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

Primary Purpose

SH2B1 Deficiency Obesity, Obesity Associated With PCSK1 Mutation (rs6232 Variant), SIM1 Deficiency Obesity

Status

Not yet recruiting

Phase

Phase 2

Locations

Study Type

Interventional

Intervention

DCCR (diazoxide choline) extended-release tablets

About this trial

This is an interventional treatment trial for SH2B1 Deficiency Obesity focused on measuring SH2B1 deficiency obesity, Obesity associated with PCSK1 mutation, (rs6232 variant), SIM1 deficiency obesity, Genetic obesity

Eligibility Criteria

5 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Open-label DCCR

Arm Description

75 - 525 mg DCCR

Outcomes

Primary Outcome Measures

Proportion of participants who achieve at least 5% body weight reduction

Secondary Outcome Measures

Leptin

Change from Baseline in serum leptin

Fasting serum insulin

Change in fasting serum insulin

Body fat mass

Change in body fat mass (DXA)

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

Full Information

NCT ID

NCT05532020

First Posted

September 4, 2022

Last Updated

September 7, 2022

Sponsor

Soleno Therapeutics, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT05532020

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

September 2022

Overall Recruitment Status

Not yet recruiting

Study Start Date

January 2023 (Anticipated)

Primary Completion Date

January 2024 (Anticipated)

Study Completion Date

March 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Soleno Therapeutics, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Product Manufactured in and Exported from the U.S.

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

SH2B1 Deficiency Obesity, Obesity Associated With PCSK1 Mutation (rs6232 Variant), SIM1 Deficiency Obesity

Keywords

SH2B1 deficiency obesity, Obesity associated with PCSK1 mutation, (rs6232 variant), SIM1 deficiency obesity, Genetic obesity

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

30 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Open-label DCCR

Arm Type

Experimental

Arm Description

75 - 525 mg DCCR

Intervention Type

Drug

Intervention Name(s)

DCCR (diazoxide choline) extended-release tablets

Other Intervention Name(s)

DCCR

Intervention Description

All participants will be titrated over a four week period to the maintenance dose. Participants will take DCCR daily for up to 52 weeks.

Primary Outcome Measure Information:

Title

Proportion of participants who achieve at least 5% body weight reduction

Description

Proportion of participants who achieve at least 5% body weight reduction

Time Frame

Baseline to Week 26

Secondary Outcome Measure Information:

Title

Leptin

Description

Change from Baseline in serum leptin

Time Frame

Baseline to Week 26

Title

Fasting serum insulin

Description

Change in fasting serum insulin

Time Frame

Baseline to Week 26

Title

Body fat mass

Description

Change in body fat mass (DXA)

Time Frame

Baseline to Week 26

Title

Feelings of hunger

Description

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

10. Eligibility

Sex

All

Minimum Age & Unit of Time

5 Years

Accepts Healthy Volunteers

Eligibility 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

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Project Manager

Phone

650-353-2051

C615ProjectManager@soleno.life

12. IPD Sharing Statement

Plan to Share IPD

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

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