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)

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)

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).

Study Overview

• Status: Active, not recruiting
• Conditions: Autism Spectrum Disorder (ASD)
• Intervention / Treatment: Drug: Placebo; Drug: RO7017773

• Study Type: Interventional
• Enrollment (Actual): 103
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Reference Study ID Number: BP41316 https://forpatients.roche.com/; Phone Number: 888-662-6728; Email: global-roche-genentech-trials@gene.com

Study Locations

• Canada
  • Alberta
    • Edmonton, Alberta, Canada, T6G 2G3
      • University of Alberta
  • British Columbia
    • Kelowna, British Columbia, Canada, V1Y 1Z9
      • Okanagan Clinical Trials
  • Newfoundland and Labrador
    • St. John's, Newfoundland and Labrador, Canada, A1B 3V6
      • Janeway Childrens Health; and Rehabilitation Centre
  • Ontario
    • East York, Ontario, Canada, M4G 1R8
      • Holland Bloorview Kids Rehabilitation Hospital; Autism Research Centre
    • London, Ontario, Canada, N6A 4G5
      • London Health Sciences Centre; Victoria Hospital
• Italy
  • Campania
    • Napoli, Campania, Italy, 80131
      • Azienda Ospedaliera Universitaria Federico II; U.O.S.D. Neuropsichiatria Infantile
  • Lazio
    • Roma, Lazio, Italy, 133
      • AOU Policlinico Tor Vergata, Università Roma Tor Vergata
  • Liguria
    • Genova, Liguria, Italy, 16147
      • Ist. G. Gaslini; UOC Neuropsichiatria Infantile
  • Lombardia
    • Bosisio Parini (LC), Lombardia, Italy, 23842
      • Istituto Scientifico Medea; U.O Psicopatologia età evolutiva
    • Pavia, Lombardia, Italy, 27100
      • ASST di Pavia; Dip. di Scienze del Sistema Nervoso e del Comportamento
  • Sicilia
    • Catania, Sicilia, Italy, 95123
      • P.O. Gaspare Rodolico; UOC Clinica Psichiatrica
  • Toscana
    • Calambrone (PI), Toscana, Italy, 56128
      • IRCCS Fondazione Stella Maris; U.O. Complessa NPI 3 - Psichiatria dello sviluppo
• Spain
  • Barcelona, Spain, 08035
    • Hospital Universitari Vall d'Hebron; Sevicio de Psiquiatría
  • Barcelona, Spain, 08007
    • IGAIN (Instituto Global de Atención Integral al Neurodesarrollo)
  • Madrid, Spain, 28009
    • Hospital General Universitario Gregorio Marañon; Instituto Provincial de Psiquiatría
  • Madrid, Spain, 28031
    • Hospital Universitario Infanta Leonor; Servicio de Psiquiatría
• United States
  • Arizona
    • Phoenix, Arizona, United States, 85006
      • Southwest Autism Research and Resource Center
  • Connecticut
    • New Haven, Connecticut, United States, 06519-1124
      • Yale University / Yale-New Haven Hospital
  • Florida
    • Orlando, Florida, United States, 32803
      • APG- Advanced Psychiatric Group
  • Illinois
    • Chicago, Illinois, United States, 60612
      • Rush University Medical Center
  • Minnesota
    • Minneapolis, Minnesota, United States, 55414-2959
      • University of Minnesota
  • New York
    • Bronx, New York, United States, 10461
      • Montefiore Medical Center
    • Orangeburg, New York, United States, 10962
      • Nathan Kline Institute
  • Ohio
    • Cleveland, Ohio, United States, 44106
      • University Hospitals
    • Columbus, Ohio, United States, 43210
      • Ohio State University
  • Pennsylvania
    • Pittsburgh, Pennsylvania, United States, 15203
      • UPMC Western Psychiatric Institute and Clinic
  • Tennessee
    • Nashville, Tennessee, United States, 37232
      • Vanderbilt Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 15 years to 45 years (Child, Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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	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

Collaborators and Investigators

• Sponsor: Hoffmann-La Roche
• Investigators: Study Director: Clinical Trials, Hoffmann-La Roche

Study record dates

Study Major Dates

• Study Start (Actual): March 31, 2021
• Primary Completion (Estimated): May 17, 2024
• Study Completion (Estimated): May 17, 2024

Study Registration Dates

• First Submitted: March 4, 2020
• First Submitted That Met QC Criteria: March 4, 2020
• First Posted (Actual): March 6, 2020

Study Record Updates

• Last Update Posted (Actual): March 29, 2024
• Last Update Submitted That Met QC Criteria: March 28, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Mental Disorders; Neurodevelopmental Disorders; Autistic Disorder; Autism Spectrum Disorder; Child Development Disorders, Pervasive
• Other Study ID Numbers: BP41316; 2019-003524-20 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: 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).

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No