A Phase 3, Double Blind, Randomized, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Setmelanotide in Patients with Acquired Hypothalamic Obesity

JRCT ID: jRCT2041230156

Registered date:22/02/2024

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Acquired hypothalamic obesity (HO)
• Date of first enrollment: 29/03/2024
• Target sample size: 12
• Countries of recruitment: Canada,Japan,Germany,Japan,Netherlands,Japan,United Kingdom,Japan,United States,Japan
• Study type: Interventional

Intervention(s)

Patients who are eligible to enter the trial will be randomized in a blinded manner on Day 1 in a 2:1 ratio, stratified by age group (>-18 years old, >-12 and <18 years old, <12 years old) and subpopulation (Japanese vs non-Japanese), to receive either setmelanotide (2) or placebo (1).
Setmelanotide 0.5 mg QD or placebo equivalent will be subcutaneous injection (SC) administered as the starting dose for all patients. After the initial dose of 0.5 mg, patients will be escalated each week in increments of either 0.5 or 1.0 mg to achieve the individual patient's therapeutic regimen.
All patients will receive trial treatment (setmelanotide or placebo, per randomization) for approximately 60 weeks.

Outcome(s)

• Primary Outcome: Mean % change in BMI from Baseline after approximately 52 weeks on a therapeutic regimen of setmelanotide compared to placebo

Key inclusion & exclusion criteria

• Age minimum: >= 4age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1.Patient has documented evidence of acquired HO
2.Aged 4 years and older
3.Documented weight gain associated with the hypothalamic injury either, and a body mass index (BMI) of >-30 kg/m2 for patients >-18 years of age, or BMI>-95th percentile for age and sex for patients 4 to <18 years of age
4.A highly effective form of contraception must be used/practiced throughout the trial and for 90 days
5.If receiving hormone replacement therapy, the dose of such therapy has remained stable for at least 2 months prior to Screening

Other protocol defined Inclusion criteria may apply.

Exclude criteria

1.Diagnosis of Prader-Willi syndrome (PWS) or Rapid-onset obesity with hypoventilation, hypothalamic, autonomic dysregulation, neuroendocrine tumor syndrome (ROHHADNET)
2.Weight loss >2% in the previous 3 months for patients aged >-18 years or >2% reduction in BMI for patients aged 4 to <18 years
3.Bariatric surgery or procedure within the last 2 years
4.Diagnosis of severe psychiatric disorders, or any suicidal ideation, attempts, or any suicidal
5.Current, clinically significant pulmonary, cardiac, metabolic, or oncologic disease considered
6.Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions (excluding non-invasive basal or squamous cell lesion)
7.History or close family history of skin cancer or melanoma
8.Participation in any clinical trial with an investigational drug/device within 3 months, prior to the first trial dose
9.Previously enrolled in a clinical trial involving setmelanotide or any previous exposure to setmelanotide
10.Inability to comply with once daily (QD) injection regimen
11.Pregnant and/or breastfeeding
12.Patients with obesity attributable to other genetic or syndromic conditions (eg, PPL [POMC, PCSK1, LEPR, collectively], BBS) prior to the hypothalamic injury

Other protocol defined Exclusion criteria may apply.

Related Information

• Primary Sponsor: Flaherty Jim
• Secondary ID(s): NCT05774756