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 Details
Study Description
Brief Summary
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).
Condition or Disease | Intervention/Treatment | Phase |
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Phase 2 |
Study Design
Arms and Interventions
Arm | Intervention/Treatment |
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Placebo Comparator: Placebo Participants will receive placebo matched to RO7017773 for approximately 12 weeks. |
Drug: Placebo
Participants will receive oral placebo for approximately 12 weeks.
|
Experimental: RO7017773 Low Dose Participants will receive a fixed low dose of RO7017773 for approximately 12 weeks. |
Drug: RO7017773
Participants will receive oral RO7017773 for approximately 12 weeks.
|
Experimental: RO7017773 High Dose Participants will receive a fixed high dose of RO7017773 for approximately 12 weeks. |
Drug: RO7017773
Participants will receive oral RO7017773 for approximately 12 weeks.
|
Outcome Measures
Primary Outcome Measures
- 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
- 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]
Eligibility Criteria
Criteria
Inclusion Criteria
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Male and female participants with Autism Spectrum Disorder according to Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5)
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Wechsler Abbreviated Scale of Intelligence (WASI-II) >/= 50 at screening or within the last 12 months prior to screening
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ASD or Autism diagnosis confirmed by Autism Diagnostic Observation Schedule (ADOS-2)
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Body mass index within the range of 18.5 to 40 kg/m2
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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
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Language, hearing, and vision compatible with the study measurements as judged by the Investigator
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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
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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
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In the Investigator's opinion, participation in the study or discontinuation of prohibited medication will not pose undue risks
Exclusion Criteria
Neurologic/Psychiatric Conditions:
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Non-verbal individuals
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Presence of chromosome 15q11.2 q13.1 duplication syndrome (Dup15q syndrome) genetically defined ASD per genetic results available prior to screening or known "syndromic" forms of ASD (e.g., fragile X syndrome, Prader Willi syndrome, Rett's syndrome, or tuberous sclerosis).
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Medical history of alcohol and/or substance abuse/dependence in the last 12 months or positive test for drugs of abuse at screening
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Initiation of a major change in psychosocial intervention within 6 weeks prior to screening. Minor changes in ongoing treatment are not considered major changes
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Clinically significant psychiatric and/or neurological disorder that may interfere with the safety or efficacy endpoints
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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
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Unstable epilepsy/seizure disorder within the past 6 months or changes in anticonvulsive therapy within the last 6 months
Other Conditions:
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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
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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
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Use of prohibited medications or herbal remedies within 6 weeks or 5 half-lives (t1/2) prior to randomization
Prior/Concurrent Clinical Study Experience:
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Donation or loss of blood over 500 mL in adults and 250 mL in adolescents within 3 months prior to randomization
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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
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Confirmed clinically significant abnormality in hematological, chemistry or coagulation laboratory parameters
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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
Contacts and Locations
Locations
Site | City | State | Country | Postal Code | |
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1 | Southwest Autism Research and Resource Center | Phoenix | Arizona | United States | 85006 |
2 | University of California at San Francisco | San Francisco | California | United States | 94115 |
3 | MCB Clinical Research Centers | Colorado Springs | Colorado | United States | 80910 |
4 | Yale University / Yale-New Haven Hospital | New Haven | Connecticut | United States | 06519-1124 |
5 | Research Centers of America, LLC | Oakland Park | Florida | United States | 33334 |
6 | APG- Advanced Psychiatric Group | Orlando | Florida | United States | 32803 |
7 | Rush University Medical Center | Chicago | Illinois | United States | 60612 |
8 | Capstone Clinical Research | Libertyville | Illinois | United States | 60048 |
9 | Lake Charles Clinical Trials, LLC | Lake Charles | Louisiana | United States | 70601 |
10 | Massachusetts General Hospital; Lurie Center for Autism | Lexington | Massachusetts | United States | 02421 |
11 | University of Minnesota | Minneapolis | Minnesota | United States | 55414-2959 |
12 | Montefiore Medical Center | Bronx | New York | United States | 10461 |
13 | Nathan Kline Institute | Orangeburg | New York | United States | 10962 |
14 | University Hospitals | Cleveland | Ohio | United States | 44106 |
15 | Ohio State University | Columbus | Ohio | United States | 43210 |
16 | UPMC Western Psychiatric Institute and Clinic | Pittsburgh | Pennsylvania | United States | 15203 |
17 | Vanderbilt Medical Center | Nashville | Tennessee | United States | 37232 |
18 | BioBehavioral Research of Austin, PC | Austin | Texas | United States | 78759 |
19 | Relaro Medical Trials | Dallas | Texas | United States | 75243 |
20 | Red Oak Psychiatry Associates, PA | Houston | Texas | United States | 77090 |
21 | Road Runner Research | San Antonio | Texas | United States | 78249 |
22 | Core Clinical Research | Kirkland | Washington | United States | 98033 |
23 | Okanagan Clinical Trials | Kelowna | British Columbia | Canada | V1Y 1Z9 |
24 | Janeway Childrens Health; and Rehabilitation Centre | St. John's | Newfoundland and Labrador | Canada | A1B 3V6 |
25 | Holland Bloorview Kids Rehabilitation Hospital; Autism Research Centre | East York | Ontario | Canada | M4G 1R8 |
26 | AOU Policlinico Tor Vergata, Università Roma Tor Vergata | Roma | Lazio | Italy | 133 |
27 | Ist. G. Gaslini; UOC Neuropsichiatria Infantile | Genova | Liguria | Italy | 16147 |
28 | Istituto Scientifico Medea; U.O Psicopatologia età evolutiva | Bosisio Parini (LC) | Lombardia | Italy | 23842 |
29 | ASST di Pavia; Dip. di Scienze del Sistema Nervoso e del Comportamento | Pavia | Lombardia | Italy | 27100 |
30 | P.O. Gaspare Rodolico; UOC Clinica Psichiatrica | Catania | Sicilia | Italy | 95123 |
31 | IRCCS Fondazione Stella Maris; U.O. Complessa NPI 3 - Psichiatria dello sviluppo | Calambrone (PI) | Toscana | Italy | 56128 |
32 | Hospital Santa Caterina; Servicio de Psiquiatría | Salt | Girona | Spain | 17190 |
33 | IGAIN (Instituto Global de Atención Integral al Neurodesarrollo) | Barcelona | Spain | 08007 | |
34 | Hospital Universitari Vall d'Hebron; Sevicio de Psiquiatría | Barcelona | Spain | 08035 | |
35 | Hospital Universitario Infanta Leonor; Servicio de Psiquiatría | Madrid | Spain | 28031 |
Sponsors and Collaborators
- Hoffmann-La Roche
Investigators
- Study Director: Clinical Trials, Hoffmann-La Roche
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- BP41316
- 2019-003524-20