Trial results
AI generated results summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
According to the results reported on ClinicalTrials.gov, this trial (NCT04966741) enrolled 12 children in total — 7 with a condition called PPL (Pomc, Pcsk1, or Lepr deficiency) and 5 with a condition called BBS (Bardet-Biedl syndrome). Both are rare genetic conditions linked to obesity. The trial was testing a medicine called setmelanotide over 52 weeks (about one year), and the main things being measured were changes in body weight relative to height — specifically something called BMI (body mass index) and a related score that compares a child's BMI to others of the same age and sex. The reported data shows that, looking at the primary (main) measures after 52 weeks: in the PPL group, 85.7% of participants (roughly 6 out of 7) had a meaningful reduction in their BMI score relative to their peers, compared with 80% (4 out of 5) in the BBS group. In terms of overall BMI change, the PPL group showed an average reported reduction of about 25.6%, while the BBS group showed an average reported reduction of about 9.7%. For the secondary (additional) measures, the reported data shows that bone age — a measure of skeletal development taken from hand and wrist X-rays — increased by an average of about one year in the PPL group and 0.7 years in the BBS group over the study period. A child development questionnaire (the ASQ-3) was also completed; results for most participants in both groups were reported as remaining "above" the cutoff (meaning on track developmentally), with no data reported showing a shift to the lowest category for either group. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
View full results on ClinicalTrials.gov ↗ · Read the linked publication on PubMed (PMID 39549719) ↗
These are the results as reported to ClinicalTrials.gov, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.
Phase 3 Obesity Trial, Completed
voxsanity.com.au · Eligibility summary from public government registries · 18 August 2026 · not medical advice
Who may and may not be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who may be able to join
- The child has obesity caused by a specific genetic condition — either a confirmed change in the POMC, PCSK1, or LEPR gene, or a confirmed diagnosis of Bardet-Biedl Syndrome (BBS), shown through genetic testing.
- The child is between 2 and 5 years old (must not have turned 6 yet) at the time of signing up.
- The child has a BMI at or above the 97th percentile for their age and gender, and weighs at least 15 kilograms (about 33 pounds).
- The child shows signs of extreme, hard-to-control hunger (known as hyperphagia).
- A parent or guardian is able to understand and follow all study requirements, including giving a daily injection, and is able to sign the consent forms.
Who may not be able to join
Each point below is a reason the trial team may not be able to accept someone. It is not a list of requirements to meet.
- The child has a blood sugar control reading (HbA1c) above 9.0% at the screening visit.
- The child has a significant history of liver disease.
- The child's kidneys are not filtering blood well enough (confirm with trial site for exact values).
- The child or a close family member has a history of melanoma (a type of skin cancer), or the child has a condition called oculocutaneous albinism.
- The child has significant skin findings related to melanoma or pre-cancerous skin lesions (certain minor skin conditions may be exceptions — confirm with trial site).
- The child has taken part in another clinical trial involving an experimental drug or device within the past 3 months.
- The child has previously been in a study involving the drug being tested in this trial (setmelanotide), or has taken setmelanotide before.
- The child has a significant allergy or sensitivity to any ingredient in the study drug.
- The child has poor liver function.
- The child has another uncontrolled health or hormonal condition known to affect body weight.
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Trial contact details on record
Principal Investigator: David Meeker, MD, Rhythm Pharmaceuticals, Inc.
Australian sites
This trial is not accepting new participants. These are the contact details ClinicalTrials.gov holds for it, kept here for reference. They are not an invitation to enrol, and the sites listed may no longer be running this trial.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial was run
These are the locations recorded on the registry, including 1 in Australia. They are a historical record: this trial is not enrolling, so they are not places you can join it.
Primary endpoints
Percentage of Participants With Greater Than or Equal to (≥) 0.2 Reduction of BMI Z-Score From Baseline to Week 52; Mean Percent Change From Baseline in BMI
Other options
Data last synced from ClinicalTrials.gov: 18 August 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.