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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04049578
Recruitment Status : Terminated (Recent analysis of Phase II balovaptan data in paediatric ASD did not support the continuation of this study. No new safety concerns were identified.)
First Posted : August 8, 2019
Last Update Posted : May 15, 2020
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche

Brief Summary:
This is 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 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.

Condition or disease Intervention/treatment Phase
Autism Spectrum Disorder Drug: Balovaptan Phase 1

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 2 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : December 19, 2019
Actual Primary Completion Date : April 23, 2020
Actual Study Completion Date : May 6, 2020

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Balovaptan Drug: Balovaptan
Participants will 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.




Primary Outcome Measures :
  1. 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 ]
  2. Plasma Concentration 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 ]
  3. Plasma Concentration of M2 Metabolite, as Applicable [ 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 ]
  4. Plasma Concentation of M3 Metabolite [ 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 ]
  5. Plasma Concentration Ratio of M2 to Balovaptan, as Applicable [ 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 ]
  6. Plasma Concentration Ratio of M3 to 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 ]

Secondary Outcome Measures :
  1. Number of Participants With Adverse Events [ Time Frame: Up to approximately week 54 ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   2 Years to 4 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04049578


Locations
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United States, Arizona
Southwest Autism Research & Resource Center
Phoenix, Arizona, United States, 85006
United States, Connecticut
Yale Child Study Center
New Haven, Connecticut, United States, 06519
United States, New York
Richmond Behavioral Associates
Staten Island, New York, United States, 10312
United States, Ohio
University Hospitals Cleveland Medical Center; Division of Child and Adolescent Psychiatry
Cleveland, Ohio, United States, 44106
United States, Tennessee
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37232
United States, Texas
Red Oak Psychiatry Associates, PA
Houston, Texas, United States, 77090
United States, Washington
Seattle Children's Research Institute
Seattle, Washington, United States, 98105
Spain
Hospital Universitari Son Espases; Institut Balear de Salut Mental de la Infancia I l'Adolescencia
Palma de Mallorca, Islas Baleares, Spain, 07014
IGAIN (Instituto Global de Atención Integral al Neurodesarrollo)
Barcelona, Spain, 08007
Hospital Clinic i Provincial; Servicio de Psiquiatria y Psicologia en niños y adolescentes
Barcelona, Spain, 08036
Sponsors and Collaborators
Hoffmann-La Roche
Investigators
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Study Director: Clinical Trials Hoffmann-La Roche
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Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT04049578    
Other Study ID Numbers: WP40877
First Posted: August 8, 2019    Key Record Dates
Last Update Posted: May 15, 2020
Last Verified: May 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: 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).

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Autistic Disorder
Autism Spectrum Disorder
Child Development Disorders, Pervasive
Neurodevelopmental Disorders
Mental Disorders