Phase II Clinical Trial to Evaluate the Efficacy and Safety of NV01-A02 in Children With Autism Spectrum Disorder

A Multi-center, Randomized, Double-blind, Placebo-controlled, Therapeutic Exploratory Phase II Clinical Trial to Evaluate the Efficacy and Safety of NV01-A02 in Children With Autism Spectrum Disorder

This is a multicenter, randomized, double-blind, placebo-controlled, therapeutic exploratory Phase 2 clinical trial designed to evaluate the efficacy and safety of NV01-A02 in pediatric participants diagnosed with Autism Spectrum Disorder (ASD).

Study Overview

• Status: Recruiting
• Conditions: Autism Spectrum Disorder (ASD
• Intervention / Treatment: Drug: Drug: NV01-A02; Drug: Drug: Placebo (ZP8396)

• Study Type: Interventional
• Enrollment (Estimated): 105
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Neuroventi; Phone Number: +82 025405630; Email: dspark@neuroventi.com

Study Locations

• Korea, Republic of
  • 서울시
    • Seoul, 서울시, Korea, Republic of, 06100
      • Recruiting
      • Neuroventi
      • Contact: Neuroventi; Phone Number: +82 025405630; Email: dspark@neuroventi.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

-

Inclusion Criteria:

Children aged 6 to 15 years (inclusive) as of the date of written consent

Individuals who meet all the diagnostic criteria for Autism Spectrum Disorder (ASD) as outlined in the Diagnostic and Statistical Manual of Mental Disorders, 5th Edition (DSM-5)

DSM-5 Diagnostic Criteria for Autism Spectrum Disorder (ASD):

• Persistent deficits in social communication and social interaction across multiple contexts, as manifested by all of the following:

A. Deficits in social-emotional reciprocity

B. Deficits in nonverbal communicative behaviors used for social interaction

C. Deficits in developing, maintaining, and understanding relationships

② Restricted, repetitive patterns of behavior, interests, or activities, as manifested by at least two of the following:

A. Stereotyped or repetitive motor movements, use of objects, or speech

B. Insistence on sameness, inflexible adherence to routines, or ritualized patterns of verbal or nonverbal behavior

C. Highly restricted, fixated interests that are abnormal in intensity or focus

D. Hyper- or hyporeactivity to sensory input or unusual interest in sensory aspects of the environment

③ Symptoms must be present in the early developmental period (but may not become fully manifest until social demands exceed limited capacities, or may be masked by learned strategies later in life)

Exclusion Criteria:

• Individuals weighing less than 16 kg or more than 70 kg

Individuals identified during screening with any of the following medical histories, comorbid conditions, or surgical histories:

• Severe psychiatric disorders (other than autism spectrum disorder) that may affect participation in the clinical trial

  • History of organic brain disease, neurological disorders, or epilepsy/seizures that are not fully controlled by medication or non-pharmacologic surgical treatment (Note: Participants with a history of simple febrile seizures may be included at the investigator's discretion)

    • Severe peripheral arterial disease or coronary insufficiency

      • Presence or history of psychotic symptoms such as delusions or hallucinations (e.g., worsening or recurrence of psychiatric illness)

        • Gastrointestinal diseases (e.g., active peptic ulcers) or history of surgery that may affect absorption of the investigational medicinal product (Note: Participants with a history of simple appendectomy or hernia surgery may be included)

          • Individuals displaying serious self-injurious or aggressive behavior requiring medical intervention during the screening period, as determined by the investigator

            • Individuals with sensory abnormalities such as congenital hearing loss

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Drug: NV01-A02; Participants will receive different dose levels of the investigational drug. The drug will be administered orally once daily	Drug: Drug: NV01-A02; NV01-A02 will be administered orally at a different dose once daily for 8 weeks. The formulation is a white, film-coated tablet, designed for use in patients with mild to moderate cognitive impairment.
Placebo Comparator: Drug: Placebo (ZP8396); Participants will receive different dose levels of the investigational drug. The drug will be administered orally once daily	Drug: Drug: Placebo (ZP8396); ZP8396 is a matching placebo tablet administered orally once daily for 8 weeks. The placebo is identical in appearance to NV01-A02 but contains no active ingredient.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from baseline in the composite scores of the major domains of the Korean Vineland Adaptive Behavior Scales-II (K-Vineland-II) at Week 8 after administration.	Change from baseline in the composite scores of the major domains of the Korean Vineland Adaptive Behavior Scales-II (K-Vineland-II) at Week 8 after administration.* For children under 7 years of age, the composite score refers to the standard scores of all four major domains: Communication, Daily Living Skills, Socialization, and Motor Skills. For children aged 7 years and older, the composite score refers to the standard scores of the three domains excluding Motor Skills: Communication, Daily Living Skills, and Socialization.	From enrollment to the end of treatment at 8 weeks

Collaborators and Investigators

• Sponsor: Neuroventi Inc.

Study record dates

Study Major Dates

• Study Start (Actual): April 1, 2025
• Primary Completion (Estimated): September 1, 2025
• Study Completion (Estimated): December 1, 2026

Study Registration Dates

• First Submitted: April 23, 2025
• First Submitted That Met QC Criteria: April 23, 2025
• First Posted (Actual): April 30, 2025

Study Record Updates

• Last Update Posted (Actual): April 30, 2025
• Last Update Submitted That Met QC Criteria: April 23, 2025
• Last Verified: April 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Mental Disorders; Neurodevelopmental Disorders; Autism Spectrum Disorder; Autistic Disorder; Child Development Disorders, Pervasive
• Other Study ID Numbers: NV01-A02

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No