Autism spectrum disorder (ASD) MedDRA version: 20.0 Level: PT Classification code 10063844 Term: Autism spectrum disorder System Organ Class: 10037175 - Psychiatric disorders MedDRA version: 20.0 Level: LLT Classification code 10003805 Term: Autism System Organ Class: 10037175 - Psychiatric disorders
Conditions
Interventions
Product Name: RO528-5119/F10
Product Code: RO528-5119/F10
Pharmaceutical Form: Dispersible tablet
INN or Proposed INN: RO5285119
CAS Number: 1228088-30-9
Current Sponsor code: RO5285119
Other descript
Sponsors
Roche Farma S. A. U. que realiza el ensayo en España y que actúa como representante F. Hoffmann-La Roche Ltd
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Males or females 2-4 years of age - Diagnosis of ASD according to Diagnostic and Statistical Manual of Mental Disorders -5 criteria for ASD - Hearing and vision compatible with the study assessments, as judged by the investigator - Ability for subject and the caregiver to comply with the study protocol, in the investigator’s judgment - Availability of a parent or other reliable caregiver who is fluent in language of the site and has frequent and sufficient contact with the subject Are the trial subjects under 18? yes Number of subjects for this age range: 5 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Neurologic and Psychiatric Exclusion Criteria - Clinically significant psychiatric and/or neurologic comorbidity that may interfere with the safety or efficacy endpoints in the view of the investigator - Clinically significant regression of any acquired language and motor function skills in the opinion of the investigator throughout the subject’s development - History of seizures with the exception of a single, non-complicated febrile seizure >= 6 months before screening - Clinical diagnosis of peripheral neuropathy or signs and symptoms indicative of peripheral neuropathy Cardiovascular Exclusion Criteria - Any clinically relevant cardiovascular disease - Confirmed elevation in cardiac troponin I (cTn I), high-sensitive cardiac troponin T (hs cTn T), N-terminal pro -B-type natriuretic peptide (NT-proBNP) or, if conducted, clinically relevant abnormality in Doppler echocardiogram - Confirmed clinically significant abnormality on ECG at screening, including, but not limited to, a QT interval corrected through use of Fridericia’s formula (QTcF) of >= 450 ms, absence of dominating sinus rhythm, or second- or third-degree atrioventricular block Other Organ Systems Exclusion Criteria - Concomitant disease or that could interfere with, or treatment of which might interfere with, the conduct of the study; or discontinuation of prohibited medication that might pose unacceptable risks to the subject in the opinion of the investigator - Evidence for current GI disease that would interfere with the conduct of the study or pose unacceptable risks in the opinion of the investigator - History of coagulopathies, bleeding disorders, or blood dyscrasias - Positive serology for HIV-1 or HIV-2 - Confirmed clinically significant abnormality in parameters of hematology, clinical chemistry, coagulation, or urinalysis, specifically a confirmed absolute neutrophil count < LLN - History of malignancy Additional Exclusion Criteria - Participation in an investigational drug study within 90 days prior to treatment assignment, or participation in a study testing an investigational medical device within 90 days prior to treatment assignment or if the device is still active - Presence of any clinically significant abnormality likely to interfere with the conduct of the study according to the judgment of the investigator - Clinically significant loss of blood within 3 months prior to screening - Unstable use of permitted medications for 4 weeks before screening - Use of prohibited medications within 30 days prior to initiation of study treatment - Other severe medical comorbidity that may interfere with the safety or efficacy endpoints
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: •To investigate the plasma exposure at steady-state (Area under the concentration-time curve at steady state [AUCss]) of balovaptan and to determine the dose that will deliver adult-equivalent exposure in subjects ages 2-4 years old;Secondary Objective: •To investigate the pharmacokinetics of balovaptan and its metabolites M2 (as applicable) and M3 in subjects ages 2-4 years •To evaluate the safety and tolerability of treatment with 10-mg equivalent dose balovaptan;Primary end point(s): 1. Balovaptan AUCss estimates, as derived using a population-pharmacokinetic (pop-PK) modeling approach;Timepoint(s) of evaluation of this end point: 1. For six-week treatment period: Week 2, Week 6, at early termination; for optional extension period: Week 12, Week 24, Week 54, at early termination | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1.Plasma concentration of balovaptan and its metabolites M2 (as applicable) and M3 at specified timepoints 2.Plasma concentration ratio of M2 (as applicable) to balovaptan and M3 to balovaptan and other pharmacokinetic parameters as appropriate 3.Incidence and severity of adverse events, with severity determined according to the Adverse Event Severity Grading Scale 4.Incidence and severity of treatment-emergent abnormalities in physical and neurologic examinations 5.Change from baseline in vital signs 6.Change from baseline in ECG parameters 7.Change from baseline in clinical laboratory test results 8.Where applicable: Incidence and severity of treatment-emergent abnormalities in echocardiogram parameters;Timepoint(s) of evaluation of this end point: 1-2. For six-week treatment period: Week 2, Week 6, at early termination; for optional extension period: Week 12, Week 24, Week 54, at early termination 3-4. Up to Week 56 5-8. From baseline (Day 1) to Week 56 | — |
Countries
Spain
Contacts
Public ContactTrial Information Support Line-TISL
F. Hoffmann-La Roche Ltd
Outcome results
None listed