An Open-Label Study of Diazoxide Choline in Patients With Genetic Obesities (NCT05532020) | Clinical Trial Compass
UnknownPhase 2
An Open-Label Study of Diazoxide Choline in Patients With Genetic Obesities
30 participantsStarted 2023-01
Plain-language 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.
Who can participate
Age range5 Years
SexALL
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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
What they're measuring
1
Proportion of participants who achieve at least 5% body weight reduction