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:
Rhythm Pharmaceuticals, Inc.
Information provided by (Responsible Party):
Rhythm Pharmaceuticals, Inc.
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
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 |
| 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
MedlinePlus related topics:
Genes and Gene Therapy
Drug Information available for:
Setmelanotide
| 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 :
- 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 :
- 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 ]
- 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 ]
- 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 ]
- 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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| 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
| Contact: Rhythm Clinical Trials | (857) 264-4280 | clinicaltrials@rhythmtx.com | |
| Contact: Physician Inquiry: Clinical Trials | (857) 264-4280 | clinicaltrials@rhythmtx.com |
Locations
Show 35 study locations
Sponsors and Collaborators
Rhythm Pharmaceuticals, Inc.
Investigators
| Study Chair: | David Meeker, MD | Rhythm Pharmaceuticals, Inc. |
| 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 |
| 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:
|
Obesity Overnutrition Nutrition Disorders Overweight Body Weight |