ARD-101 for Prader-Willi Syndrome
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.12345Why 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.12567
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
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive ARD-101 in increasing doses over 4 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- ARD-101
Find a Clinic Near You
Who Is Running the Clinical Trial?
Aardvark Therapeutics, Inc.
Lead Sponsor
Children's Hospital Colorado
Collaborator
Stanford University
Collaborator