Prader-Willi SyndromePhase 2NCT05153434
A Study of Oral ARD-101 in Patients With Prader-Willi Syndrome
A Phase 2 study testing ARD-101 in people with Prader-Willi Syndrome, run by Aardvark Therapeutics, Inc. at 2 sites in United States.
Trial summary
A Phase 2, Single-Arm, Open-Label Study to Evaluate the Safety and Efficacy of ARD-101 in Patients with Prader-Willi Syndrome Read more about this study
- Who is it for?
- Ages 17 to 65 years
- Conditions the study is for
- Prader-Willi Syndrome
- What is being tested?
- ARD-101
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 19 participants
- Where
- 2 sitesUnited States
- Study dates
- 27 May 2022to 24 Sep 2024
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male and female subjects, 17-65 years of age
- Provide voluntary, written informed consent (parent(s) / legal guardian(s) of participant); provide voluntary, written assent (participants, as appropriate)
- PWS due to chromosome 15 micro-deletion, maternal uniparental disomy, or imprinting defect, confirmed by fluorescent in situ hybridization, chromosomal microarray, and/or methylation studies
- BMI ≥ 18.5 kg/m²
- Qualifying HQ-CT score
Who cannot join
- Use of weight loss agents, including herbal medication, within 3 months prior to enrollment
- Diagnosis of schizophrenia, bipolar disorder, personality disorder, or other DSM-III disorders which the investigator believes will interfere significantly with study compliance
- Clinically significant illness in the 8 weeks prior to enrollment
- Current, clinically significant liver, renal, pulmonary, cardiac, oncologic, or gastrointestinal (GI) disease
- Diagnosis of type 1 diabetes mellitus or other active endocrine disorders (e.g., Cushing syndrome, or thyroid dysfunction except if on stable adequate thyroid or glucocorticoid replacement supplement)
- Significant history of abuse of drugs within 1 year prior to enrollment or a positive Drugs of Abuse (DOA) test at screening
- History of alcohol abuse within 1 year prior to enrollment or currently drinks in excess of 21 units per week (3 servings or units/day)
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Children's Hospital Colorado
Aurora, Colorado, 80045
Stanford University
Palo Alto, California, 94304
About this study
Official title: A Phase 2, Single-Arm, Open-Label Study to Evaluate the Safety and Efficacy of ARD-101 in Patients With Prader-Willi Syndrome (PWS)
A Phase 2, Single-Arm, Open-Label Study to Evaluate the Safety and Efficacy of ARD-101 in Patients with Prader-Willi Syndrome
Read the detailed description
This is a Phase 2, open-label study to investigate the effects of ARD-101 in subjects with Prader-Willi Syndrome. The study will consist of a Screening Period (up to 28 days), a Treatment Period (28 days), and a Follow-up Period (End-of-Study Visit within 14 days after receiving the last dose of ARD-101). The screening procedures will be initiated upon completion of the informed consent process. Following completion of screening procedures and confirmation of eligibility, subjects will be enrolled to receive ARD-101 in an outpatient setting and will be instructed to visit the clinical center periodically for safety and efficacy assessments.
ARD-101 will be provided as a fixed dose of 200 mg BID for 28 days (Group 1) and then in a dose escalation of 1 week at 400 mg BID, 1 week at 600 mg BID, then 2 weeks at 800 mg BID (Group 2).
- Conditions
- Prader-Willi Syndrome
- Being tested
- ARD-101
- Medical subject headings
- Prader-Willi Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ARD-101Drug
Oral administration of ARD-101 taken BID (twice daily) for 28 days.
| Study group | Role | What they receive |
|---|---|---|
| ARD-101 (Fixed Dose) | Experimental | ARD-101: 4 weeks dosing at 200 mg BID (twice daily) Receives: Drug: ARD-101 |
| ARD-101 (Dose Escalation) | Experimental | ARD-101: 1 week at 400 mg BID (twice daily), second week at 600 mg BID, third and fourth weeks at 800 mg BID. Oral administration. Receives: Drug: ARD-101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment Emergent Adverse Events (TEAE)
The incidence of treatment emergent adverse events (TEAE) reported through the 28 days of treatment and the 14 days post treatment phase. TEAEs were those events occurring after treatment began.
Measured: Adverse Events were collected from time of informed consent through end of study; approximately 70 days (28 days of screening, 28 days of treatment, 14 days post treatment follow up).
Secondary outcomes (2)
Efficacy Evaluation of Hyperphagia in Prader-Willi Syndrome
Quantitative evaluation of hyperphagia via the Hyperphagia Questionnaire for Clinical Trials (HQ-CT). The HQCT is a 9-item hyperphasia questionnaire which is validated for use in PWS clinical trials. Score will range from 0 (no hyperphagia behaviors) to 36 (most severe hyperphagia behaviors)
Measured: Baseline, Day 15, Day 28
Change in Body Weight
Total weight change at the end of treatment (day 28) from baseline
Measured: Baseline to day 28
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Dec 2021
- Study start27 May 2022
- Primary completion24 Sep 2024
- Study completion24 Sep 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Prader-Willi SyndromeStudies 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
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- Aardvark Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Children's Hospital Colorado, Stanford University
- Responsible party
- Sponsor
- Investigators
- Shawn McCandless, MD, Principal investigator, Children's Hospital Colorado
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05153434
- Sponsor's protocol ID
- AARD-203
- Status last verified
- Sep 2026
- First submitted
- 30 Nov 2021
- Last updated
- 8 Sep 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- 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 8 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Single-Arm, Open-Label Study to Evaluate the Safety and Efficacy of ARD-101 in Patients With Prader-Willi Syndrome (PWS)
- ClinicalTrials.gov ID
- NCT05153434
- Acronym / sponsor ID
- AARD-203
- Conditions
- Prader-Willi Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 19 participants (actual)
- Lead sponsor
- Aardvark Therapeutics, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Children's Hospital Colorado, Stanford University
- Study start
- 27 May 2022 (actual)
- Primary completion
- 24 Sep 2024 (actual)
- Study completion
- 24 Sep 2024 (actual)
- First posted
- 10 Dec 2021 (actual)
- Results
- Posted 8 Sep 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A Phase 2, Single-Arm, Open-Label Study to Evaluate the Safety and Efficacy of ARD-101 in Patients with Prader-Willi Syndrome
Detailed description
This is a Phase 2, open-label study to investigate the effects of ARD-101 in subjects with Prader-Willi Syndrome. The study will consist of a Screening Period (up to 28 days), a Treatment Period (28 days), and a Follow-up Period (End-of-Study Visit within 14 days after receiving the last dose of ARD-101). The screening procedures will be initiated upon completion of the informed consent process. Following completion of screening procedures and confirmation of eligibility, subjects will be enrolled to receive ARD-101 in an outpatient setting and will be instructed to visit the clinical center periodically for safety and efficacy assessments.
ARD-101 will be provided as a fixed dose of 200 mg BID for 28 days (Group 1) and then in a dose escalation of 1 week at 400 mg BID, 1 week at 600 mg BID, then 2 weeks at 800 mg BID (Group 2).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ARD-101 (Fixed Dose) | Experimental | Drug: ARD-101ARD-101: 4 weeks dosing at 200 mg BID (twice daily) |
| ARD-101 (Dose Escalation) | Experimental | Drug: ARD-101ARD-101: 1 week at 400 mg BID (twice daily), second week at 600 mg BID, third and fourth weeks at 800 mg BID. Oral administration. |
Who may qualify
- Ages
- 17 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Male and female subjects, 17-65 years of age
- Provide voluntary, written informed consent (parent(s) / legal guardian(s) of participant); provide voluntary, written assent (participants, as appropriate)
- PWS due to chromosome 15 micro-deletion, maternal uniparental disomy, or imprinting defect, confirmed by fluorescent in situ hybridization, chromosomal microarray, and/or methylation studies
- BMI ≥ 18.5 kg/m²
- Qualifying HQ-CT score
Key exclusion criteria (7 listed)
- Use of weight loss agents, including herbal medication, within 3 months prior to enrollment
- Diagnosis of schizophrenia, bipolar disorder, personality disorder, or other DSM-III disorders which the investigator believes will interfere significantly with study compliance
- Clinically significant illness in the 8 weeks prior to enrollment
- Current, clinically significant liver, renal, pulmonary, cardiac, oncologic, or gastrointestinal (GI) disease
- Diagnosis of type 1 diabetes mellitus or other active endocrine disorders (e.g., Cushing syndrome, or thyroid dysfunction except if on stable adequate thyroid or glucocorticoid replacement supplement)
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 |
|---|---|---|
| ARD-101Oral administration of ARD-101 taken BID (twice daily) for 28 days. | Drug | ARD-101 (Dose Escalation), ARD-101 (Fixed Dose) |
Primary outcome
Incidence of Treatment Emergent Adverse Events (TEAE)
Time frame: Adverse Events were collected from time of informed consent through end of study; approximately 70 days (28 days of screening, 28 days of treatment, 14 days post treatment follow up).
The incidence of treatment emergent adverse events (TEAE) reported through the 28 days of treatment and the 14 days post treatment phase. TEAEs were those events occurring after treatment began.
2 secondary outcomes
Efficacy Evaluation of Hyperphagia in Prader-Willi Syndrome
Time frame: Baseline, Day 15, Day 28
Change in Body Weight
Time frame: Baseline to day 28
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Sep 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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.
Shawn McCandless, MD principal investigator
Children's Hospital Colorado
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ARD-101 in people with Prader-Willi Syndrome, run by Aardvark Therapeutics, Inc. at 2 sites 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 5 inclusion and 7 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 8 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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. 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?
Yes. Results are posted in the registry (first posted 8 Sep 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Incidence of Treatment Emergent Adverse Events (TEAE) (time frame: Adverse Events were collected from time of informed consent through end of study; approximately 70 days (28 days of screening, 28 days of treatment, 14 days post treatment follow up).). 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 8 Sep 2026.