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EMANATE: A Study of Setmelanotide in Patients With Specific Gene Variants in the MC4R Pathway

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05093634
Recruitment Status : Recruiting
First Posted : October 26, 2021
Last Update Posted : January 27, 2023
Sponsor:
Information provided by (Responsible Party):
Rhythm Pharmaceuticals, Inc.

Brief Summary:

The protocol describes a randomized, double-blind, placebo-controlled trial with independent sub-studies of setmelanotide in patients with obesity and at least one of the specific gene variants in the Melanocortin-4 Receptor pathway:

  • POMC or PCSK1 (Sub-study 035a)
  • LEPR (Sub-study 035b)
  • SRC1 (Sub-study 035c)
  • SH2B1 (Sub-study 035d)

The objectives and endpoints are identical for these sub-studies.


Condition or disease Intervention/treatment Phase
Obesity Genetic Obesity Drug: Setmelanotide Drug: Placebo Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 400 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Trial: Multiple Independent Sub-studies of Setmelanotide in Patients With POMC/PCSK1, LEPR, NCOA1(SRC1), or SH2B1 Gene Variants in the Melanocortin-4 Receptor Pathway
Actual Study Start Date : December 10, 2021
Estimated Primary Completion Date : December 2024
Estimated Study Completion Date : December 2024

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: POMC or PCSK1 variant
1:1 Randomization
Drug: Setmelanotide
Subcutaneous Injection

Drug: Placebo
Subcutaneous Injection

Experimental: LEPR variant
1:1 Randomization
Drug: Setmelanotide
Subcutaneous Injection

Drug: Placebo
Subcutaneous Injection

Experimental: NCOA1 (SRC1) variant
1:1 Randomization
Drug: Setmelanotide
Subcutaneous Injection

Drug: Placebo
Subcutaneous Injection

Experimental: SH2B1 variant
1:1 Randomization
Drug: Setmelanotide
Subcutaneous Injection

Drug: Placebo
Subcutaneous Injection




Primary Outcome Measures :
  1. Difference in mean change in body weight in patients treated with setmelanotide compared to placebo, assessed as percent change in BMI [ Time Frame: Baseline to 52 weeks ]

Secondary Outcome Measures :
  1. The proportion of patients who achieve at least 5% reduction in BMI in patients treated with setmelanotide compared to placebo [ Time Frame: Baseline to 52 weeks ]
  2. The difference in mean change in body weight in adult patients treated with setmelanotide compared to placebo, assessed as percent change in baseline body weight [ Time Frame: Baseline to 52 weeks ]
  3. The difference in mean percent change in the weekly average most hunger score in patients treated with setmelanotide compared to placebo [ Time Frame: Baseline to 52 weeks ]
  4. The proportion of patients who achieve at least 10% reduction in BMI in patients treated with setmelanotide compared to placebo [ Time Frame: Baseline to 52 weeks ]


Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patients must have a pre-identified:

    • Heterozygous genetic variant in the POMC gene or PCSK1 gene
    • Heterozygous genetic variant in the LEPR gene
    • Homozygous, heterozygous, or compound heterozygous variant in the NCOA1 (SRC1)
    • Homozygous, heterozygous, or compound heterozygous variant in SH2B1 gene, or chromosomal 16p11.2 deletion encompassing the SH2B1 gene
  • Between 6 and 65 years of age at the time of provision of informed consent/assent
  • Obesity, defined as BMI ≥30 kg/m2 for patients ≥18 years of age or BMI ≥95th percentile for age and gender for patients 6 up to 17 years of age
  • Patient and/or parent or guardian is able to understand and comply with the requirements of the study and is able to understand and sign the written informed consent/assent
  • Patient and/or parent or guardian reports that patient experienced childhood obesity, defined as the patient and/or parent or guardian reporting that the patient was significantly overweight during childhood
  • Agree to use a highly effective form of contraception throughout the study and for 90 days following the study
  • Reported history of lifestyle intervention of diet and exercise
  • Reported history of hyperphagia

Key Exclusion Criteria:

  • Weight loss of 2% or greater in the previous 3 months
  • Recent history of bariatric surgery
  • Significant psychiatric disorder(s)
  • Suicidal ideation, attempt or behavior
  • Clinically significant pulmonary, cardiac, endocrine/metabolic, hepatic or oncologic disease
  • Glycated hemoglobin (HbA1C) >10% at Screening
  • History of significant liver disease or severe kidney disease
  • History or close family history (parents or siblings) of melanoma, or patient history of oculocutaneous albinism
  • Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions (excluding non-invasive basal or squamous cell lesion)
  • Participation in any clinical study with an investigational drug/device within 3 months prior to the first day of dosing
  • Previously enrolled in a clinical study involving setmelanotide or any previous exposure to setmelanotide
  • Significant hypersensitivity to any excipient in the study drug
  • If female, pregnant or breastfeeding

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05093634


Contacts
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Contact: Rhythm Clinical Trials (857) 264-4280 clinicaltrials@rhythmtx.com
Contact: Physician Inquiry: Clinical Trials (857) 264-4280 clinicaltrials@rhythmtx.com

Locations
Show Show 35 study locations
Sponsors and Collaborators
Rhythm Pharmaceuticals, Inc.
Investigators
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Study Chair: David Meeker, MD Rhythm Pharmaceuticals, Inc.
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Responsible Party: Rhythm Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT05093634    
Other Study ID Numbers: RM-493-035
First Posted: October 26, 2021    Key Record Dates
Last Update Posted: January 27, 2023
Last Verified: January 2023

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Keywords provided by Rhythm Pharmaceuticals, Inc.:
Melanocortin-4 Receptor Pathway
Genetic Obesity
Hunger
Hyperphagia
POMC
PCSK1
LEPR
SH2B1
NCOA1 (SRC1)
Additional relevant MeSH terms:
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Obesity
Overnutrition
Nutrition Disorders
Overweight
Body Weight