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)

NCT04299464 | Completed Phase 2 Results FDA Drug

• 2 other identifiers: BP413162019-003524-20
• Study Type: interventional
• Target: 104
• Locations: 4 countries
• Sites: 21

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).

Conditions

Autism Spectrum Disorder (ASD)

Outcome Measures

Primary Outcomes (1)

• 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.

  Baseline to Week 12

Secondary Outcomes (11)

• Number of Participants With at Least One Adverse Events (AEs)

  Up to Week 18

• Number of Participants With at Least One Serious Adverse Events (SAEs)

  Up to Week 18

• Number of Participants Discontinuing Treatment Due to AEs

  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)

  Baseline up to Week 18

• Change From Baseline in Karolinska Sleepiness Scale (KSS) Score for Assessing Daytime Sleepiness

  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

Study Arms (3)

Placebo

PLACEBO COMPARATOR

Participants will receive placebo matched to RO7017773 for approximately 12 weeks.

Drug: Placebo

RO7017773 Low Dose

EXPERIMENTAL

Participants will receive a fixed low dose of RO7017773 for approximately 12 weeks.

Drug: RO7017773

RO7017773 High Dose

EXPERIMENTAL

Participants will receive a fixed high dose of RO7017773 for approximately 12 weeks.

Drug: RO7017773

Interventions

Placebo DRUG

Participants will receive oral placebo for approximately 12 weeks.

Placebo

RO7017773 DRUG

Participants will receive oral RO7017773 for approximately 12 weeks.

RO7017773 High Dose; RO7017773 Low Dose

Eligibility Criteria

• Age: 15 Years - 45 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Hoffmann-La Roche: lead

Study Sites (21)

Southwest Autism Research and Resource Center

Phoenix, Arizona, 85006, United States

Location

Yale University / Yale-New Haven Hospital

New Haven, Connecticut, 06519-1124, United States

Location

APG- Advanced Psychiatric Group

Orlando, Florida, 32803, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55414-2959, United States

Location

Nathan Kline Institute

Orangeburg, New York, 10962, United States

Location

Montefiore Medical Center

The Bronx, New York, 10461, United States

Location

University Hospitals

Cleveland, Ohio, 44106, United States

Location

Ohio State University

Columbus, Ohio, 43210, United States

Location

UPMC Western Psychiatric Institute and Clinic

Pittsburgh, Pennsylvania, 15203, United States

Location

Vanderbilt Medical Center

Nashville, Tennessee, 37212, United States

Location

Okanagan Clinical Trials

Kelowna, British Columbia, V1Y 1Z9, Canada

Location

Janeway Childrens Health

St. John's, Newfoundland and Labrador, A1B 3V6, Canada

Location

Holland Bloorview Kids Rehabilitation Hospital

East York, Ontario, M4G 1R8, Canada

Location

London Health Sciences Centre

London, Ontario, N6A 4G5, Canada

Location

Ist. G. Gaslini

Genoa, Liguria, 16147, Italy

Location

Istituto Scientifico Medea

Bosisio Parini (LC), Lombardy, 23842, Italy

Location

P.O. Gaspare Rodolico

Catania, Sicily, 95123, Italy

Location

IGAIN (Instituto Global de Atención Integral al Neurodesarrollo)

Barcelona, 08007, Spain

Location

Hospital Universitari Vall d'Hebron

Barcelona, 08035, Spain

Location

Hospital General Universitario Gregorio Marañon

Madrid, 28009, Spain

Location

MeSH Terms

Conditions

Autism Spectrum Disorder

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Results Point of Contact

• Title: Medical Communications
• Organization: Hoffmann-La Roche

genentech@druginfo.com

800 821-8590

Study Officials

• Clinical Trials

  Hoffmann-La Roche

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 4, 2020
• First Posted: March 6, 2020
• Study Start: March 31, 2021
• Primary Completion: May 15, 2024
• Study Completion: May 15, 2024
• Last Updated: January 28, 2025
• Results First Posted: January 28, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will share

Locations

ES (3); CA (4); IT (3); US (11)