A Trial of Setmelanotide in Acquired Hypothalamic Obesity

Hypothalamic Obesity Clinical Trial

Official title:

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

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT05774756
• Other study ID #: RM-493-040
• Status: Enrolling by invitation
• Phase: Phase 3
• Start date: April 26, 2023
• Est. completion date: April 16, 2025

Study information

• Verified date: May 2024
• Source: Rhythm Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The goal of this trial 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 acquired Hypothalamic Obesity (HO). To determine how well setmelanotide works and how safe it is, patients with HO will take a daily injection of either setmelanotide or placebo and complete trial assessments for up to 60 weeks.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 120
• Est. completion date: April 16, 2025
• Est. primary completion date: April 16, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 4 Years and older

Eligibility

Key Inclusion Criteria:

1. Documented evidence of acquired hypothalamic obesity (HO)

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 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. Patients with obesity attributable to other genetic or syndromic conditions (eg, PPL [POMC, PCSK1, LEPR, collectively], BBS) prior to the hypothalamic injury.

13. If receiving hormone replacement therapy, dose has remained stable for at least 2 months before Screening

Other protocol defined Inclusion/Exclusion criteria may apply.

Related Conditions & MeSH terms

• Hypothalamic Obesity
• Obesity

Intervention

Drug:
• Setmelanotide
Solution for daily subcutaneous injection
• Placebo
Placebo matched to setmelanotide for daily subcutaneous injection

Locations

Country	Name	City	State
Canada	CIUSSS de l'Estrie-CHUS Endocrinology	Sherbrooke	Quebec
Canada	Hospital for Sick Children	Toronto	Ontario
Canada	BC Children's Hospital	Vancouver	British Columbia
Germany	Universitaetsklinikum Hamburg-Eppendorf (UKE) - Ambulanzzentrum des UKE GmbH	Hamburg
Germany	Medicover Neuroendokrinologie	München
Germany	Universitaetsklinikum Ulm - Klinik fuer Kinder- und Jugendmedizin	Ulm
Japan	Nagano Children's Hospital	Azumino City	Nagano
Japan	Chiba University Hospital	Chiba-shi	Chiba
Japan	Nagoya City University Hospital	Nagoya	Aichi
Netherlands	Prinses Maxima Center for Pediatric Oncology	Utrecht
United Kingdom	Hull University Teaching Hospital	Hull
United Kingdom	Great Ormond Street Hospital for Children	London
United Kingdom	UCL Great Ormond Street Institute of Child Health	London
United States	Children's Hospital Colorado	Aurora	Colorado
United States	UAN Pediatric Endocrinology	Birmingham	Alabama
United States	Ann and Robert H. Lurie Children's Hospital	Chicago	Illinois
United States	Ohio State Wexner Medical Center	Columbus	Ohio
United States	University of Florida	Gainesville	Florida
United States	University of Iowa Stead Family Department of Pediatrics	Iowa City	Iowa
United States	Vanderbilt University School of Medicine	Nashville	Tennessee
United States	Columbia University Irving Medical Center	New York	New York
United States	Icahn School of Medicine at Mount Sinai	New York	New York
United States	Lynn Health Science Institute	Oklahoma City	Oklahoma
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Children's Minnesota	Saint Paul	Minnesota
United States	Rady Children's Hospital	San Diego	California
United States	Seattle Children's Hospital, Research and Foundation - Center for Integrative Brain Research	Seattle	Washington

Sponsors (1)

Lead Sponsor	Collaborator
Rhythm Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  Canada,  Germany,  Japan,  Netherlands,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Mean % change in BMI		From Baseline to Week 60
Secondary	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 to Week 60
Secondary	Mean change in the weekly average of the daily most hunger score in patients =12 years old		From Baseline to Week 60
Secondary	Proportion of patients with a =2 point reduction in the weekly average of the daily most hunger score		From Baseline to Week 60
Secondary	Mean change in Symptoms of Hyperphagia total score		From Baseline to Week 60
Secondary	Proportion of patients with a =10% reduction in BMI		From Baseline to Week 60
Secondary	Mean percent change in weight in patients =18 years		From Baseline to Week 60
Secondary	Mean BMI-z score and BMI percentile reduction in patients <18 (using combined height and weight to report BMI in kg/m2)		From Baseline to Week 60
Secondary	Proportion of patients aged =4 to <18 years with =0.2-point reduction of BMI Z-score		From Baseline to Week 60
Secondary	Proportion of patients with BMI <30 kg/m2 (patients aged =18 years) or <95th percentile (patients aged <18 years)		From Baseline to Week 60
Secondary	Mean change in physical functioning score and total score for the Impact of Weight on Quality of Life-Lite (IWQOL)		From Baseline to Week 60
Secondary	Safety and tolerability of setmelanotide compared to placebo assessed by frequency and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)		From Baseline to Week 60