A bounded review of sponsor-funded Phase 3 studies in rare genetic obesity syndromes: two small single-arm trials with withdrawal periods and one randomized trial followed by an open-label period.12
Bottom line from this bounded source set
The reviewed studies reported substantial weight reductions in some participants with specific genetic diagnoses. The cohorts were very small, and the Bardet-Biedl and Alström trial reported inconclusive results for the Alström subgroup.12
Reviewed study record
POMC- and LEPR-deficiency trials
Design
Two sponsor-funded, single-arm, open-label Phase 3 trials included a placebo-controlled withdrawal sequence for participants who first met a weight-loss threshold.1
Population
The trials enrolled ten participants with POMC-deficiency obesity and 11 with LEPR-deficiency obesity, all at least six years old.1
Outcomes reported
At approximately one year, at least 10% weight loss was reported for eight of ten participants in the POMC trial and five of 11 in the LEPR trial.1
Limitations
The cohorts were exceptionally small and lacked a parallel control group for the primary one-year endpoint; withdrawal eligibility also depended on an initial response.1
Reviewed study record
Bardet-Biedl and Alström syndrome trial
Design
A sponsor-funded Phase 3 trial began with a 14-week double-blind randomized placebo-controlled period and continued with a 52-week open-label period.2
Population
The trial randomized 38 participants aged six years or older with obesity and Bardet-Biedl syndrome or Alström syndrome.2
Outcomes reported
After 52 weeks of treatment, 32.3% of participants aged at least 12 years with Bardet-Biedl syndrome reached at least 10% weight reduction; results were reported as inconclusive for Alström syndrome.2
Limitations
The primary endpoint followed the open-label period, the syndrome-specific groups were small, and the reported result should not be generalized beyond the defined genetic populations.2
No registered Crossref update in the reviewed snapshotChecked August 19, 2026 for DOI 10.1016/s2213-8587(20)30364-8. This bounded result is not proof that the publication has never been corrected or retracted. Open the pinned Crossref work endpoint ↗
Publication update registered by CrossrefErratum ↗ · publisher metadata · February 1, 2023. Crossref metadata identifies the notice but does not by itself explain what wording changed.