This is a phase 2 open-label, one-arm, clinical study to evaluate the efficacy and safety of DCCR (diazoxide choline) extended-release tablets over 1 year of treatment, in patients with genetic obesities.
Patients aged 5 years and older with obesity due to SH2B1 deficiency obesity, obesity associated with PCSK1 mutation (rs6232 variant), or SIM1 deficiency obesity will be enrolled into this phase 2 open-label clinical trial at a single clinical center in North America. All eligible participants will receive DCCR (diazoxide choline) extended-release tablets for 1 year.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
30
All participants will be titrated over a four week period to the maintenance dose. Participants will take DCCR daily for up to 52 weeks.
Proportion of participants who achieve at least 5% body weight reduction
Proportion of participants who achieve at least 5% body weight reduction
Time frame: Baseline to Week 26
Leptin
Change from Baseline in serum leptin
Time frame: Baseline to Week 26
Fasting serum insulin
Change in fasting serum insulin
Time frame: Baseline to Week 26
Body fat mass
Change in body fat mass (DXA)
Time frame: Baseline to Week 26
Feelings of hunger
Change in self-reported feelings of hunger measured on a visual analog scale (VAS), 0 - 100 with 0 being no feelings of hunger and 100 being the most extreme sensation of hunger
Time frame: Baseline to Week 26
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