Open-Label Study of Setmelanotide in Hypothalamic Obesity
Study Details
Study Description
Brief Summary
Open-label, single-arm study designed to evaluate the body weight response to setmelanotide administered subcutaneously (SC) daily in patients with hypothalamic obesity (HO).
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 2 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Setmelanotide Dosage: 1.0, 2.0, 3.0 mg daily for patients 6 to <16 years of age, and 2.0 to 3.0 mg daily for patients ≥16 years of age. All patients will receive study treatment for 16 weeks. |
Drug: Setmelanotide
Investigational product: Setmelanotide for Subcutaneous (SC) injection.
|
Outcome Measures
Primary Outcome Measures
- Change in Body Weight Related Measure [16 weeks]
Proportion of patients with ≥5% reduction from baseline in BMI after 16 weeks of setmelanotide treatment compared to a historic control of <5% in this patient population.
Secondary Outcome Measures
- Change in body weight related composite measures [16 weeks]
Composite proportion of patients aged ≥6 to <18 years with ≥0.2 reduction of BMI Z-score and patients aged ≥18 years with 5% reduction of body weight from baseline after 16 weeks of setmelanotide.
- Change in body weight related measure in children and adolescents [16 weeks]
Proportion of patients aged ≥6 to <18 years with ≥0.2 reduction of BMI Z-score from baseline after 16 weeks of setmelanotide.
- Change in body weight related measure in adults [16 weeks]
Proportion of patients aged ≥18 years with ≥5% reduction of body weight from baseline after 16 weeks of setmelanotide.
Eligibility Criteria
Criteria
Inclusion Criteria:
Patients must meet all of the following criteria to be eligible for study participation:
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Patient has documented evidence of HO, including:
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Recent evidence of hypothalamic injury on magnetic resonance imaging (MRI); AND
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Diagnosis of craniopharyngioma or other non-malignant brain tumor affecting the hypothalamic region; AND
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Has undergone surgery, or chemotherapy, or radiation ≥6 months and ≤15 years before Screening.
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Patient has either unilateral hypothalamic lesions or bilateral hypothalamic lesions, as assessed by MRI.
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Aged 6 to 40 years.
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Obesity, documented by a BMI ≥35 kg/m2 for patients ≥18 years of age or BMI ≥95th percentile for age and gender for patients 6 to <18 years of age.
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Documented increase in BMI (change from pre-surgery baseline in BMI z-score ≥0.2 for patients <18 years of age or BMI >5% for patients >18 years of age) either during the first 6 months following surgery or within 1 year before surgery AND still present at Screening.
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More than 6 months after the end of post-tumor treatment, including chemotherapy, surgery, or radiation.
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Highly effective contraception throughout the study and for 90 days following the study.
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Ability to communicate well with the Investigator, understand and comply with the requirements of the study, and understand and sign the written informed consent, or, for patients aged <18 years, a parent/legal guardian that can sign.
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If receiving hormone replacement therapy, the dose of such therapy has remained stable for at least 2 months prior to Screening.
Exclusion Criteria:
Patients meeting any of the following criteria are not eligible for study participation:
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Weight gain >5% in the previous 3 months.
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Weight loss ≥2% in the previous 3 months.
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Note: Dietary and/or exercise regimens, with or without the use of medications, supplements, or herbal treatments associated with weight loss are allowed if:
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the regimen and/or dose has been stable for at least 3 months prior to randomization
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the patient has not experienced weight loss ≥2% during the previous 3 months, AND
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the patient intends to keep the regimen and/or dose stable throughout the course of the study.
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Bariatric surgery or procedure within the last 6 months.
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Diagnosis of severe psychiatric disorders
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HbA1c >10.0% at Screening.
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Current, clinically significant pulmonary, cardiac, or oncologic disease considered severe enough to interfere with the study and/or confound the results.
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Glomerular filtration rate (GFR) <30mL/min/1.73m^2 during Screening.
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Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions.
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History or close family history (parents or siblings) of skin cancer or melanoma
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Participation in any clinical study with an investigational drug/device within 3 months, prior to the first setmelanotide dose.
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Previously enrolled in a clinical study involving setmelanotide or any previous exposure to setmelanotide.
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Inability to comply with once daily (QD) injection regimen.
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Pregnant and/or breastfeeding, or desiring to become pregnant during this trial.
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Cognitive impairment that, in the Investigator's opinion, precludes participation to the study and completions of study procedures or questionnaires.
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Patient is, in Investigator's opinion, otherwise not suitable to participate in the study.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Rady Children's Hospital | San Diego | California | United States | 92123 |
| 2 | University of Florida | Gainesville | Florida | United States | 32610-0296 |
| 3 | Children's Minnesota | Saint Paul | Minnesota | United States | 55102 |
| 4 | Vanderbilt University School of Medicine | Nashville | Tennessee | United States | 37232 |
| 5 | Seattle Children's Research Institute | Seattle | Washington | United States | 98101 |
Sponsors and Collaborators
- Rhythm Pharmaceuticals, Inc.
Investigators
- Study Chair: Cecilia Scimia, MD, PhD, Rhythm Pharmaceuticals, Inc.
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- RM-493-030