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Setmelanotide in combination with diazoxide in patients with Prader-Willi syndrome.

30 patients around the world

Setmelanotide in combination with diazoxide in patients with Prader-Willi syndrome.

Available in Argentina

This is a 2-stage study designed to evaluate the efficacy and safety of setmelanotide in combination with diazoxide in patients with obesity due to Prader-Willi syndrome (PWS) who are between 12 and 65 years of age.

Worldwide Clinical Trials

1Research sites

30Patients around the world

This study is for people with

Rare diseases

Prader-willi disease

Obesity

Requirements for the patient

To 65 years

All Gender

Medical requirements

: Confirmed diagnosis of PWS as determined by the investigator at the time of selection.
Age between 12 and 65 years, inclusive, at the time of signing informed consent and/or assent.
Hyperphagia defined by a total score on the Hyperphagia Questionnaire, Clinical Trials (HQ-CT) of greater than or equal to 13 at selection.
BMI greater than or equal to 30 kg/m² for patients aged 18 years or older, or BMI equal to or less than 2 standard deviations above the WHO growth reference median for children aged 5 to 19 years.
The patient or the parent, guardian, or caregiver must be able to communicate well with the investigator, understand and comply with the trial requirements (including the administration of oral medications [BiD] and daily injection regimen [QD], as well as all other trial procedures), and understand written informed consent/assent. Patients who cannot comply with trial procedures should not be enrolled.
Able to comply with the contraceptive requirements described.
Living in a stable care environment (e.g., with parents or in chronic care facility) for at least 6 months prior to selection, with a caregiver who has cared for the patient for at least 6 months prior to selection.
Willing to sign informed consent/assent.
Current treatment with prior pituitary hormone therapies is allowed: Growth hormone therapy, Hypogonadism, Central hypothyroidism, Central adrenal insufficiency.

: 1. Weight loss greater than 3% in the past 3 months.
2. Metabolic and bariatric surgery (MBS) (e.g., gastric bypass/band/sleeve, duodenal switch, gastric balloon, intestinal barrier, etc.) in the past 6 months. All patients with a history of bariatric surgery or procedures must be reviewed with the sponsor prior to enrollment.
Any of the following abnormal laboratory values at screening:
1. Diabetes diagnosticada (tipo I ou II) ou glicose em jejum maior ou igual a 126 mg/dl (7 mmol/l) e/ou HbA1c maior ou igual a 6,5% na triagem
Estimated glomerular filtration rate (eGFR) less than 30 ml/min/1.73 m² at screening. In patients over 18 years old, the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation must be used to calculate eGFR.
c. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) greater than or equal to 3 ULN
d. Total bilirubin greater than or equal to 2 ULN
e. Free T4 and/or T4 outside the reference range
f. IGF-1 levels outside the reference range for sex, age, and pubertal stage
4. Documented diagnosis of major and unstable current psychiatric disorders (e.g., major depressive disorder, bipolar disorder, schizophrenia, etc.) or documented worsening of psychiatric condition that required changes in treatment regimen within the past 2 years, or other related psychiatric risks deemed by the investigator to interfere with trial compliance or patient safety.
5. Clinically significant depression or suicidal tendencies, defined by any suicidal ideation of type 4 or 5 on the Columbia Suicide Severity Rating Scale (C-SSRS) during screening, any lifetime suicide attempt, any suicidal behavior in the last month, or a score of 15 or greater on the Patient Health Questionnaire-9 (PHQ-9) during the screening process.
6. Current and clinically significant pulmonary, cardiovascular, endocrine/metabolic, hepatic, or oncologic disease that is considered serious enough to interfere with the trial and/or confound the results.
7. Systolic blood pressure greater than 160 mmHg and/or diastolic blood pressure greater than 100 mmHg in the supine position at the screening visit.
8. Significant current liver disease other than metabolic-associated fatty liver disease (MAFLD) or metabolic-associated steatohepatitis (MASH). Patients with MAFLD or MASH will not be excluded based on this criterion.
9. History or close family history (parents or siblings) of melanoma or history of oculocutaneous albinism in the patient.
10. Significant dermatological findings related to melanoma or premalignant skin lesions (excluding non-invasive basal cell or squamous cell lesions), determined as part of a comprehensive skin evaluation conducted by the investigator or designated person during screening. If concerning lesions are identified during screening, the patient must be excluded and the investigator should follow best practices for dermatology referral.
11. In the investigator's opinion, the patient is not suitable to participate in the trial.
12. Participation in any clinical trial with an investigational drug/device within 3 months or 5 half-lives, whichever is longer, before the first dose administration day.
13. Previously enrolled in a clinical trial involving setmelanotide or diazoxide/controlled-release diazoxide choline (DCCR) or any prior exposure to setmelanotide or diazoxide/DCCR.
14. Hypersensitivity to the active ingredient or any excipients of the investigational drugs (active and placebo), thiazides, or sulfonamides.
15. Pregnant or breastfeeding women, or those planning or wishing to become pregnant during the trial.

Sites

Instituto de Investigaciones Clínicas Quilmes

Recruiting

Sarmiento 315, Quilmes, Buenos Aires, Argentina

SponsorWorldwide Clinical Trials

Conditions

Prader-willi diseaseObesity

Requirements

To 65 yearsAll Gender

Unique study IDclinicaltrials.gov

NCT0000000