DAYBREAK: Phase 2 Study in Patients With Specific Gene Defects in the MC4R Pathway
Study Details
Study Description
Brief Summary
2 stage (open-label run-in, followed by randomized, double-blind, placebo-controlled withdrawal), study trial of setmelanotide in patients with obesity and specific gene defects variants in the melanocortin-4 receptor (MC4R) pathway.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 2 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Active Comparator: Setmelanotide Dosage: Patients 12 years of age and older: Setmelanotide 2 mg once daily (QD) for approximately 14 days, then increased to setmelanotide 3 mg QD for the remainder of the study. Patients 6 to <12 years of age: Setmelanotide 1 mg QD for approximately 7 days, then increased to setmelanotide 2 mg QD for approximately 7 days, then increased to setmelanotide 3 mg QD for the remainder of the study. Mode of administration: -Subcutaneous (SC) injection |
Drug: Setmelanotide
Subcutaneous injection
|
| Placebo Comparator: Placebo Placebo (vehicle) in a sterile solution for injection |
Drug: Placebo
Subcutaneous injection
|
Outcome Measures
Primary Outcome Measures
- The proportion patients with obesity and genetic variants in the MC4R pathway who achieve a clinically meaningful reduction in body weight. [From baseline to End of Treatment, up to 40 weeks. Total participation in the study will last up to 52 weeks, including the Screening Period and the EOS Visit.]
To evaluate the proportion of patients with obesity and genetic variants in the MC4R pathway who achieve a clinically meaningful reduction in body weight in response to setmelanotide
Eligibility Criteria
Criteria
Inclusion Criteria:
- Patients must meet all of the following criteria to be eligible for study participation:
-
Patients must have a pre-identified genetic variant in an established MC4R pathway gene that contributes to obesity.
-
Ages of 6 and 65
-
Obesity, defined as BMI ≥40 kg/m2 for patients≥18 years of age or BMI ≥97th percentile for age and gender for patients 6 up to 17 years of age based on the US CDC criteria.
-
Study participant and/or parent or guardian is able to understand all study procedures
-
Use of highly effective contraception
Exclusion Criteria:
- Patients meeting any of the following criteria are not eligible for study participation:
-
Recent intensive diet and/or exercise regimen with or without the use of weight loss agents including herbal medications that has resulted in weight loss.
-
Use of any medication that is approved to treat obesity within 3 months of first dose of study drug.
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Bariatric surgery within the previous 6 months.
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Diagnosis of schizophrenia, bipolar disorder, personality disorder, major depressive disorder, or other psychiatric disorder(s) that the Investigator believes will interfere significantly with study compliance.
-
Any suicidal ideation of type 4 or 5 on the Columbia-Suicide Severity Rating Scale (C-SSRS) during Screening, any suicide attempt in the past 20 years or any suicidal behavior in the last month.
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Current, clinically significant pulmonary, cardiac, or oncologic disease considered severe enough to interfere with the study
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Has significant features of (or meets the diagnostic criteria for) a genetic syndrome that is associated with obesity
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HbA1C >10.0% at Screening.
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History of significant liver disease other than non-alcoholic fatty liver disease (NAFLD) or nonalcoholic steatohepatitis (NASH).
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Glomerular filtration rate (GFR) <60 mL/min at Screening.
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History or close family history (parents or siblings) of melanoma, or patient history of oculocutaneous albinism.
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Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions (excluding non-invasive basal or squamous cell lesion)
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Patient is, in the opinion of the Study Investigator, not suitable to participate in the study.
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Participation in any clinical study with an investigational drug/device within 3 months prior to the first day of dosing.
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Patients previously enrolled in a clinical study involving setmelanotide or any previous exposure to setmelanotide.
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Significant hypersensitivity to any excipient in the study drug.
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Females who are breastfeeding or nursing.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Metro Detroit Endocrinology Center | Dearborn | Michigan | United States | 48126 |
| 2 | Mayo Clinic | Rochester | Minnesota | United States | 55905 |
| 3 | Mississippi Center for Advanced Medicine | Madison | Mississippi | United States | 39110 |
| 4 | Ten's Medical Center - Pediatric Endocrinology Clinic | Staten Island | New York | United States | 10308 |
| 5 | University of North Carolina | Chapel Hill | North Carolina | United States | 27517 |
| 6 | Geisinger Clinic | Danville | Pennsylvania | United States | 17822 |
| 7 | Endocrine Associates of Dallas and Plano | Dallas | Texas | United States | 75231 |
| 8 | Accurate Clinical Research | Houston | Texas | United States | 77065 |
| 9 | Rio Grande Valley Endocrine Center | McAllen | Texas | United States | 78503 |
| 10 | Clinical Trials of Texas | San Antonio | Texas | United States | 78229 |
| 11 | Javara Research | The Woodlands | Texas | United States | 77382 |
| 12 | Hospital Sant Joan de Deu | Barcelona | Spain | ||
| 13 | Hospital General de Valencia | Valencia | Spain | ||
| 14 | University of Cambridge | Cambridge | United Kingdom |
Sponsors and Collaborators
- Rhythm Pharmaceuticals, Inc.
Investigators
- Study Chair: David Meeker, MD, Rhythm Pharmaceuticals, Inc.
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- RM-493-034