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

A Blinded, Cross-Over Study to Evaluate the Safety and Efficacy of ARD-501 in Patients With Autism Spectrum Disorder

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

Study Overview

• Status: Completed
• Conditions: Autism Spectrum Disorder
• Intervention / Treatment: Drug: Low Dose ARD-501; Drug: Placebo; Drug: High Dose ARD-501

Detailed Description

This study is a blinded, placebo controlled, cross-over trial evaluating the safety and efficacy of two dose levels in subjects with Autism Spectrum Disorder.

A total of up to 12 subjects will be enrolled in the clinical study.

A screening process will be initiated upon completion of the informed consent process. Subjects will be assessed for eligibility through screening tests conducted within 28 days prior to enrollment. Following completion of screening tests and confirmation of eligibility, subjects will be enrolled to complete pre-dosing requirements and questionnaires during screening window as per protocol.

There are two phases. In Phase 1, all patients will be given ARD-501 at 0.2mg/kg, dependent on their body weight (BW), for 7 days followed by a 7-day washout period. Subsequently, in Phase 2, patients will be blinded and randomized at a 1:1 ratio in two groups. Each group will be exposed to ARD-501 at 0.5mg/kg BW and placebo in alternate order. Each dosing week is followed by a 7-day washout.

All available safety and tolerability data will be evaluated throughout study conduct.

• Study Type: Interventional
• Enrollment (Actual): 5
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Nevada
    • Las Vegas, Nevada, United States, 89128
      • Center For Psychiatry and Behavioral Medicine Inc

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

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).

Exclusion Criteria:

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.
• Women who are pregnant (i.e., have a positive pregnancy test), intending to become pregnant, breast feeding, or women of child-bearing potential who are unwilling to use contraception as required in the study inclusion criteria or maintain abstinence during the course of the study
• 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
• Patients weighing more than 275 lb (124.7 kg)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Crossover Assignment
• Masking: Double

Number of Arms

5

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Phase 1: Low Dose; ARD-501 for 7 days at 0.2 mg/kg	Drug: Low Dose ARD-501; Titratable, liquid formulation, taken orally. The intervention is given relative to the body weight of the individual. 0.2 mg/kg dosing.
Placebo Comparator: Phase 2: Placebo; Placebo for 7 days	Drug: Placebo; Titratable, liquid formulation, taken orally.
Experimental: Phase 2: High Dose; ARD-501 for 7 days at 0.5 mg/kg	Drug: High Dose ARD-501; Titratable, liquid formulation, taken orally. The intervention is given relative to the body weight of the individual. 0.5 mg/kg dosing.
Experimental: Phase 2: Crossover Placebo to High Dose; ARD-501 for 7 days at 0.5 mg/kg	Drug: High Dose ARD-501; Titratable, liquid formulation, taken orally. The intervention is given relative to the body weight of the individual. 0.5 mg/kg dosing.
Placebo Comparator: Phase 2: Crossover High Dose to Placebo; Placebo for 7 days	Drug: Placebo; Titratable, liquid formulation, taken orally.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Assessment of change in the Clinical Global Impression - Severity/Improvement (CGI-S/I) scale	To evaluate efficacy in subjects with ASD on 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)	To evaluate efficacy in social responsiveness in subjects with ASD on the Social Responsiveness Scale, Second Edition (SRS™-2, version-adjusted for age).	Baseline to Week 5

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Assessment of change in Gastrointestinal Severity Index (GSI)	To evaluate the efficacy of improvement in the gastrointestinal severity as assessed by the 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	To evaluate efficacy of improvement or 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)	To evaluate efficacy in adaptive behavior as assessed by change in the Adaptive Behavior Assessment System (ABAS-3)	Baseline to Week 5
Assessment of change on the Aberrant Behavior Checklist, Second Edition (ABC-2)	To evaluate efficacy in deviant interactions as assessed by change in the Aberrant Behavior Checklist (ABC-2)	Baseline to Week 5
Assessment of change on the Difficulties in Emotion Regulation Scale (DERS)	To evaluate efficacy in emotional response and management as assessed by change in the Difficulties in Emotion Regulation Scale (DERS)	Baseline to Week 5

Collaborators and Investigators

• Sponsor: Aardvark Therapeutics, Inc.
• Collaborators: Center for Psychiatry And Behavioral Medicine Inc.
• Investigators: Principal Investigator: Ann Childress, MD, Center For Psychiatry And Behavioral Medicine

Study record dates

Study Major Dates

• Study Start (Actual): August 1, 2024
• Primary Completion (Actual): January 13, 2025
• Study Completion (Actual): January 13, 2025

Study Registration Dates

• First Submitted: October 20, 2023
• First Submitted That Met QC Criteria: November 6, 2023
• First Posted (Actual): November 13, 2023

Study Record Updates

• Last Update Posted (Actual): May 9, 2025
• Last Update Submitted That Met QC Criteria: May 8, 2025
• Last Verified: January 1, 2025

More Information

Terms related to this study

• Keywords: ASD; Autism; High Opioid Tone
• Additional Relevant MeSH Terms: Mental Disorders; Neurodevelopmental Disorders; Autism Spectrum Disorder; Autistic Disorder; Child Development Disorders, Pervasive
• Other Study ID Numbers: AARD-501.2

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

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