An Open-Label Study of Diazoxide Choline in Patients With Genetic Obesities

An Open-Label Study to Assess the Preliminary Efficacy and Safety of DCCR (Diazoxide Choline) Extended-Release Tablets in Patients With Genetic Obesities

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.

Study Overview

• Status: Not yet recruiting
• Conditions: SH2B1 Deficiency Obesity; Obesity Associated With PCSK1 Mutation (rs6232 Variant); SIM1 Deficiency Obesity
• Intervention / Treatment: Drug: DCCR (diazoxide choline) extended-release tablets

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.

• Study Type: Interventional
• Enrollment (Anticipated): 30
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Project Manager; Phone Number: 650-353-2051; Email: C615ProjectManager@soleno.life

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Open-label DCCR; 75 - 525 mg DCCR	Drug: DCCR (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.; Other Names: DCCR

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of participants who achieve at least 5% body weight reduction	Proportion of participants who achieve at least 5% body weight reduction	Baseline to Week 26

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Leptin	Change from Baseline in serum leptin	Baseline to Week 26
Fasting serum insulin	Change in fasting serum insulin	Baseline to Week 26
Body fat mass	Change in body fat mass (DXA)	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	Baseline to Week 26

Collaborators and Investigators

• Sponsor: Soleno Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start (Anticipated): January 1, 2023
• Primary Completion (Anticipated): January 1, 2024
• Study Completion (Anticipated): March 1, 2024

Study Registration Dates

• First Submitted: September 4, 2022
• First Submitted That Met QC Criteria: September 7, 2022
• First Posted (Actual): September 8, 2022

Study Record Updates

• Last Update Posted (Actual): September 8, 2022
• Last Update Submitted That Met QC Criteria: September 7, 2022
• Last Verified: September 1, 2022

More Information

Terms related to this study

• Keywords: SH2B1 deficiency obesity; Obesity associated with PCSK1 mutation; (rs6232 variant); SIM1 deficiency obesity; Genetic obesity
• Additional Relevant MeSH Terms: Overnutrition; Nutrition Disorders; Overweight; Body Weight; Obesity; Molecular Mechanisms of Pharmacological Action; Antihypertensive Agents; Vasodilator Agents; Antimetabolites; Gastrointestinal Agents; Hypolipidemic Agents; Lipid Regulating Agents; Nootropic Agents; Lipotropic Agents; Diazoxide; Choline
• Other Study ID Numbers: C615

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No