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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04966741




Registration number
NCT04966741
Ethics application status
Date submitted
29/03/2021
Date registered
19/07/2021

Titles & IDs
Public title
Setmelanotide in Pediatric Participants With Rare Genetic Diseases of Obesity
Scientific title
A Phase 3 Multi-Center, 1-Year, Open-Label Study of Setmelanotide in Pediatric Patients Aged 2 to <6 Years of Age With Rare Genetic Causes of Obesity
Secondary ID [1] 0 0
RM-493-033
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Bardet-Biedl Syndrome 0 0
POMC Deficiency Obesity 0 0
PCSK1 Deficiency Obesity 0 0
LEPR Deficiency Obesity 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders
Diet and Nutrition 0 0 0 0
Obesity
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Setmelanotide

Experimental: Setmelanotide: PPL Group - Participants with POMC)/PCSK1/LEPR biallelic mutations collectively referred to as PPL received setmelanotide at a dose of 0.5 milligrams (mg) per day (QD) via SC injection for 52 weeks. The dose was escalated by increments of 0.5 mg every 2 weeks, if tolerated, at the dose escalation visits (Weeks 2, 4, and 6) to a maximum dose of 0.5 to 2.0 mg QD with the maximum dose based on body weight. Following the last dose in this study, participants who were considered likely to benefit from continued setmelanotide treatment and who had completed this trial could be eligible to enter an open-label long-term extension (LTE) trial with setmelanotide.

Experimental: Setmelanotide: BBS Group - Participants with BBS received setmelanotide at a dose of 0.5 mg QD via SC injection for 52 weeks. The dose was escalated by increments of 0.5 mg every 2 weeks, if tolerated, at the dose escalation visits (Weeks 2, 4, and 6) to a maximum dose of 0.5 to 2.0 mg QD with the maximum dose based on body weight. Following the last dose in this study, participants who were considered likely to benefit from continued setmelanotide treatment and who had completed this trial could be eligible to enter an open-label long-term extension (LTE) trial with setmelanotide.


Treatment: Drugs: Setmelanotide
SC injection once daily.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percentage of Participants With Greater Than or Equal to (=) 0.2 Reduction of BMI Z-Score From Baseline to Week 52
Timepoint [1] 0 0
Baseline up to Week 52
Primary outcome [2] 0 0
Mean Percent Change From Baseline in BMI
Timepoint [2] 0 0
Baseline, Week 52
Secondary outcome [1] 0 0
Mean Absolute Change From Baseline in BMI Z-score
Timepoint [1] 0 0
Baseline, Week 52
Secondary outcome [2] 0 0
Mean Change From Baseline in Percent of the 95th Percentile of BMI
Timepoint [2] 0 0
Baseline, Week 52
Secondary outcome [3] 0 0
Mean Change From Baseline in Bone Age
Timepoint [3] 0 0
Baseline, Week 52
Secondary outcome [4] 0 0
Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3)
Timepoint [4] 0 0
From Baseline to Week 52
Secondary outcome [5] 0 0
Change From Baseline in Body Weight
Timepoint [5] 0 0
Baseline, Week 52
Secondary outcome [6] 0 0
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Timepoint [6] 0 0
From first dose of study drug up to Week 56
Secondary outcome [7] 0 0
Number of Participants With TEAEs Graded by Severity
Timepoint [7] 0 0
From first dose of study drug up to Week 56

Eligibility
Key inclusion criteria
Key

1. Participants must have obesity due to either:

1. POMC, PCSK1, or LEPR deficiency, confirmed by genetic testing demonstrating biallelic variants that are interpreted as pathogenic, likely pathogenic, or of undetermined significance (VUS) by the American College of Medical Genetics and Genomics criteria (ACMG), or
2. BBS confirmed clinical and genetic diagnosis
2. Age between 2 to <6 years at the time of informed consent
3. Obesity, defined as body mass index (BMI) =97th percentile for age and gender and body weight of at least 15 kilograms (kg) at the time of enrollment.
4. Symptoms or behaviors of hyperphagia
5. Parent or guardian of study participant is able to understand and comply with the requirements of the study (including QD injection regimen and all other study procedures) and is able to understand and sign the written consent/assent.

Key
Minimum age
2 Years
Maximum age
5 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion Criteria

1. Glycated hemoglobin (HbA1c) >9.0% at screening
2. History of significant liver disease
3. Glomerular filtration rate (GFR) <60 milliliter per minute per 1.73 meter square (mL/min/1.73 m^2)
4. History or close family history of melanoma, or participant history of oculocutaneous albinism.
5. Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions (excluding non-invasive basal or squamous cell lesion)
6. Participation in any clinical study with an investigational drug/device within 3 months prior to the first day of dosing.
7. Previously enrolled in a clinical study involving setmelanotide or any previous exposure to setmelanotide.
8. Significant hypersensitivity to any excipient in the study drug.
9. Inadequate hepatic function
10. Any other uncontrolled endocrine, metabolic or medical condition(s) known to impact body weight

Other protocol defined Inclusion/Exclusion criteria may apply.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
8/03/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
8/11/2024
Sample size
Target
Accrual to date
Final
12
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Sydney Children's Hospital - Randwick
Recruitment postcode(s) [1] 0 0
NSW 2031 - Randwick
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Colorado
Country [2] 0 0
United States of America
State/province [2] 0 0
New York
Country [3] 0 0
United States of America
State/province [3] 0 0
Wisconsin
Country [4] 0 0
Spain
State/province [4] 0 0
Madrid
Country [5] 0 0
United Kingdom
State/province [5] 0 0
Cambridge

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Rhythm Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
David Meeker, MD
Address 0 0
Rhythm Pharmaceuticals, Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT04966741