A Study to Investigate the Pharmacokinetics, Safety, and Tolerability of Balovaptan in Children With Autism Spectrum Disorder

Roche

Status and phase

Terminated

Phase 1

Conditions

Autism Spectrum Disorder

Treatments

Drug: Balovaptan

Study type

Interventional

Funder types

Industry

Identifiers

NCT04049578

WP40877

Details and patient eligibility

About

This was a Phase Ib, multicenter, open-label study in children 2-4 years old with autism spectrum disorder (ASD) to investigate the pharmacokinetics, safety, and tolerability of an oral dose of balovaptan once a day (QD). The study was to consists of a 6-week treatment period to evaluate the pharmacokinetics of balovaptan in 2 to 4 year old children followed by an optional extension period of 48 weeks.

Enrollment

2 patients

Sex

All

Ages

2 to 4 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Diagnosis of ASD according to Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5) criteria for ASD. Diagnostics will be performed by a team of autism experts and confirmed by Autism Diagnostic Observation ScheduleTM, Second Edition (ADOS-2) criteria. The DSM-5 criteria for diagnosis of autism must be met with the highest confidence in the opinion of the investigator. Children with ambiguous diagnostic results cannot be enrolled in the study. If the ADOS-2 assessment has been performed by a certified rater and documented within 12 months of the screening visit, it is not mandatory to repeat it unless the subject was assessed below an age of 2 years.
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.

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
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
Confirmed systolic or diastolic blood pressure above the 95th percentile or below the 5th percentile according to the Centers for Disease Control and Prevention (CDC) norm tables referring to stature (height)-for-age percentiles
Confirmed heart rate: >150 bpm in 2-year old children, >135 bpm in 3-year old children, or >120 bpm in 4-year old children.
Concomitant disease or condition 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 (ANC) <LLN Children with confirmed CPK elevations exceeding 2 x upper limit of normal (ULN) will be excluded
History of malignancy
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 (or 5 times the half-life, whichever is longer) prior to initiation of study treatment