day 1), Days 14, 42, 63, and 84
Number of Participants With Daytime Sleepiness Assessed Using Sudden Onset of Sleep Questionnaire
A sleep questionnaire was developed specifically for this study. Each participant (or support person for adolescents aged 15 to 17 years and for low-functioning participants) was asked to answer a series of 8 questions. The questions and their corresponding responses are as follows: a. Have you ever fallen asleep or have you been likely to fall asleep during your waking time? (Yes/No); b. Was this episode? (Gradual with awareness/Sudden and unpredictable/Sudden with awareness); c. Of the recent episode, how often does this occur? (Every day/Less frequently/Once a week/Other); d. Do you feel worried about falling asleep during the day? (Yes/No); e. Did the episode (or episodes) disrupt your daily activities? (Considerably/Marginally/No); f. Did this episode (or episodes) disrupt your social life (Considerably/Marginally/No); g. In the case of such an episode, was awakening? (Difficult/Easy/Normal). Categories with non-zero values are only reported here.
Time frame: Baseline (Day 1), Days 7, 14, 42, 63, and 84
Change From Baseline to Week 12 in Behavior/Symptoms as Measured by All Domains of the Repetitive Behavior Scale-Revised (RBS-R) Score
The RBS-R is a 43-item informant-based questionnaire, assessing the variety of restricted and repetitive behaviors (RRBs) in individuals with ASD. The scale is grouped into six subscales: Stereotyped, Self-Injurious, Compulsive, Ritualistic, Sameness, and Restricted Behaviors. For each item, behaviors are rated on a 4-point scale: 0-Behavior does not occur, 1-Behavior occurs and is a mild problem, 2-Behavior occurs and is a moderate problem, 3-Behavior occurs and is a severe problem. A total RBS-R score is calculated as the sum of the scores for the 43 items. The total score ranges from 0 to 129 and higher scores are indicative of more severe RRBs.
Time frame: Baseline to Week 12
Change From Baseline to Week 12 on the Vineland-3 Socialization Domain
Vineland-3 is a semi-structured interview measuring an individual's adaptive behavior across 3 domains: Communication, Socialization \& Daily Living skills. Each domain consists of 3 subdomains. Subdomain raw scores are based on item responses (3-point scale: 0=never present; 1=sometimes present; 2=usually present) \& are calculated for each subdomain of Socialization (interpersonal relationships, play and leisure time, coping skills) domain as sum of the scores for each item in the subdomain. Raw scores for the 3 Socialization subdomains are used to derive GSVs (range=10-164). A conversion table for mapping raw scores to GSV scores is found in the Vineland-3 manual (Sparrow et al. 2016). GSV is a person-ability score used to track an individual's progress. Vineland-3 Socialization Domain GSV score is calculated as a mean GSV score (summing the 3 GSV subdomain scores \& dividing by 3). Vineland-3 Socialization Domain GSV score range = 10-145. Higher score =better adaptive functioning.
Time frame: Baseline to Week 12
Change From Baseline to Week 12 on the Vineland-3 Communication Domain
Vineland-3 is a semi-structured interview measuring an individual's adaptive behavior across 3 domains: Communication, Socialization \& Daily Living skills. Each domain consists of 3 subdomains. Subdomain raw scores are based on item responses (3-point scale: 0=never present; 1=sometimes present; 2=usually present) \& is calculated for each subdomain of the Communication (receptive, expressive, written) domain as the sum of the scores for each item within the subdomain. Raw scores for each of the 3 Communication subdomains are used to derive Growth Scale Values (GSVs; range from 10-197). A conversion table for mapping raw scores to GSV scores is found in Vineland-3 manual (Sparrow et al. 2016). GSV is a person-ability score used to track an individual's progress. Vineland-3 Socialization Domain GSV score is calculated as mean GSV score (summing the 3 GSV subdomain scores \& dividing by 3). Vineland-3 Socialization Domain GSV score range=10-174. Higher score=better adaptive functioning.
Time frame: Baseline to Week 12
Participants took part in the study across 26 investigative sites in 4 countries (United States, Canada, Spain, and Italy) from 31 March 2021 to 15 May 2024.
| Milestone | Placebo | Alogabat 20 mg | Alogabat 60 mg |
|---|---|---|---|
| Started | 34 | 34 | 36 |
| Completed | 32 | 30 | 29 |
| Not completed | 2 | 4 | 7 |
| Withdrew: Adverse event | 1 | 0 | 2 |
| Withdrew: Non-compliance with study drug | 1 | 0 | 0 |
| Withdrew: Reason not specified | 0 | 1 | 3 |
| Withdrew: Physician decision | 0 | 1 | 1 |
| Withdrew: Withdrawal by subject | 0 | 2 | 1 |
Vineland-3 is a semi-structured interview that measures an individual's adaptive behavior across 3 domains: Communication, Socialization, and Daily Living skills. Each domain is composed of 3 subdomains. Subdomain raw score is based on item responses (3-point scale: 0=never present; 1=sometimes present; 2=usually present) and is calculated for each subdomain of the three main domains as the sum of the scores for each item within the subdomain. Raw scores of the 9 subdomains are used to derive Growth Scale Values (GSVs; range = 10-197). A conversion table for mapping raw scores to GSV scores is found in Appendix 3, Table B.2 in the Vineland-3 manual (Sparrow et al. 2016). GSV is a person-ability score used to track an individual's progress. Vineland-3 ABC Composite GSV score is calculated as the mean GSV score (summing the 9 GSV subdomain scores and dividing by 9; Vineland-3 ABC Composite GSV scores can range from 10-154). A higher score indicates better adaptive functioning.
| score on a scale | Placebo | Alogabat 20 mg | Alogabat 60 mg |
|---|---|---|---|
| Change From Baseline to Week 12 in the Adaptive Behavior Composite (ABC) Score of the Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) | 3.250 (2.011 to 4.490) | 2.819 (1.469 to 4.169) | 2.807 (1.499 to 4.115) |
An AE is an untoward medical occurrence in a participant administered a pharmaceutical product and regardless of the causal relationship with the treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom/disease temporally associated with the use of an investigational product, whether or not considered related to the investigational product.
| Participants | Placebo | Alogabat 20 mg | Alogabat 60 mg |
|---|---|---|---|
| Number of Participants With at Least One Adverse Events (AEs) | 24 | 22 | 22 |
An AE is an untoward medical occurrence in a participant administered a pharmaceutical product and regardless of causal relationship with the treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom/disease temporally associated with the use of an investigational product, whether or not considered related to investigational product. A SAE is any significant hazard, contraindication, side effect that is fatal or life-threatening, requires hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, is medically significant or requires intervention to prevent one or other of the outcomes listed above.
| Participants | Placebo | Alogabat 20 mg | Alogabat 60 mg |
|---|---|---|---|
| Number of Participants With at Least One Serious Adverse Events (SAEs) | 0 | 0 | 0 |
An AE is an untoward medical occurrence in a participant administered a pharmaceutical product and regardless of the causal relationship with the treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom/disease temporally associated with the use of an investigational product, whether or not considered related to the investigational product.
| Participants | Placebo | Alogabat 20 mg | Alogabat 60 mg |
|---|---|---|---|
| Number of Participants Discontinuing Treatment Due to AEs | 1 | 0 | 2 |
C-SSRS=assessment tool used to assess lifetime suicidality of participant (at baseline) as well as any new instances of suicidality (C-SSRS since last visit). Structured interview prompts recollection of suicidal ideation, including intensity of ideation, behavior, and attempts with actual/potential lethality. Categories have binary responses (yes/no) and include Wish to be Dead; Non-specific Active Suicidal Thoughts; Active Suicidal Ideation with Any Methods (Not Plan) without Intent to Act; Active Suicidal Ideation with Some Intent to Act, without Specific Plan; Active Suicidal Ideation with Specific Plan and Intent, Preparatory Acts and Behavior; Aborted Attempt; Interrupted Attempt; Actual Attempt (non-fatal); Completed Suicide. Suicidal ideation/behavior is indicated by a "yes" answer to any of the listed categories. Score of 0 is assigned if no suicide risk is present. Score of 1 or higher= suicidal ideation or behavior. Categories with non-zero values are only reported here.
| Participants | Placebo | Alogabat 20 mg | Alogabat 60 mg |
|---|---|---|---|
| Wish to be Dead | 2 | 1 | 1 |
| Non-specific Active Suicidal Thoughts | 1 | 1 | 1 |
| Self-Injurious Behavior Without Suicidal Intent | 1 | 0 | 1 |
The KSS measures the subjective level of sleepiness at a particular time during the day. On this scale, participants (or support persons for adolescents aged 15 to 17 years and low-functioning participants) indicate which level best reflects the psycho-physical state experienced in the last 5 minutes. The KSS is a 9-point scale (1=extremely alert, 9=very sleepy, great effort to keep awake, fighting sleep). A decrease in KSS score or negative change from baseline indicate an improvement in sleepiness.
| score on a scale | Placebo | Alogabat 20 mg | Alogabat 60 mg |
|---|---|---|---|
| Baseline (Day 1: Predose) | 4.97 ± 2.10 | 4.88 ± 2.28 | 4.17 ± 2.37 |
| Change from Baseline at Day 1: 3-4 hours Post Dose | -0.88 ± 2.00 | -0.58 ± 2.12 | 0.19 ± 2.20 |
| Change from Baseline at Day 14: Predose | -0.03 ± 1.91 | -0.13 ± 2.12 | -0.03 ± 2.15 |
| Change from Baseline at Day 14: 3-4 hours Post Dose | -0.76 ± 1.70 | -0.84 ± 2.60 | 0.34 ± 2.72 |
| Change from Baseline at Day 42: Predose | -0.50 ± 2.05 | -0.94 ± 2.66 | -0.94 ± 1.82 |
| Change from Baseline at Day 42: 3-4 hours Post Dose | -0.74 ± 1.77 | -1.00 ± 2.79 | -0.26 ± 2.34 |
| Change from Baseline at Day 84: Predose | -1.52 ± 2.23 | -0.83 ± 2.65 | -0.48 ± 2.13 |
| Change from Baseline at Day 84: 3-4 hours Post Dose | -1.13 ± 2.00 | -0.83 ± 2.80 | -0.30 ± 2.45 |
The ESS is a brief, self-administered eight-item questionnaire that measures daytime sleepiness in adults. Participants were asked to rate on a scale of 0-3 the chances that, "over the past month" and "since last visit", he/she would have dozed in eight specific situations that are commonly met in daily life (0 = would never doze and 3 = high chance of dozing). The ESS score is the sum of eight item-scores and can range from 0 to 24. A lower ESS score or a negative change from baseline score indicates an improvement in daytime sleepiness.
| score on a scale | Placebo | Alogabat 20 mg | Alogabat 60 mg |
|---|---|---|---|
| Baseline (Day 1) | 5.00 ± 3.69 | 6.15 ± 4.23 | 4.54 ± 2.67 |
| Change from Baseline at Day 14 | 0.24 ± 2.42 | -0.72 ± 3.36 | 0.73 ± 4.58 |
| Change from Baseline at Day 42 | 0.62 ± 2.52 | -2.08 ± 4.15 | 0.50 ± 3.39 |
| Change from Baseline at Day 84 | 0.38 ± 3.40 | -1.46 ± 4.28 | 0.26 ± 3.41 |
The ESS-CHAD is a brief, support person-administered eight-item questionnaire that measures daytime sleepiness in children and adolescents. Each item asked the support persons of adolescents and participants with an IQ score \<70 to rate on a scale of 0-3 the chances that "Over the past month," and "since last visit", "your child" would have dozed in eight specific situations that are commonly met in daily life ( 0 to 3 where 0 = would never doze and 3 = high chance of dozing). The ESS score is the sum of eight item scores and can range from 0 to 24. A lower ESS score or negative change from baseline score indicates an improvement in daytime sleepiness.
| score on a scale | Placebo | Alogabat 20 mg | Alogabat 60 mg |
|---|---|---|---|
| Baseline (Day 1) | 4.20 ± 3.83 | 6.20 ± 1.30 | 6.86 ± 4.53 |
| Change from Baseline at Day 14 | -1.00 ± 2.92 | -0.40 ± 3.36 | -1.00 ± 2.28 |
| Change from Baseline at Day 42 | -1.40 ± 2.70 | -2.00 ± 2.16 | 1.00 ± 2.92 |
| Change from Baseline at Day 63 | -1.00 ± 1.41 | -2.50 ± 0.71 | -2.00 ± 1.41 |
| Change from Baseline at Day 84 | 1.40 ± 4.51 | -1.25 ± 4.19 | -1.50 ± 1.29 |
A sleep questionnaire was developed specifically for this study. Each participant (or support person for adolescents aged 15 to 17 years and for low-functioning participants) was asked to answer a series of 8 questions. The questions and their corresponding responses are as follows: a. Have you ever fallen asleep or have you been likely to fall asleep during your waking time? (Yes/No); b. Was this episode? (Gradual with awareness/Sudden and unpredictable/Sudden with awareness); c. Of the recent episode, how often does this occur? (Every day/Less frequently/Once a week/Other); d. Do you feel worried about falling asleep during the day? (Yes/No); e. Did the episode (or episodes) disrupt your daily activities? (Considerably/Marginally/No); f. Did this episode (or episodes) disrupt your social life (Considerably/Marginally/No); g. In the case of such an episode, was awakening? (Difficult/Easy/Normal). Categories with non-zero values are only reported here.
| Participants | Placebo | Alogabat 20 mg | Alogabat 60 mg |
|---|---|---|---|
| a. Baseline: Day 1 (No) | 22 | 28 | 29 |
| a. Baseline: Day 1 (Yes) | 11 | 6 | 7 |
| a. Day 7 (No) | 30 | 25 | 30 |
| a. Day 7 (Yes) | 1 | 8 | 2 |
| a. Day 14 (No) | 22 | 26 | 26 |
| a. Day 14 (Yes) | 8 | 6 | 8 |
| a. Day 42 (No) | 26 | 29 | 28 |
| a. Day 42 (Yes) | 6 | 3 | 4 |
| a. Day 63 (No) | 3 | 2 | 1 |
| a. Day 63 (Yes) | 0 | 0 | 1 |
| a. Day 84 (No) | 26 | 30 | 27 |
| a. Day 84 (Yes) | 7 | 1 | 4 |
| b. Baseline: Day 1 (Gradual with Awareness) | 10 | 5 | 6 |
| b. Baseline: Day 1 (Sudden and Unpredictable (Without Awareness)) | 1 | 0 | 0 |
| b. Baseline: Day 1 (Sudden with Awareness) | 1 | 1 | 1 |
| b. Day 7 (Gradual with Awareness) | 1 | 7 | 1 |
| b. Day 7 (Sudden and Unpredictable (Without Awareness)) | 0 | 1 | 0 |
| b. Day 7 (Sudden with Awareness) | 0 | 0 | 1 |
| b. Day 14 (Gradual with Awareness) | 10 | 5 | 5 |
| b. Day 14 (Sudden and Unpredictable (Without Awareness)) | 0 | 2 | 0 |
| b. Day 14 (Sudden with Awareness) | 0 | 0 | 3 |
| b. Day 42 (Gradual with Awareness) | 6 | 3 | 2 |
| b. Day 42 (Sudden with Awareness) | 1 | 0 | 2 |
| b. Day 63 (Gradual with Awareness) | 1 | — | 0 |
| b. Day 63 (Sudden with Awareness) | 0 | — | 1 |
| b. Day 84 (Gradual with Awareness) | 7 | 1 | 3 |
| b. Day 84 (Sudden and Unpredictable (Without Awareness)) | 0 | 0 | 1 |
| b. Day 84 (Sudden with Awareness) | 0 | 0 | 1 |
| c. Baseline: Day 1 (Everyday) | 5 | 1 | 1 |
| c. Baseline: Day 1 (Less Frequently) | 1 | 1 | 2 |
| c. Baseline: Day 1 (Once a Week) | 4 | 4 | 3 |
| c. Baseline: Day 1 (Other) | 0 | 0 | 1 |
| c. Day 7 (Everyday) | 0 | 0 | 1 |
| c. Day 7 (Less Frequently) | 0 | 2 | 1 |
| c. Day 7 (Once a Month) | 0 | 1 | 0 |
| c. Day 7 (Once a Week) | 1 | 1 | 0 |
| c. Day 7 (Other) | 0 | 4 | 0 |
| c. Day 14 (Everyday) | 2 | 0 | 4 |
| c. Day 14 (Less Frequently) | 0 | 3 | 0 |
| c. Day 14 (Once a Week) | 6 | 2 | 2 |
| c. Day 14 (Other) | 2 | 2 | 2 |
| c. Day 42 (Everyday) | 3 | 0 | 2 |
| c. Day 42 (Less Frequently) | 1 | 0 | 2 |
| c. Day 42 (Once a Month) | 0 | 1 | 0 |
| c. Day 42 (Once a Week) | 1 | 2 | 0 |
| c. Day 42 (Other) | 1 | 0 | 0 |
| c. Day 63 (Everyday) | 1 | — | 1 |
| c. Day 84 (Everyday) | 0 | 0 | 2 |
| c. Day 84 (Less Frequently) | 3 | 0 | 0 |
| c. Day 84 (Once a Week) | 1 | 1 | 2 |
| c. Day 84 (Other) | 3 | 0 | 0 |
| d. Baseline: Day 1 (No) | 5 | 3 | 7 |
| d. Baseline: Day 1 (Yes) | 7 | 4 | 0 |
| d. Day 7 (No) | 0 | 6 | 2 |
| d. Day 7 (Yes) | 1 | 2 | 0 |
| d. Day 14 (No) | 8 | 6 | 8 |
| d. Day 14 (Yes) | 2 | 1 | 0 |
| d. Day 42 (No) | 4 | 1 | 3 |
| d. Day 42 (Yes) | 2 | 2 | 1 |
| d. Day 63 (No) | 0 | — | 1 |
| d. Day 63 (Yes) | 1 | — | 0 |
| d. Day 84 (No) | 4 | 0 | 4 |
| d. Day 84 (Yes) | 3 | 1 | 0 |
| e. Baseline: Day 1 (Considerably) | 0 | 1 | 0 |
| e. Baseline: Day 1 (Marginally) | 8 | 5 | 1 |
| e. Baseline: Day 1 (No) | 4 | 1 | 6 |
| e. Day 7 (Considerably) | 0 | 1 | 1 |
| e. Day 7 (Marginally) | 1 | 1 | 0 |
| e. Day 7 (No) | 0 | 6 | 1 |
| e. Day 14 (Considerably) | 0 | 1 | 1 |
| e. Day 14 (Marginally) | 3 | 1 | 2 |
| e. Day 14 (No) | 7 | 5 | 5 |
| e. Day 42 (Marginally) | 1 | 0 | 0 |
| e. Day 42 (No) | 5 | 3 | 4 |
| e. Day 63 (Marginally) | 1 | — | 0 |
| e. Day 63 (No) | 0 | — | 1 |
| e. Day 84 (Considerably) | 0 | 0 | 1 |
| e. Day 84 (Marginally) | 2 | 0 | 1 |
| e. Day 84 (No) | 5 | 1 | 2 |
| f. Baseline: Day 1 (Marginally) | 3 | 3 | 1 |
| f. Baseline: Day 1 (No) | 9 | 4 | 6 |
| f. Day 7 (Considerably) | 0 | 0 | 1 |
| f. Day 7 (Marginally) | 0 | 1 | 0 |
| f. Day 7 (No) | 1 | 7 | 1 |
| f. Day 14 (Considerably) | 1 | 0 | 2 |
| f. Day 14 (Marginally) | 0 | 1 | 1 |
| f. Day 14 (No) | 9 | 6 | 5 |
| f. Day 42 (Considerably) | 0 | 0 | 1 |
| f. Day 42 (Marginally) | 0 | 0 | 1 |
| f. Day 42 (No) | 6 | 3 | 2 |
| f. Day 63 (No) | 1 | — | 1 |
| f. Day 84 (Considerably) | 0 | 0 | 1 |
| f. Day 84 (Marginally) | 1 | 0 | 0 |
| f. Day 84 (No) | 6 | 1 | 3 |
| g. Baseline: Day 1 (Difficult) | 1 | 0 | 0 |
| g. Baseline: Day 1 (Easy) | 7 | 5 | 4 |
| g. Baseline: Day 1 (Normal) | 4 | 2 | 3 |
| g. Day 7 (Easy) | 1 | 5 | 1 |
| g. Day 7 (Normal) | 0 | 3 | 1 |
| g. Day 14 (Easy) | 6 | 5 | 3 |
| g. Day 14 (Normal) | 4 | 2 | 5 |
| g. Day 42 (Easy) | 4 | 3 | 3 |
| g. Day 42 (Normal) | 2 | 0 | 1 |
| g. Day 63 (Easy) | 1 | — | 0 |
| g. Day 63 (Normal) | 0 | — | 1 |
| g. Day 84 (Easy) | 6 | 1 | 2 |
| g. Day 84 (Normal) | 1 | 0 | 2 |
The RBS-R is a 43-item informant-based questionnaire, assessing the variety of restricted and repetitive behaviors (RRBs) in individuals with ASD. The scale is grouped into six subscales: Stereotyped, Self-Injurious, Compulsive, Ritualistic, Sameness, and Restricted Behaviors. For each item, behaviors are rated on a 4-point scale: 0-Behavior does not occur, 1-Behavior occurs and is a mild problem, 2-Behavior occurs and is a moderate problem, 3-Behavior occurs and is a severe problem. A total RBS-R score is calculated as the sum of the scores for the 43 items. The total score ranges from 0 to 129 and higher scores are indicative of more severe RRBs.
| score on a scale | Placebo | Alogabat 20 mg | Alogabat 60 mg |
|---|---|---|---|
| Change From Baseline to Week 12 in Behavior/Symptoms as Measured by All Domains of the Repetitive Behavior Scale-Revised (RBS-R) Score | -6.695 (-9.772 to -3.618) | -4.954 (-8.071 to -1.836) | -8.410 (-11.621 to -5.200) |
Vineland-3 is a semi-structured interview measuring an individual's adaptive behavior across 3 domains: Communication, Socialization \& Daily Living skills. Each domain consists of 3 subdomains. Subdomain raw scores are based on item responses (3-point scale: 0=never present; 1=sometimes present; 2=usually present) \& are calculated for each subdomain of Socialization (interpersonal relationships, play and leisure time, coping skills) domain as sum of the scores for each item in the subdomain. Raw scores for the 3 Socialization subdomains are used to derive GSVs (range=10-164). A conversion table for mapping raw scores to GSV scores is found in the Vineland-3 manual (Sparrow et al. 2016). GSV is a person-ability score used to track an individual's progress. Vineland-3 Socialization Domain GSV score is calculated as a mean GSV score (summing the 3 GSV subdomain scores \& dividing by 3). Vineland-3 Socialization Domain GSV score range = 10-145. Higher score =better adaptive functioning.
| score on a scale | Placebo | Alogabat 20 mg | Alogabat 60 mg |
|---|---|---|---|
| Change From Baseline to Week 12 on the Vineland-3 Socialization Domain | 6.298 (4.302 to 8.294) | 3.315 (1.162 to 5.468) | 1.824 (-0.220 to 3.868) |
Vineland-3 is a semi-structured interview measuring an individual's adaptive behavior across 3 domains: Communication, Socialization \& Daily Living skills. Each domain consists of 3 subdomains. Subdomain raw scores are based on item responses (3-point scale: 0=never present; 1=sometimes present; 2=usually present) \& is calculated for each subdomain of the Communication (receptive, expressive, written) domain as the sum of the scores for each item within the subdomain. Raw scores for each of the 3 Communication subdomains are used to derive Growth Scale Values (GSVs; range from 10-197). A conversion table for mapping raw scores to GSV scores is found in Vineland-3 manual (Sparrow et al. 2016). GSV is a person-ability score used to track an individual's progress. Vineland-3 Socialization Domain GSV score is calculated as mean GSV score (summing the 3 GSV subdomain scores \& dividing by 3). Vineland-3 Socialization Domain GSV score range=10-174. Higher score=better adaptive functioning.
| score on a scale | Placebo | Alogabat 20 mg | Alogabat 60 mg |
|---|---|---|---|
| Change From Baseline to Week 12 on the Vineland-3 Communication Domain | 1.624 (0.087 to 3.162) | 2.903 (1.228 to 4.578) | 4.321 (2.693 to 5.948) |
Collected over Up to Week 18. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Placebo | 0/34 (0%) | 0/34 (0%) | 16/34 (47.1%) |
| Alogabat 20 mg | 0/34 (0%) | 0/34 (0%) | 20/34 (58.8%) |
| Alogabat 60 mg | 0/36 (0%) | 0/36 (0%) | 17/36 (47.2%) |
| Event | Placebo | Alogabat 20 mg | Alogabat 60 mg |
|---|---|---|---|
| SomnolenceNervous system disorders | 5/34 | 7/34 | 6/36 |
| HeadacheNervous system disorders | 5/34 | 3/34 | 2/36 |
| IrritabilityPsychiatric disorders | 2/34 | 1/34 | 4/36 |
| NauseaGastrointestinal disorders | 0/34 | 1/34 | 4/36 |
| NasopharyngitisInfections and infestations | 1/34 | 3/34 | 1/36 |
| HypersomniaNervous system disorders | 2/34 | 3/34 | 3/36 |
| Sudden onset of sleepNervous system disorders | 0/34 | 3/34 | 2/36 |
| AnxietyPsychiatric disorders | 3/34 | 1/34 | 1/36 |
| FatigueGeneral disorders | 0/34 | 2/34 | 3/36 |
| GastroenteritisInfections and infestations | 0/34 | 2/34 | 1/36 |
Safety population included all participants randomized to study treatment and who received at least one dose of the study treatment, whether prematurely withdrawn from the study or not.
| Age, Continuous(years) | Placebo | Alogabat 20 mg | Alogabat 60 mg | Total |
|---|---|---|---|---|
| Mean | 25.3 ± 8.1 | 24.8 ± 7.1 | 25.0 ± 7.5 | 25.0 ± 7.5 |
| Sex: Female, Male(Participants) | Placebo | Alogabat 20 mg | Alogabat 60 mg | Total |
|---|---|---|---|---|
| Female | 9 | 8 | 9 | 26 |
| Male | 25 | 26 | 27 | 78 |
| Ethnicity (NIH/OMB)(Participants) | Placebo | Alogabat 20 mg | Alogabat 60 mg | Total |
|---|---|---|---|---|
| Hispanic or Latino | 11 | 6 | 6 | 23 |
| Not Hispanic or Latino | 22 | 28 | 29 | 79 |
| Unknown or Not Reported | 1 | 0 | 1 | 2 |
| Race (NIH/OMB)(Participants) | Placebo | Alogabat 20 mg | Alogabat 60 mg | Total |
|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 0 |
| Asian | 1 | 1 | 1 | 3 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 |
| Black or African American | 0 | 4 | 3 | 7 |
| White | 31 | 28 | 27 | 86 |
| More than one race | 0 | 0 | 2 | 2 |
| Unknown or Not Reported | 2 | 1 | 3 | 6 |
Documents are hosted by the registry — open the source record to download them.
Plan to share: Yes — Qualified researchers may request access to individual patient level data through the clinical study data request platform (www.vivli.org). Further details on Roche's criteria for eligible studies are available here (https://vivli.org/ourmember/roche/). For further details on Roche's Global Policy on the Sharing of Clinical Information and how to request access to related clinical study documents, see here (https://www.roche.com/research_and_development/who_we_are_how_we_work/clinical_trials/our_commitment_to_data_sharing.htm).
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