A Trial of Setmelanotide in Patients With Congenital Hypothalamic Obesity (Sub-study of NCT05774756)

Phase 3

Not yet recruiting

• Conditions: Hypothalamic Obesity; Multiple Pituitary Hormone Deficiency Genetic Form; Septo-Optic Dysplasia; Optic Nerve Hypoplasia; Childhood-onset Combined Pituitary Hormone Deficiency; Pituitary Stalk Interruption Syndrome
• Interventions: Drug: Setmelanotide; Drug: Placebo

• Registration Number: NCT06760546
• Lead Sponsor: Rhythm Pharmaceuticals, Inc.

Brief Summary

This is a sub-study of Study RM-493-040 (NCT05774756).

The goal of this sub-study is to learn how well Setmelanotide works to improve weight reduction, hunger, and quality of life in patients 4 years of age and older with congenital Hypothalamic Obesity (cHO). To determine how well setmelanotide works and how safe it is, patients with cHO will take a daily injection of either setmelanotide or placebo and complete trial assessments for up to 26 weeks on a therapeutic regimen.

Detailed Description

Not available

Basic Information
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Recruitment & Eligibility
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Study & Design

Study Timeline

• Status Verified: 2024-12
• Study First Submitted: 2024-12-31
• Study First Submitted QC: 2024-12-31
• Last Update Submitted: 2024-12-31
• Study First Posted: 2025-01-07
• Last Update Posted: 2025-01-07
• Study Start: 2025-03-30
• Primary Completion: 2027-01-30
• Study Completion: 2027-01-30

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 39

Inclusion Criteria

1. Diagnosis of multiple pituitary hormone deficiency (MPHD), or septo-optic dysplasia (SOD), or optic nerve hypoplasia (ONH), or Childhood-onset combined pituitary hormone deficiency (CPHD), or Pituitary Stalk Interruption Syndrome (PSIS) with at least one pituitary deficiency 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
2. Age 4 years and older
3. Weight gain associated with the hypothalamic injury and a 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. Agree to use a highly effective form of contraception throughout the study and for 90 days after the study

Key

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Exclusion 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 last 2 years
4. Diagnosis of severe psychiatric disorders; any suicidal ideation, attempt or behavior
5. Current, clinically significant pulmonary, cardiac, metabolic, or oncologic disease
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. If female, pregnant and/or breastfeeding.
12. If receiving hormone replacement therapy, dose has remained stable for at least 2 months before Screening

Other protocol defined Inclusion/Exclusion criteria may apply.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Setemelanotide	Setmelanotide	Randomized 2:1 (Setmelanotide: Placebo)
Placebo	Placebo	Randomized 2:1 (Setmelanotide: Placebo)

Primary Outcome Measures

Name	Time	Method
Mean % change in BMI	From Baseline after 26 weeks on a therapeutic regimen

Secondary Outcome Measures

Name	Time	Method
Proportion of patients with ≥5% reduction in BMI in adult patients (≥18 years of age) or a BMI Z-score reduction of ≥0.2 points in pediatric patients (<18 years of age)	From Baseline after 26 weeks on a therapeutic regimen
Mean change in the weekly average of the daily most hunger score in patients ≥12 years old	From Baseline after 26 weeks on a therapeutic regimen
Proportion of patients with a ≥2 point reduction in the weekly average of the daily most hunger score	From Baseline after 26 weeks on a therapeutic regimen
Mean change in Symptoms of Hyperphagia total score	From Baseline after 26 weeks on a therapeutic regimen
Proportion of patients with a ≥10% reduction in BMI	From Baseline after 26 weeks on a therapeutic regimen
Mean percent change in weight in patients ≥18 years	From Baseline after 26 weeks on a therapeutic regimen
Mean BMI-z score and BMI percentile reduction in patients <18 (using combined height and weight to report BMI in kg/m2)	From Baseline after 26 weeks on a therapeutic regimen
Proportion of patients aged ≥4 to <18 years with ≥0.2-point reduction of BMI Z-score	From Baseline after 26 weeks on a therapeutic regimen
Proportion of patients with BMI <30 kg/m2 (patients aged ≥18 years) or <95th percentile (patients aged <18 years)	From Baseline after 26 weeks on a therapeutic regimen
Mean change in physical functioning score and total score for the Impact of Weight on Quality of Life-Lite (IWQOL)	From Baseline after 26 weeks on a therapeutic regimen
Safety and tolerability of setmelanotide compared to placebo assessed by frequency and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)	From Baseline after 26 weeks on a therapeutic regimen