Obesity Associated With PCSK1 Mutation (rs6232 Variant), SH2B1 Deficiency Obesity, SIM1 Deficiency Obesity
Conditions
Keywords
SH2B1 deficiency obesity, Obesity associated with PCSK1 mutation, (rs6232 variant), SIM1 deficiency obesity, Genetic obesity
Brief summary
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.
Detailed description
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.
Interventions
DRUGDCCR (diazoxide choline) extended-release tablets
All participants will be titrated over a four week period to the maintenance dose. Participants will take DCCR daily for up to 52 weeks.
Sponsors
Soleno Therapeutics, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
5 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * Provide voluntary, written informed consent (parent(s) / legal guardian(s) of patient); provide voluntary, written assent (patients, as appropriate) * Genetically-confirmed diagnosis of SH2B1 deficiency obesity, obesity associated with PCSK1 mutation (rs6232 variant), or SIM1 deficiency obesity Key
Exclusion criteria
* Have participated in an interventional clinical study (i.e., investigational drug or device, approved drugs or device evaluated for unapproved use) within 60 days prior to Screening * Any known disease and/or condition, which would prevent, in the opinion of the Investigator, the patient from completing all study visits and assessments required by the protocol * Positive urine pregnancy test (in females of child-bearing potential) or females who are pregnant or breastfeeding, and/or plan to become pregnant or to breast-feed during or within 30 days after study participation
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of participants who achieve at least 5% body weight reduction | Baseline to Week 26 | Proportion of participants who achieve at least 5% body weight reduction |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Leptin | Baseline to Week 26 | Change from Baseline in serum leptin |
| Fasting serum insulin | Baseline to Week 26 | Change in fasting serum insulin |
| Body fat mass | Baseline to Week 26 | Change in body fat mass (DXA) |
| Feelings of hunger | Baseline to Week 26 | 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 |
Contacts
Primary ContactProject Manager
Outcome results
None listed