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Autism Spectrum DisorderPhase 1NCT04049578

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

A Phase 1 study testing Balovaptan in people with Autism Spectrum Disorder, run by Hoffmann-La Roche at 5 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

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). Read more about this study

Who is it for?
Ages 2 to 4 years
Conditions the study is for
Autism Spectrum Disorder
What is being tested?
Balovaptan
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
2 participants
Where
5 sitesUnited States
Study dates
19 Dec 2019to 6 May 2020
Phase
Phase 1
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 2 to 4 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • 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.

Who cannot join

  • 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
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  • 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

Trial locations and contacts

5 sites in 1 country.

5 sites in 1 country

United States5 sites

  • University Hospitals Cleveland Medical Center; Division of Child and Adolescent Psychiatry

    Cleveland, Ohio, 44106

  • Red Oak Psychiatry Associates, PA

    Houston, Texas, 77090

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232

  • Southwest Autism Research & Resource Center

    Phoenix, Arizona, 85006

  • Richmond Behavioral Associates

    Staten Island, New York, 10312

Other trials in United States

About this study

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

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.

Being tested
Balovaptan
Medical subject headings
Autism Spectrum Disorder

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • BalovaptanDrug

    Participants were to receive an oral dose of balovaptan once a day (QD) for a 6-week treatment period, followed by an optional extension period of 48 weeks.

Study groups in NCT04049578
Study groupRoleWhat they receive
BalovaptanExperimentalNot described
Receives: Drug: Balovaptan

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Area Under the Curve at Steady State (AUCss) of Balovaptan

    Measured: 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

  2. Plasma Concentration of Balovaptan

    Measured: 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

  3. Plasma Concentration of M2 Metabolite, as Applicable

    Measured: 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

  4. Plasma Concentation of M3 Metabolite

    Measured: 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

  5. Plasma Concentration Ratio of M2 to Balovaptan, as Applicable

    Measured: 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

  6. Plasma Concentration Ratio of M3 to Balovaptan

    Measured: 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 outcomes (1)

  1. Number of Participants With Adverse Events

    Measured: Up to approximately week 20

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted8 Aug 2019
  2. Study start19 Dec 2019
  3. Primary completion23 Apr 2020
  4. Study completion6 May 2020

Why it stopped: Recent analysis of Phase II balovaptan data in paediatric ASD did not support the continuation of this study. No new safety concerns were identified.

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
2 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04049578
Sponsor's protocol ID
WP40877
Status last verified
Sep 2020
First submitted
6 Aug 2019
Last updated
14 Oct 2020
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No: Qualified researchers may request access to individual patient level data through the clinical study data request platform (www.clinicalstudydatarequest.com). Further details on Roche's criteria for eligible studies are available here (https://clinicalstudydatarequest.com/Study-Sponsors/Study-Sponsors-Roche.aspx). For further details on Roche's Global Policy on Sharing of Clinical Study Information and how to request access to related clinical study documents, see here (https://www.roche.com/research\_and\_development/who\_we\_are\_how\_we\_work/clinical\_trials/our\_commitment\_to\_data\_sharing.htm).

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Balovaptan in people with Autism Spectrum Disorder, run by Hoffmann-La Roche at 5 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 14 Oct 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 14 Oct 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Area Under the Curve at Steady State (AUCss) of Balovaptan (time frame: 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). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04049578.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Oct 2020.

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