SH2B1 Deficiency ObesityPhase 2NCT05532020

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

A Phase 2 study testing DCCR (diazoxide choline) extended-release tablets in people with SH2B1 Deficiency Obesity and Obesity Associated With PCSK1 Mutation (rs6232 Variant), run by Soleno Therapeutics, Inc..

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record

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Trial 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. Read more about this study

Who is it for?: Ages 5 years and older
Conditions the study is for: SH2B1 Deficiency Obesity, Obesity Associated With PCSK1 Mutation (rs6232 Variant), SIM1 Deficiency Obesity
What is being tested?: DCCR (diazoxide choline) extended-release tablets
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 0 participants
Where: No sites listed yet
Study dates: Jan 2023to Mar 2024
Phase: Phase 2
Are there results?: None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 5 years and older
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

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

Who cannot join

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

About this study

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

Read the 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.

Conditions: SH2B1 Deficiency Obesity, Obesity Associated With PCSK1 Mutation (rs6232 Variant), SIM1 Deficiency Obesity
Being tested: DCCR (diazoxide choline) extended-release tablets
Keywords: SH2B1 deficiency obesity, Obesity associated with PCSK1 mutation, (rs6232 variant), SIM1 deficiency obesity, Genetic obesity

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

DCCR (diazoxide choline) extended-release tabletsDrug
Also called DCCR
All participants will be titrated over a four week period to the maintenance dose. Participants will take DCCR daily for up to 52 weeks.

Study groups in NCT05532020
Study group	Role	What they receive
Open-label DCCR	Experimental	75 - 525 mg DCCR Receives: Drug: DCCR (diazoxide choline) extended-release tablets

What is being measured

The primary outcome is the main question the study is designed to answer.

Proportion of participants who achieve at least 5% body weight reduction
Proportion of participants who achieve at least 5% body weight reduction
Measured: Baseline to Week 26

Secondary outcomes (4)

Leptin
Change from Baseline in serum leptin
Measured: Baseline to Week 26
Fasting serum insulin
Change in fasting serum insulin
Measured: Baseline to Week 26
Body fat mass
Change in body fat mass (DXA)
Measured: 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
Measured: Baseline to Week 26

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Jan 2023Mar 2024

First posted8 Sep 2022
Study startJan 2023 (estimated)
Primary completionJan 2024 (estimated)
Study completionMar 2024 (estimated)

Why it stopped: Company Decision

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for SH2B1 Deficiency ObesityStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 0 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT05532020
Sponsor's protocol ID: C615
Status last verified: Oct 2026
First submitted: 4 Sep 2022
Last updated: 13 Oct 2026
Results posted: No
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Study at a glance

From the registry record, last updated by the sponsor on 13 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

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
ClinicalTrials.gov ID: NCT05532020
Acronym / sponsor ID: C615
Conditions: 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
Phase: Phase 2
Study type: Interventional
Recruitment status: WithdrawnReason stopped: Company Decision
Enrolment: 0 participants (actual)
Lead sponsor: Soleno Therapeutics, Inc.Industry (pharma, biotech, devices)
Study start: Jan 2023 (estimated)
Primary completion: Jan 2024 (estimated)
Study completion: Mar 2024 (estimated)
First posted: 8 Sep 2022 (actual)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Open-label DCCR	Experimental	Drug: DCCR (diazoxide choline) extended-release tablets75 - 525 mg DCCR

Who may qualify

Ages: 5 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (2 listed)

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 (3 listed)

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

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
DCCR (diazoxide choline) extended-release tabletsAlso: DCCRAll participants will be titrated over a four week period to the maintenance dose. Participants will take DCCR daily for up to 52 weeks.	Drug	Open-label DCCR

Primary outcome

Proportion of participants who achieve at least 5% body weight reduction
Time frame: Baseline to Week 26
Proportion of participants who achieve at least 5% body weight reduction

4 secondary outcomes

Leptin
Time frame: Baseline to Week 26
Fasting serum insulin
Time frame: Baseline to Week 26
Body fat mass
Time frame: Baseline to Week 26
Feelings of hunger
Time frame: Baseline to Week 26

Results and safety

The study was withdrawn before enrolling participants, so there are no results.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 13 Oct 2026.
Individual participant dataSharing planned: No

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

Questions about this trial

What is this clinical trial studying?

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 2 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: withdrawn, as of the sponsor's last update on 13 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was withdrawn before enrolling participants, so there are no results.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Proportion of participants who achieve at least 5% body weight reduction (time frame: Baseline to Week 26). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05532020.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Oct 2026.