Setmelanotide trial in BBS and AS patients with obesity resub
Research type
Research Study
Full title
A Phase 3 trial of Setmelanotide (RM-493), a Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström syndrome (AS) Patients with Moderate to Severe Obesity
IRAS ID
275153
Contact name
Philip Beales
Contact email
Sponsor organisation
Rhythm Pharmaceuticals, Inc.
Eudract number
2018-004058-11
Duration of Study in the UK
1 years, 9 months, 0 days
Research summary
Summary of Research
This is designed for patients with Bardet-Biedl Syndrome (BBS) or Alström syndrome (AS) which have Moderate to Severe Obesity and hyperphagia (increased appetite and hunger with impaired satiety).
Setmelanotide is a novel investigational drug, which works by activating neurons responsible for decreased appetite and weight. So far research results have demonstrated that the use of setmelanotide has been associated with significant weight and appetite reduction and that it has been well-tolerated.
It is anticipated that approximately 30 male and female patients 6 years of age and above (including at least 20 with BBS and at least 6 with AS) will be included in the study across approximately 10 centers worldwide. Patients under 16 years of age will not be enrolled in UK.
The study will consist of 3 treatment periods. Eligible patients will enter a 14-week, randomized, double-blind, placebo-controlled treatment period (Period 1) that will be followed by a 38-week open-label treatment period (Period 2) in which all patients will receive setmelanotide. The primary analysis will be performed after Period 2. Following Period 2, patients will continue to receive open-label setmelanotide for 14 weeks (Period 3) after which they may be enrolled into a separate treatment extension study.
The patients will daily receive sub-cutaneous injections of setmelanotide.
Study procedures primarily include frequent obesity measurements including hunger assessment, body composition assessments (including total body weight, fat mass, and non-bone lean mass) and waist circumference. Other study procedures include vital signs, collection of blood and urine samples, physical examination and ECG.
There is also optional sub-study to evaluate PK.
Summary of Results
The results of the study showed patients with BBS achieved clinically significant weight loss, reductions in hunger and improvements in health-related quality of life, all of which are highly meaningful to patients with BBS and their caregivers. The primary endpoint was the proportion of patients ≥12 years old in the full analysis set achieving ≥10% weight reduction after 52 weeks and this was achieved with patients having significant weight and hunger reductions. There were three evaluable patients with Alström syndrome and none of them achieved clinically significant weight loss.
The safety profile observed in this study was consistent with that observed with setmelanotide in previous clinical trials in patients with other rare MC4R pathway diseases.
REC name
Yorkshire & The Humber - Sheffield Research Ethics Committee
REC reference
19/YH/0429
Date of REC Opinion
16 Jan 2020
REC opinion
Further Information Favourable Opinion