ARD-101 for Prader-Willi Syndrome

No longer recruiting at 1 trial location

Overseen By Andreas Niethammer, MD, PhD

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: Aardvark Therapeutics, Inc.

Must not be taking: Weight loss agents

Disqualifiers: Schizophrenia, Bipolar, Liver disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 15

Duration: 4 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called ARD-101, an experimental therapy, to evaluate its safety and effectiveness for individuals with Prader-Willi Syndrome, a genetic disorder affecting various aspects of life, including appetite and growth. Participants will gradually increase their dose of ARD-101 over four weeks to help researchers understand its impact. This trial suits those with Prader-Willi Syndrome confirmed by genetic testing and who have maintained a stable body weight for about two months. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

Do I need to stop my current medications to join the trial?

The trial requires that you stay on a stable dose of any allowed chronic medications, meaning no changes in medication or dose for at least 30 days before starting the study. If you're taking medications commonly used for Prader-Willi Syndrome, they must have been stable for more than 3 months.

Is there any evidence suggesting that ARD-101 is likely to be safe for humans?

Research shows that ARD-101 is generally safe and well-tolerated. Earlier studies found that ARD-101 primarily affects the gut but also impacts the whole body. It triggers hormone release in the gut. Previous research on healthy adults confirmed its safety and tolerability.

These findings suggest that ARD-101 could be a safe option for those considering a trial for Prader-Willi syndrome treatment. However, consulting a healthcare provider before joining a clinical trial is essential. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Prader-Willi Syndrome?

Most treatments for Prader-Willi Syndrome focus on controlling appetite and managing behavior through hormone replacement and appetite suppressants. But ARD-101 works differently, targeting the gut-brain axis to regulate hunger and metabolism more naturally. Researchers are excited because this unique mechanism could offer more balanced appetite control and potentially fewer side effects compared to existing options. Additionally, ARD-101's flexible dosing increases over four weeks, which might help in finding the optimal therapeutic effect for each individual.

What evidence suggests that ARD-101 might be an effective treatment for Prader-Willi Syndrome?

Research has shown that ARD-101, the investigational treatment in this trial, may help treat Prader-Willi Syndrome. In studies, 11 out of 12 participants reported feeling less extreme hunger, and four nearly stopped experiencing symptoms. Caregivers observed an average drop of 8.5 points on a hunger-related questionnaire, indicating reduced hunger. These results suggest that ARD-101 could effectively reduce hunger and improve the quality of life for people with Prader-Willi Syndrome. ^{1 2 5 6 7}

Are You a Good Fit for This Trial?

This trial is for individuals aged 17-65 with Prader-Willi Syndrome, a stable body weight, and no history of severe mental health issues or substance abuse. Participants must not be on insulin but can use certain other diabetes medications if doses have been stable. They should not have significant organ disease or recent medical treatments that could affect the study.

Inclusion Criteria

I was treated with ARD-101 and 3 months have passed since my last dose.

Your blood sugar level when you haven't eaten is less than 140 mg/dL.

You have a high score on a specific test called HQ-CT.

Exclusion Criteria

You have had serious thoughts about hurting yourself or trying to end your own life.

You have a PHQ-9 score of 10 or higher.

I have not had any serious illnesses in the last 8 weeks.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

4 weeks

1 visit (in-person)

Treatment

Participants receive ARD-101 in increasing doses over 4 weeks

4 weeks

Periodic visits for safety and efficacy assessments

Follow-up

Participants are monitored for safety and effectiveness after treatment

2 weeks

End-of-Study Visit within 14 days after last dose

What Are the Treatments Tested in This Trial?

Interventions

• ARD-101

Trial Overview

The trial is testing ARD-101, an oral medication for Prader-Willi Syndrome. It's a Phase 2 study where all participants receive the drug to see how safe it is and how well it works (since there's no comparison group).

How Is the Trial Designed?

Group I: ARD-101 Experimental Treatment 1 Intervention

First week 400 mg of ARD-101 twice daily, second week 600 mg of ARD-101 twice daily, third week 800 mg of ARD-101 twice daily, fourth week 800 mg of ARD-101 twice daily.

Who Is Running the Clinical Trial?

Aardvark Therapeutics, Inc.

Lead Sponsor

Trials

5

Recruited

150+

Children's Hospital Colorado

Collaborator

Trials

121

Recruited

5,135,000+

Stanford University

Collaborator

Trials

2,527

Recruited

17,430,000+

Citations

1.

ir.aardvarktherapeutics.com

ir.aardvarktherapeutics.com/news-releases/news-release-details/aardvark-therapeutics-reports-positive-phase-ii-clinical-data

Aardvark Therapeutics Reports Positive Phase II Clinical ...

ARD-101 shows strong efficacy signals including reduction of hunger and body fat composition in the treatment of Prader-Willi Syndrome, ...

2.

clinicaltrials.gov

clinicaltrials.gov/study/NCT05153434

A Study of Oral ARD-101 in Patients With Prader-Willi ...

This is a Phase 2, open-label study to investigate the effects of ARD-101 in subjects with Prader-Willi Syndrome.

3.

fpwr.org

fpwr.org/blog/exploring-the-hero-trial-investigating-ard-101-for-hyperphagia-in-pws

Exploring the HERO Trial: Investigating ARD‑101 ...

Promising Phase 2 Results · Out of 12 participants, 11 showed reduced hyperphagia, and 4 achieved near-complete symptom relief. · ARD‑101 also ...

4.

praderwillinews.com

praderwillinews.com/news/ard-101-phase-3-trial-expands-pws-patients-young-10/

ARD-101 Phase 3 trial expands to PWS patients as young ...

Results showed that caregivers reported an average 8.5-point drop on the Hyperphagia Questionnaire for Clinical Trials (HQ-CT), reflecting a ...

5.

clinicaltrialvanguard.com

clinicaltrialvanguard.com/news/aardvark-gets-fda-alignment-for-prader-willi-phase-3-trial/

Aardvark Gets FDA Alignment for Prader-Willi Phase 3 Trial

Aardvark Therapeutics lowered the minimum age of eligibility in its Phase 3 HERO trial of ARD-101 for hyperphagia in Prader–Willi syndrome ...

6.

clinicaltrials.gov

clinicaltrials.gov/study/NCT06828861

ARD-101 for Treatment of PWS: The Hunger Elimination or ...

The goal of this clinical trial is to learn if ARD-101 works to treat hyperphagia-related behavior in patients with Prader-Willi syndrome (PWS).

7.

praderwillinews.com

praderwillinews.com/ard-101-for-prader-willi-syndrome/

ARD-101 for Prader-Willi syndrome

After a Phase 1 clinical trial showed that ARD-101 was safe and well tolerated in healthy adults, the investigational treatment was tested in a ...