A study using RM-493 in patients with POMC deficiency obesity
Research type
Research Study
Full title
An Open Label, 1-Year Trial, including a Double-Blind Placebo-Controlled Withdrawal Period, of Setmelanotide (RM-493), a Melanocortin 4 Receptor (MC4R) Agonist, in Early Onset POMC Deficiency Obesity due to Bi-Allelic Loss-of-Function POMC or PCSK1 Genetic Mutation
IRAS ID
212603
Contact name
I. Sadaf Farooqi
Contact email
Sponsor organisation
Rhythm Pharmaceutical Inc.
Eudract number
2016-002320-83
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 0 months, 0 days
Research summary
This study is designed for patients with POMC and PCSK1 genetic deficiency obesity. This is an ultra- rare orphan condition characterised by severe and early onset of obesity due to abnormal increase of appetite and hunger leading to excessive food intake. There have been less than 25 cases currently described in the literature, with all cases diagnosed as children and with only 3 reaching adulthood thus far.
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.
A total of 10 patients will be recruited to receive daily sub-cutaneous injections of setmelanotide for approximately one year. The study will begin with an initial dose titration period of 2 to 12 weeks to find the optimal therapeutic dose for each individual patient. Thereafter, patients will continue on an open label treatment period and will receive their optimal individual therapeutic dose for an additional 10 weeks. Patients who demonstrate at least 5 kg weight loss at the end of the open label treatment period will continue onto the double-blind withdrawal period lasting 8 weeks. During this period, the patients will receive a 4-week period of placebo treatment. Following the withdrawal period, all patients will complete approximately 1 year of total therapeutic dosing.
Study procedures include vital signs, collection of blood and urine samples, physical examination, weight and waist circumference measurements, ECG, questionnaires and body composition assessments.
There are 5 optional sub-studies built in the protocol for additional tests, such as 24-hour PK collection and blood pressure monitoring, photographs, energy expenditure and skin colour assessment.The study sponsor is Rhythm Pharmaceutical, Inc.
REC name
East of England - Cambridge South Research Ethics Committee
REC reference
16/EE/0392
Date of REC Opinion
28 Nov 2016
REC opinion
Further Information Favourable Opinion