Autism Spectrum DisorderPhase 2NCT06126653
A Study to Evaluate ARD-501 in Patients With Autism Spectrum Disorder
A Phase 2 study testing Low Dose ARD-501 and High Dose ARD-501 in people with Autism Spectrum Disorder, run by Aardvark Therapeutics, Inc. at 1 site in United States.
Trial summary
This is a blinded, placebo controlled, cross-over trial evaluating the safety of two dose-levels of ARD-501 in subjects with ASD. Read more about this study
- Who is it for?
- Ages 17 to 30 years
- Conditions the study is for
- Autism Spectrum Disorder
- What is being tested?
- Low Dose ARD-501 and High Dose ARD-501
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Aug 2024to 13 Jan 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 17 to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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).
Who cannot join
- 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.
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- 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)
Trial locations and contacts
1 site in 1 country.
United States1 site
Center for Psychiatry and Behavioral Medicine Inc
Las Vegas, Nevada, 89128
About this study
Official title: 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.
Read the 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.
- Conditions
- Autism Spectrum Disorder
- Being tested
- Low Dose ARD-501, High Dose ARD-501, Placebo
- Keywords
- Autism, High Opioid Tone, ASD
- Medical subject headings
- Autism Spectrum Disorder, Autistic Disorder
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Low Dose ARD-501Drug
Titratable, liquid formulation, taken orally. The intervention is given relative to the body weight of the individual. 0.2 mg/kg dosing.
High Dose ARD-501Drug
Titratable, liquid formulation, taken orally. The intervention is given relative to the body weight of the individual. 0.5 mg/kg dosing.
PlaceboDrug
Titratable, liquid formulation, taken orally.
| Study group | Role | What they receive |
|---|---|---|
| Phase 1: Low Dose | Experimental | ARD-501 for 7 days at 0.2 mg/kg Receives: Drug: Low Dose ARD-501 |
| Phase 2: Placebo | Placebo comparator | Placebo for 7 days Receives: Drug: Placebo |
| Phase 2: High Dose | Experimental | ARD-501 for 7 days at 0.5 mg/kg Receives: Drug: High Dose ARD-501 |
| Phase 2: Crossover Placebo to High Dose | Experimental | ARD-501 for 7 days at 0.5 mg/kg Receives: Drug: High Dose ARD-501 |
| Phase 2: Crossover High Dose to Placebo | Placebo comparator | Placebo for 7 days Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: Baseline to Week 5
Secondary outcomes (2)
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
Measured: 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).
Measured: Baseline to Week 5
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Nov 2023
- Study start1 Aug 2024
- Primary completion13 Jan 2025
- Study completion13 Jan 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autism Spectrum DisorderStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- This is a blinded, placebo controlled, cross-over trial
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Aardvark Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Center for Psychiatry And Behavioral Medicine Inc.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06126653
- Sponsor's protocol ID
- AARD-501.2
- Status last verified
- Jan 2025
- First submitted
- 20 Oct 2023
- Last updated
- 9 May 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Blinded, Cross-Over Study to Evaluate the Safety and Efficacy of ARD-501 in Patients With Autism Spectrum Disorder
- ClinicalTrials.gov ID
- NCT06126653
- Acronym / sponsor ID
- AARD-501.2
- Conditions
- Autism Spectrum Disorder
- Keywords
- Autism, High Opioid Tone, ASD
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Aardvark Therapeutics, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Center for Psychiatry And Behavioral Medicine Inc.
- Study start
- 1 Aug 2024 (actual)
- Primary completion
- 13 Jan 2025 (actual)
- Study completion
- 13 Jan 2025 (actual)
- First posted
- 13 Nov 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a blinded, placebo controlled, cross-over trial evaluating the safety of two dose-levels of ARD-501 in subjects with ASD.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind (participant, investigator)
- Number of arms
- 5
- Model description
- This is a blinded, placebo controlled, cross-over trial
| Arm | Type | Receives |
|---|---|---|
| Phase 1: Low Dose | Experimental | Drug: Low Dose ARD-501ARD-501 for 7 days at 0.2 mg/kg |
| Phase 2: Placebo | Placebo comparator | Drug: PlaceboPlacebo for 7 days |
| Phase 2: High Dose | Experimental | Drug: High Dose ARD-501ARD-501 for 7 days at 0.5 mg/kg |
| Phase 2: Crossover Placebo to High Dose | Experimental | Drug: High Dose ARD-501ARD-501 for 7 days at 0.5 mg/kg |
| Phase 2: Crossover High Dose to Placebo | Placebo comparator | Drug: PlaceboPlacebo for 7 days |
Who may qualify
- Ages
- 17 to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (17 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Low Dose ARD-501Titratable, liquid formulation, taken orally. The intervention is given relative to the body weight of the individual. 0.2 mg/kg dosing. | Drug | Phase 1: Low Dose |
| High Dose ARD-501Titratable, liquid formulation, taken orally. The intervention is given relative to the body weight of the individual. 0.5 mg/kg dosing. | Drug | Phase 2: Crossover Placebo to High Dose, Phase 2: High Dose |
| PlaceboTitratable, liquid formulation, taken orally. | Drug | Phase 2: Crossover High Dose to Placebo, Phase 2: Placebo |
Primary outcome
Assessment of the incidence of Treatment-Emergent Adverse Events (TEAE)
Time frame: Baseline to Week 5
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).
2 secondary outcomes
Assessment of change in the Clinical Global Impression - Severity/Improvement (CGI-S/I) scale
Time frame: Baseline to Week 5
Assessment of change on the Social Responsiveness Scale, Second Edition (SRS™-2, version-adjusted for age)
Time frame: Baseline to Week 5
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 May 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Low Dose ARD-501 and High Dose ARD-501 in people with Autism Spectrum Disorder, run by Aardvark Therapeutics, Inc. at 1 site 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 7 inclusion and 17 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: finished, as of the sponsor's last update on 9 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Assessment of the incidence of Treatment-Emergent Adverse Events (TEAE) (time frame: Baseline to Week 5). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 9 May 2025.