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)

Autism Spectrum Disorder (ASD) Clinical Trial

Official title:

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)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04299464
• Other study ID #: BP41316
• Secondary ID: 2019-003524-20
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: March 31, 2021
• Est. completion date: May 17, 2024

Study information

• Verified date: March 2024
• Source: Hoffmann-La Roche
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 103
• Est. completion date: May 17, 2024
• Est. primary completion date: May 17, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 15 Years to 45 Years

Eligibility

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

Related Conditions & MeSH terms

• Autism Spectrum Disorder
• Autism Spectrum Disorder (ASD)
• Autistic Disorder
• Child Development Disorders, Pervasive

Intervention

Drug:
• Placebo
Participants will receive oral placebo for approximately 12 weeks.
• RO7017773
Participants will receive oral RO7017773 for approximately 12 weeks.

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Hoffmann-La Roche

Countries where clinical trial is conducted

United States,  Canada,  Italy,  Spain, 

Outcome

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