Single-Patient trial aims to tackle obesity in rare bone disorder
NCT ID NCT07496463
First seen Jun 27, 2026 · Last updated Jul 24, 2026 · Updated 2 times
Summary
This phase 2 trial tests the drug setmelanotide in one person with pseudohypoparathyroidism type 1a (PHP1a), a rare genetic disorder that often causes severe obesity. The participant will receive daily injections of setmelanotide for six months to see if it leads to weight loss and improves metabolic health. The study is very early and small, so results will be preliminary.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Setmelanotide (a daily injection that targets the brain's appetite control pathway)
- What this could lead to
- If it works, this could point toward a treatment for obesity in people with pseudohypoparathyroidism type 1a, a rare genetic condition.
- What could go wrong
- This is a very early, single-patient study with no control group, so results may not apply to others. The drug may cause side effects like nausea or skin reactions.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
-
About 1 person
The number the study aims to enrol. It can still change while the study runs.
- Started
-
Jul 2026
- Expected to finish
-
Apr 2027
An estimate. End dates often move.
- Lead sponsor
-
Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
Children (under 18), adults (18 to 64) and older adults (65 and over)
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Known patient with PHP1a (confirmed GNAS mutation) * Optimized therapy for diabetes and dyslipidemia Exclusion Criteria: \- Use of medications that may affect endpoints that are changed within 3 months prior to Baseline or that are likely to require a change in dose during the open-label treatment period
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Obesity are added.
Genom att skicka in godkänner du våra Användarvillkor
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
-
The places running it
1 site. The list below names each one and where it is.
-
The official record
The full official record for this study. This one lists no contact details, but it is the first place any would appear.
-
A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
-
Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Custom knee guides put to the test in obese patients
- A pill for obesity? early human trial puts MDR-001 to the test
- Can a weekly shot help people who still struggle with weight after bariatric surgery?
- Do GLP-1 Weight-Loss drugs change how teens eat and feel?
- Can electrical muscle stimulation boost exercise benefits after menopause?
- Can a cold cloth on the neck calm Post-Surgery nausea?