A Study to Evaluate ARD-501 in Patients With Autism Spectrum Disorder

NCT06126653 | Completed Phase 2 FDA Drug

• 1 other identifier: AARD-501.2
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

Brief Summary

This is a blinded, placebo controlled, cross-over trial evaluating the safety of two dose-levels of ARD-501 in subjects with ASD.

Conditions

Autism Spectrum Disorder

Keywords

Autism; High Opioid Tone; ASD

Outcome Measures

Primary Outcomes (1)

• Assessment of the incidence of Treatment-Emergent Adverse Events (TEAE)

  To measure the efficacy evaluation of safety in subjects with autism spectrum disorder (ASD) by assessment of the incidence of Treatment-Emergent Adverse Events (TEAE).

  Baseline to Week 5

Secondary Outcomes (2)

• Assessment of change in the Clinical Global Impression - Severity/Improvement (CGI-S/I) scale

  Baseline to Week 5

• Assessment of change on the Social Responsiveness Scale, Second Edition (SRS™-2, version-adjusted for age)

  Baseline to Week 5

Other Outcomes (5)

• Assessment of change in Gastrointestinal Severity Index (GSI)

  Baseline to Week 5

• Assessment of change in Pain Detection Threshold (PDT) and Pain Tolerating Threshold (PTT) as measured during the Cold Pressor Test

  Baseline to Week 5

• Assessment of change on the Adaptive Behavior Assessment System (ABAS-3)

  Baseline to Week 5

Study Arms (5)

Phase 1: Low Dose

EXPERIMENTAL

ARD-501 for 7 days at 0.2 mg/kg

Drug: Low Dose ARD-501

Phase 2: Placebo

PLACEBO COMPARATOR

Placebo for 7 days

Drug: Placebo

Phase 2: High Dose

EXPERIMENTAL

ARD-501 for 7 days at 0.5 mg/kg

Drug: High Dose ARD-501

Phase 2: Crossover Placebo to High Dose

EXPERIMENTAL

ARD-501 for 7 days at 0.5 mg/kg

Drug: High Dose ARD-501

Phase 2: Crossover High Dose to Placebo

PLACEBO COMPARATOR

Placebo for 7 days

Drug: Placebo

Interventions

Low Dose ARD-501 DRUG

Titratable, liquid formulation, taken orally. The intervention is given relative to the body weight of the individual. 0.2 mg/kg dosing.

Phase 1: Low Dose

High Dose ARD-501 DRUG

Titratable, liquid formulation, taken orally. The intervention is given relative to the body weight of the individual. 0.5 mg/kg dosing.

Phase 2: Crossover Placebo to High Dose; Phase 2: High Dose

Placebo DRUG

Titratable, liquid formulation, taken orally.

Phase 2: Crossover High Dose to Placebo; Phase 2: Placebo

Eligibility Criteria

• Age: 17 Years - 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Subjects must meet all the following criteria to be eligible for participation in this study:
• Male and female subjects, 17-30 years of age
• Able and willing to sign consent and comply with study protocol
• Diagnostic confirmation of ASD as confirmed by gold standard clinical interview using Diagnostic and Statistical Manual of Mental Disorders 5th Edition (DSM-5) criteria and administration of the Autism Diagnostic Observation Schedule-2, Module 3 or 4.
• General good health as determined by physical exam, medical and psychiatric history and safety labs as defined by the Principal Investigator or designee.
• Male study participants who are sexually active with a female partner of childbearing potential must be surgically sterilized, or agree to use highly effective methods of birth control (defined below), and not rely on barrier methods and spermicide alone, from the time of screening until 1 week after final dose of study drug.
• Female participants of childbearing potential may be included in the study provided that they choose an effective contraception method that: 1) is not user dependent as permanent sterilization, intrauterine devices, and implants); or 2) is a user dependent short-acting hormonal method of contraception (e.g.injection, oral, transdermal, and intravaginal).

You may not qualify if:

• Subjects who meet any of the following criteria will be excluded from study participation:
• Allergy or hypersensitivity to ARD-501.
• Inability to swallow study drug.
• Unstable dosing of any mood, anxiety or behavior medications in 4 weeks prior to baseline visit.
• Concomitant use of scheduled benzodiazepines, baclofen, gabapentin, pregabalin, or supplements with impact on the γ-aminobutyric acid (GABA) system.
• Concomitant use of any cannabinoid or related product.
• Any use of opioid medication
• Unstable seizure disorder as defined by any seizure in the 6 months prior to baseline visit and/or a change in any anti-convulsant drug dosing in the 60 days prior to study screen.
• Abnormal baseline safety lab assessments including, but not limited to alanine transaminase (ALT) or aspartate aminotransferase (AST) greater than 1.5x the upper limit of normal, total bilirubin or creatinine greater than 1x the upper limit of normal, other clinically relevant lab abnormality, or abnormality in electrocardiogram (ECG), heart rate (HR), or blood pressure (BP) at screening as determined by the investigator or designee.
• History of or current abuse of drugs or alcohol including prescription medication.
• Inability to attend scheduled study visits, plans for family relocation during the study, or any other criteria that the investigator may determine to be associated with inability to complete the study
• History of major depressive disorder or history of other severe psychiatric disorders (e.g., schizophrenia or bipolar disorder) within the last 2 years.
• Suicidal ideas and behavior as assessed by the Columbia suicide severity scale (C-SSRS)
• Consumption of more than 2 units (males) or 1 unit (females) per day of alcohol during the study
• Any condition(s), including psychiatric disorders such as, but not restricted to bipolar disorders, that the investigator or primary physician believes may not be appropriate for participating the study

Sponsors & Collaborators

• Aardvark Therapeutics, Inc.: lead
• Center for Psychiatry And Behavioral Medicine Inc.: collaborator

Study Sites (1)

Center for Psychiatry and Behavioral Medicine Inc

Las Vegas, Nevada, 89128, United States

Location

MeSH Terms

Conditions

Autism Spectrum Disorder; Autistic Disorder

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: Double blinded
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: This is a blinded, placebo controlled, cross-over trial

Study Record Dates

• First Submitted: October 20, 2023
• First Posted: November 13, 2023
• Study Start: August 1, 2024
• Primary Completion: January 13, 2025
• Study Completion: January 13, 2025
• Last Updated: May 9, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)