A Study Investigate the Pharmacokinetics, Safety, and Tolerability of Balovaptan in Children Ages 2–4 Years with Autism Spectrum Disorder

Phase 1

• Conditions: Autism spectrum disorder (ASD); MedDRA version: 20.0Level: PTClassification code 10063844Term: Autism spectrum disorderSystem Organ Class: 10037175 - Psychiatric disorders; MedDRA version: 20.0Level: LLTClassification code 10003805Term: AutismSystem Organ Class: 10037175 - Psychiatric disorders; Therapeutic area: Psychiatry and Psychology [F] - Mental Disorders [F03]

• Registration Number: EUCTR2019-000989-38-ES
• Lead Sponsor: Roche Farma S. A. U. que realiza el ensayo en España y que actúa como representante F. Hoffmann-La Roche Ltd

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-06-11
• Last Update Posted: 2 June 2020

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 5

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

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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified