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)

Sponsor
Hoffmann-La Roche (Industry)
Overall Status
Recruiting
CT.gov ID
NCT04299464
Collaborator
(none)
105
35
3
30
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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
Phase 2

Study Design

Study Type:
Interventional
Anticipated Enrollment :
105 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose:
Treatment
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)
Actual Study Start Date :
Mar 31, 2021
Anticipated Primary Completion Date :
Sep 30, 2023
Anticipated Study Completion Date :
Sep 30, 2023

Arms and Interventions

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.

Outcome Measures

Primary Outcome Measures

  1. 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

  1. Percentage of Participants with Adverse Events (AEs) [Up to Week 18]

  2. Percentage of Participants with Serious Adverse Events (SAEs) [Up to Week 18]

  3. Percentage of Participants Discontinuing Treatment due to AEs [From Baseline up to Week 12]

  4. Change from Baseline Over Time in Suicide Risk Using the Columbia-Suicide-Severity Rating Scale (C-SSRS) [Days 14, 42, 84, 98, 126]

  5. Change from Baseline to Week 12 in Behavior/Symptoms as Measured by all Domains of the Repetitive Behavior Scale-Revised (RBS-R) [Week 12]

  6. Change from Baseline to Week 12 on the Vineland-3 Socialization Domain [Week 12]

  7. Change from baseline to Week 12 on the Vineland-3 Communication domains [Week 12]

Eligibility Criteria

Criteria

Ages Eligible for Study:
15 Years to 45 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No

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) 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).

  • 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

Contacts and Locations

Locations

Site City State Country Postal Code
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.
Responsible Party:
Hoffmann-La Roche
ClinicalTrials.gov Identifier:
NCT04299464
Other Study ID Numbers:
  • BP41316
  • 2019-003524-20
First Posted:
Mar 6, 2020
Last Update Posted:
Aug 25, 2022
Last Verified:
Aug 1, 2022
Individual Participant Data (IPD) Sharing Statement:
Yes
Plan to Share IPD:
Yes
Studies a U.S. FDA-regulated Drug Product:
Yes
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:

Study Results

No Results Posted as of Aug 25, 2022