Autism Spectrum Disorder Clinical Trial
Official title:
A Phase Ib, Multicenter, Open-Label, 6-Week Study With a 48-Week Extension to Investigate the Pharmacokinetics, Safety, and Tolerability of Balovaptan in Children Ages 2-4 Years With Autism Spectrum Disorder
Verified date | September 2020 |
Source | Hoffmann-La Roche |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Terminated |
Enrollment | 2 |
Est. completion date | May 6, 2020 |
Est. primary completion date | April 23, 2020 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 2 Years to 4 Years |
Eligibility |
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 |
Country | Name | City | State |
---|---|---|---|
United States | University Hospitals Cleveland Medical Center; Division of Child and Adolescent Psychiatry | Cleveland | Ohio |
United States | Red Oak Psychiatry Associates, PA | Houston | Texas |
United States | Vanderbilt University Medical Center | Nashville | Tennessee |
United States | Southwest Autism Research & Resource Center | Phoenix | Arizona |
United States | Richmond Behavioral Associates | Staten Island | New York |
Lead Sponsor | Collaborator |
---|---|
Hoffmann-La Roche |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Area Under the Curve at Steady State (AUCss) of Balovaptan | Predose, 2, 4, 6 hours postdose at Week 2; predose at Week 6; predose at Week 12; predose at Week 24, predose at Week 54 | ||
Primary | Plasma Concentration of Balovaptan | Predose, 2, 4, 6 hours postdose at Week 2; predose at Week 6; predose at Week 12; predose at Week 24, predose at Week 54 | ||
Primary | Plasma Concentration of M2 Metabolite, as Applicable | Predose, 2, 4, 6 hours postdose at Week 2; predose at Week 6; predose at Week 12; predose at Week 24, predose at Week 54 | ||
Primary | Plasma Concentation of M3 Metabolite | Predose, 2, 4, 6 hours postdose at Week 2; predose at Week 6; predose at Week 12; predose at Week 24, predose at Week 54 | ||
Primary | Plasma Concentration Ratio of M2 to Balovaptan, as Applicable | Predose, 2, 4, 6 hours postdose at Week 2; predose at Week 6; predose at Week 12; predose at Week 24, predose at Week 54 | ||
Primary | Plasma Concentration Ratio of M3 to Balovaptan | Predose, 2, 4, 6 hours postdose at Week 2; predose at Week 6; predose at Week 12; predose at Week 24, predose at Week 54 | ||
Secondary | Number of Participants With Adverse Events | Up to approximately week 20 |
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT05207956 -
App for Strengthening Services In Specialized Therapeutic Support
|
N/A | |
Completed |
NCT03286621 -
Development of Eye-tracking Based Markers for Autism in Young Children
|
||
Completed |
NCT02608333 -
Efficiency of Early Intervention for Autism Spectrum Disorder
|
N/A | |
Recruiting |
NCT05935722 -
Evaluation of a Home-based Parenting Support Program: Parenting Young Children
|
N/A | |
Active, not recruiting |
NCT06259539 -
A YouTube Curriculum for Children With Autism and Obesity
|
N/A | |
Active, not recruiting |
NCT06303791 -
Digital-based Psychosocial Intervention for Parents of Children With Neurodevelopmental Disorders
|
N/A | |
Enrolling by invitation |
NCT05017779 -
A Hybrid Effectiveness-implementation Trial of a High School-based Executive Function Treatment for Autistic Youth
|
N/A | |
Completed |
NCT04772898 -
Effectiveness of a 6-week Hippotherapy Program in Children With Autism Spectrum Disorder
|
N/A | |
Recruiting |
NCT04987541 -
The Therapeutic Effect of TBS Stimulation on Emotion Regulation in Autism Spectrum Disorder
|
N/A | |
Completed |
NCT04308915 -
Mobile-based Games for Cognitive Training in Children With Neurodevelopmental Disorders
|
N/A | |
Completed |
NCT06038435 -
The Effect of Cognitive Orientation Approach on Daily Occupational Performance With Autism Spectrum Disorder
|
N/A | |
Terminated |
NCT04049981 -
Investigation of Mechanisms of Action in Superpower Glass
|
Phase 1/Phase 2 | |
Completed |
NCT03693313 -
The Effect of CrossFit Kids on Social Skills in Children With Autism Spectrum Disorder (CrossFit KAMP)
|
N/A | |
Recruiting |
NCT04107064 -
Achieving Steady Work Among Adults With Autism Through Specialized Employment Program
|
N/A | |
Recruiting |
NCT03812068 -
Parent-mediated Developmental Behavioral Intervention
|
N/A | |
Completed |
NCT03206996 -
Exposure Therapy for Auditory Sensitivity in Autism
|
N/A | |
Completed |
NCT02299700 -
Study to Evaluate the Janssen Autism Knowledge Engine in Children and Adults With Autism Spectrum Disorder
|
N/A | |
Completed |
NCT03422016 -
Electroretinogram in Autistic Spectrum Disorders
|
||
Active, not recruiting |
NCT03548779 -
North Carolina Genomic Evaluation by Next-generation Exome Sequencing, 2
|
N/A | |
Recruiting |
NCT05114538 -
Improving the Part C Early Intervention Service Delivery System for Children With ASD
|
N/A |