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

Phase 2

Not yet recruiting

• Conditions: SH2B1 Deficiency Obesity; Obesity Associated With PCSK1 Mutation (rs6232 Variant); SIM1 Deficiency Obesity
• Interventions: Drug: DCCR (diazoxide choline) extended-release tablets

• Registration Number: NCT05532020
• Lead Sponsor: Soleno Therapeutics, Inc.

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.

Study Timeline

• Status Verified: 2022-09
• Study First Submitted: 2022-09-04
• Study First Submitted QC: 2022-09-07
• Last Update Submitted: 2022-09-07
• Study First Posted: 2022-09-08
• Last Update Posted: 2022-09-08
• Study Start: 2023-01
• Primary Completion: 2024-01
• Study Completion: 2024-03

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 30

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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Open-label DCCR	DCCR (diazoxide choline) extended-release tablets	75 - 525 mg DCCR

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
Leptin	Baseline to Week 26	Change from Baseline in serum leptin
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
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)