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Clinical Trial Details — Status: Enrolling by invitation

Administrative data

NCT number NCT05774756
Other study ID # RM-493-040
Secondary ID
Status Enrolling by invitation
Phase Phase 3
First received
Last updated
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
Health authority
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.

Study Design


Related Conditions & MeSH terms


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
See also
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