NCT04049578

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

Terminated Phase 1 Results posted Last updated 14 October 2020

What this trial tests

Phase 1 trial testing Balovaptan in Autism Spectrum Disorder in 2 participants. Terminated before completion.

Timeline

19 December 2019

Primary endpoint
23 April 2020

6 May 2020

Quick facts

Lead sponsor	Hoffmann-La Roche
Phase	Phase 1
Status	Terminated
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	2
Start date	19 December 2019
Primary completion	23 April 2020
Estimated completion	6 May 2020
Sites	5 locations across United States

Drugs / interventions tested

• Balovaptan — full drug profile →

Conditions studied

• Autism Spectrum Disorder — all drugs for Autism Spectrum Disorder →

Sponsor

Hoffmann-La Roche — full company profile →

Who can join

Adults 2 to 4, any sex, with Autism Spectrum Disorder. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Number of Participants With Adverse Events Secondary · Up to approximately week 20

Group	Value	95% CI
Balovaptan	2

Adverse events — posted to ClinicalTrials.gov

Time frame: From the first study drug to the data cutoff date: 6 May 2020 (up to approximately 20 weeks). Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Balovaptan

Serious: 0/2 (0%)

Deaths: 0/2

Other adverse events (5 terms — click to expand)

Reaction	System	Balovaptan
Blood thyroid stimulating hormone increased	Investigations	—
Abnormal weight gain	Metabolism and nutrition disorders	—
Psychomotor hyperactivity	Nervous system disorders	—
Cough	Respiratory, thoracic and mediastinal disorders	—
Haematoma	Vascular disorders	—

Data from ClinicalTrials.gov NCT04049578 adverse events section.

Sponsor's own description

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.

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

1. Role of Oxytocin and Vasopressin in Neuropsychiatric Disorders: Therapeutic Potential of Agonists and Antagonists.
   Cid-Jofré V, Moreno M, Reyes-Parada M, Renard GM. · · 2021 · cited 23× · PMID 34769501 · DOI 10.3390/ijms222112077