A 12-Week Placebo-Controlled Study to Investigate the Efficacy, Safety, and Tolerability of RO7017773 in Participants Aged 15-45 Years With Autism Spectrum Disorder (ASD)

Study Overview
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Brief Summary
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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).

Official Title
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A Phase II Multicenter, Randomized, Double-Blind, 12-Week Treatment, 3-Arm, Parallel-Group, Placebo-Controlled Study to Investigate the Efficacy, Safety, and Tolerability of RO7017773 in Participants Aged 15-45 Years With Autism Spectrum Disorder (ASD)

Conditions
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Autism Spectrum Disorder (ASD)

Intervention / Treatment
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* Drug: Placebo
* Drug: RO7017773


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria Male and female participants with Autism Spectrum Disorder according to Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5) Wechsler Abbreviated Scale of Intelligence (WASI-II) >/= 50 at screening or within the last 12 months prior to screening ASD or Autism diagnosis confirmed by Autism Diagnostic Observation Schedule (ADOS-2) Body mass index within the range of 18.5 to 40 kg/m2 Female Participants: is eligible if she is not pregnant, not breastfeeding, and women of childbearing potential (WOCBP), who agree to remain abstinent or use contraceptive methods that result in a failure rate of < 1% per year during the treatment period and for at least 28 days after the last dose of study drug Language, hearing, and vision compatible with the study measurements as judged by the Investigator Allowed existing treatment regimens should be stable for 8 weeks prior to screening. Investigator expects stability of these treatments and behavioral interventions for the duration of the study In the Investigator's opinion, able to participate and deemed appropriate for participation in the study, capable of following the study SoA and able to comply with the study restrictions In the Investigator's opinion, participation in the study or discontinuation of prohibited medication will not pose undue risks Exclusion Criteria Neurologic/Psychiatric Conditions: Non-verbal individuals Presence of chromosome 15q11.2 q13.1 duplication syndrome (Dup15q syndrome), known syndromic forms of ASD (confirmed per genetic results available at screening): fragile X syndrome, Prader Willi syndrome, Rett's syndrome, tuberous sclerosis, and Angelman syndrome, as well as genetic alterations strongly associated with ASD per genetic results available at screening affecting the following genes: CHD8, ANDP, SHANK3 Medical history of alcohol and/or substance abuse/dependence in the last 12 months or positive test for drugs of abuse at screening Initiation of a major change in psychosocial intervention within 6 weeks prior to screening. Minor changes in ongoing treatment are not considered major changes Clinically significant psychiatric and/or neurological disorder that may interfere with the safety or efficacy endpoints Risk of suicidal behavior in the opinion of a certified clinician or as evidenced by a yes to questions 4 and/or 5 of Columbia-Suicide-Severity Rating Scale (C-SSRS) taken at screening and baseline with respect to the last 12 months, or any suicide attempt in the past 5 years Unstable epilepsy/seizure disorder within the past 6 months or changes in anticonvulsive therapy within the last 6 months Other Conditions: Medical history of malignancy if not considered cured or if occurred within the last 3 years with the exception of fully excised non-melanoma skin cancers or in-situ carcinoma of the cervix that has been successfully treated Concomitant disease, condition or treatment which would either interfere with the conduct of the study or pose an unacceptable risk to the participant in the opinion of the Investigator Prior/Concomitant Therapy Use of prohibited medications or herbal remedies within 6 weeks or 5 half-lives (t1/2) prior to randomization Prior/Concurrent Clinical Study Experience: Donation or loss of blood over 500 mL in adults and 250 mL in adolescents within 3 months prior to randomization Participation in an investigational drug study within 1 month or 5 times the t1/2 of the investigational molecule prior to randomization or participation in a study testing an investigational medical device within 1 month prior to randomization or if the device is still active Diagnostic Assessments Confirmed clinically significant abnormality in hematological, chemistry or coagulation laboratory parameters Positive test result at screening for hepatitis B surface antigen, hepatitis C virus (HCV, untreated), or human immunodeficiency virus (HIV)-1 and -2. HCV participants who have been successfully treated and who test negative for HCV RNA, may be considered eligible for entry into the study Other Exculsions: Uncorrected hypokalemia or hypomagnesaemia

Ages Eligible for Study
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Minimum Age: 15 Years
Maximum Age: 45 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Placebo Comparator: Placebo<br>Participants will receive placebo matched to RO7017773 for approximately 12 weeks. | Drug: Placebo<br>* Participants will receive oral placebo for approximately 12 weeks.<br>|
| Experimental: RO7017773 Low Dose<br>Participants will receive a fixed low dose of RO7017773 for approximately 12 weeks. | Drug: RO7017773<br>* Participants will receive oral RO7017773 for approximately 12 weeks.<br>|
| Experimental: RO7017773 High Dose<br>Participants will receive a fixed high dose of RO7017773 for approximately 12 weeks. | Drug: RO7017773<br>* Participants will receive oral RO7017773 for approximately 12 weeks.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from Baseline to Week 12 in the Adaptive Behavior Composite score of the Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) |  | Week 12 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Participants with Adverse Events (AEs) |  | Up to Week 18 | 
| Percentage of Participants with Serious Adverse Events (SAEs) |  | Up to Week 18 | 
| Percentage of Participants Discontinuing Treatment due to AEs |  | From Baseline up to Week 12 | 
| Change from Baseline Over Time in Suicide Risk Using the Columbia-Suicide-Severity Rating Scale (C-SSRS) |  | Days 14, 42, 84, 98, 126 | 
| Change from Baseline to Week 12 in Behavior/Symptoms as Measured by all Domains of the Repetitive Behavior Scale-Revised (RBS-R) |  | Week 12 | 
| Change from Baseline to Week 12 on the Vineland-3 Socialization Domain |  | Week 12 | 
| Change from baseline to Week 12 on the Vineland-3 Communication domains |  | Week 12 |