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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)

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ClinicalTrials.gov Identifier: NCT04299464
Recruitment Status : Recruiting
First Posted : March 6, 2020
Last Update Posted : June 1, 2022
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche

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
Autism Spectrum Disorder (ASD) Drug: Placebo Drug: RO7017773 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated 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 : March 31, 2021
Estimated Primary Completion Date : September 30, 2023
Estimated Study Completion Date : September 30, 2023

Resource links provided by the National Library of Medicine


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.




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) [ Time Frame: Week 12 ]

Secondary Outcome Measures :
  1. Percentage of Participants with Adverse Events (AEs) [ Time Frame: Up to Week 18 ]
  2. Percentage of Participants with Serious Adverse Events (SAEs) [ Time Frame: Up to Week 18 ]
  3. Percentage of Participants Discontinuing Treatment due to AEs [ Time Frame: From Baseline up to Week 12 ]
  4. Change from Baseline Over Time in Suicide Risk Using the Columbia-Suicide-Severity Rating Scale (C-SSRS) [ Time Frame: 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) [ Time Frame: Week 12 ]
  6. Change from Baseline to Week 12 on the Vineland-3 Socialization Domain [ Time Frame: Week 12 ]
  7. Change from baseline to Week 12 on the Vineland-3 Communication domains [ Time Frame: Week 12 ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   15 Years to 45 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04299464


Contacts
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Contact: Reference Study ID Number: BP41316 https://forpatients.roche.com/ 888-662-6728 global-roche-genentech-trials@gene.com

Locations
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Sponsors and Collaborators
Hoffmann-La Roche
Investigators
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Study Director: Clinical Trials Hoffmann-La Roche
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Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT04299464    
Other Study ID Numbers: BP41316
2019-003524-20 ( EudraCT Number )
First Posted: March 6, 2020    Key Record Dates
Last Update Posted: June 1, 2022
Last Verified: May 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
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).

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Autistic Disorder
Autism Spectrum Disorder
Child Development Disorders, Pervasive
Neurodevelopmental Disorders
Mental Disorders