RECRUITING

A Study of RM-718 in Healthy Subjects and Patients With MC4R Pathway Impairment

Conditions

Hypothalamic Obesity Prader-Willi Syndrome PWS melanocortin 4 receptor (MC4R)MC4R agonism obesity

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

The purpose of this study is to evaluate the safety, tolerability, and PK of RM-718 in healthy subjects with obesity and in patients with MC4R Pathway Impairment

Official Title

A Study of RM-718 Weekly Formulation in Healthy Subjects With Obesity and in Patients With Obesity Due to MC4R Impairment

Quick Facts

Study Start:2024-03-05

Study Completion:2028-11-01

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06239116

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:12 Years to 65 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:Yes

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
* Male and female subjects in good health aged 18-55 years of age at Screening. * Body mass index (BMI) ≥30 kg/m2. * Subjects who are medically healthy with normal or clinically insignificant screening results. * Subjects must use a highly effective form of contraception and follow the study contraception requirements. * Ability to communicate well with the Investigator, understand and comply with the requirements of the trial, and understand English and sign the written informed consent. * Male and female patients with HO, aged 12-65 years of age at Screening. * Patient has documented evidence of acquired HO defined as: * Diagnosis of craniopharyngioma or other brain lesion affecting the hypothalamic region and has undergone surgery, or chemotherapy, or radiation therapy involving the hypothalamus at least 6 months before Screening, OR * Documented injury to the hypothalamus at least 6 months before Screening for which surgery/radiation is not indicated. * Weight gain associated with the hypothalamic injury either before or following therapy (surgery and/or following chemotherapy or radiotherapy), and a BMI of ≥30 kg/m2 for patients ≥18 years of age or BMI ≥95th percentile for age and sex for patients 12 to \<18 years of age. * Patients must use a highly effective form of contraception and follow the study contraception requirements. * Ability to communicate with the Investigator, understand and comply with the requirements of the trial, and understand and sign the written informed consent and assent (for patients aged \<18 years), and informed consent for a parent or guardian of any patient \<18. * Confirmed diagnosis of PWS as determined by the Investigator at the time of Screening. * Age ≥12 to 65, inclusive, at the time of signing Informed Consent and/or Assent. * BMI ≥30 kg/m2 for patients ≥18 years of age or BMI ≥95th Percentile for age and sex for patients \<18 years of age based on the US CDC criteria. * Able to meet contraception requirements.	* Any clinically significant abnormalities on screening laboratories or physical examination as determined by the Investigator. * Active or history of any significant medical condition such as and including renal, hepatic, pulmonary, gastrointestinal, cardiovascular, genitourinary, endocrine, immunologic, metabolic, neurologic or hematological disease. * Obesity due to genetic, syndromic, or endocrine etiologies. * History of renal transplant, end stage renal disease. * Diagnosis of severe psychiatric disorders. * Current, clinically significant pulmonary, cardiac, metabolic, or oncologic disease considered severe enough to interfere with the trial and/or confound the results. * Cigarette smoking or dependence on caffeine, alcohol or drugs; unable or unwilling to abstain completely from caffeine, alcohol and related substances for 24 hours prior to and after study visits. * History of recent surgery (within 60 days of Screening). * Participation in any clinical trial with an investigational drug/device within 3 months or 5 half-lives, whichever is longer, prior to the first trial dose. * Pregnant and/or breastfeeding or desiring to become pregnant during this trial. * Diagnosis of Prader-Willi syndrome (PWS) or Rapid-onset obesity with hypoventilation, hypothalamic, autonomic dysregulation, neuroendocrine tumor syndrome (ROHHADNET). * Weight loss \>2% in the previous 3 months for patients aged ≥18 years or \>2% reduction in BMI for patients aged 12 to \<18 years and/or anti-obesity medications for the treatment of obesity. * Bariatric surgery or procedure within the last 2 years. * Diagnosis of severe psychiatric disorders; any suicidal ideation, attempt or behavior. * Current, clinically significant pulmonary, cardiac, metabolic, or oncologic disease considered severe enough to interfere with the trial and/or confound the results. * History of renal transplant, end stage renal disease. * Participation in any clinical trial with an investigational drug/device within 3 months or 5 half-lives, whichever is longer, prior to the first trial dose, or previous participation in a trial with setmelanotide. * Pregnant and/or breastfeeding or desiring to become pregnant during this trial. * Obesity attributable to other genetic or syndromic conditions (eg, PPL \[pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), leptin receptor (LEPR), collectively\], Bardet-Biedl syndrome \[BBS\]) prior to the hypothalamic injury. * Weight loss \>2% in the previous 3 months for patients aged ≥18 years or \>2% reduction in BMI for patients aged 12 to \<18 years or therapies for the treatment of obesity or hyperphagia. * Metabolic and bariatric surgery (MBS) or procedure within last 6 months. * Diagnosis of severe psychiatric disorders; any suicidal ideation, attempt or behavior. * Current, clinically significant pulmonary, cardiac, metabolic, or oncologic disease considered severe enough to interfere with the trial and/or confound the results. * Pregnant and/or breastfeeding or desiring to become pregnant during this trial.

Inclusion Criteria

Exclusion Criteria

* Male and female subjects in good health aged 18-55 years of age at Screening.
* Body mass index (BMI) ≥30 kg/m2.
* Subjects who are medically healthy with normal or clinically insignificant screening results.
* Subjects must use a highly effective form of contraception and follow the study contraception requirements.
* Ability to communicate well with the Investigator, understand and comply with the requirements of the trial, and understand English and sign the written informed consent.
* Male and female patients with HO, aged 12-65 years of age at Screening.
* Patient has documented evidence of acquired HO defined as:
* Diagnosis of craniopharyngioma or other brain lesion affecting the hypothalamic region and has undergone surgery, or chemotherapy, or radiation therapy involving the hypothalamus at least 6 months before Screening, OR
* Documented injury to the hypothalamus at least 6 months before Screening for which surgery/radiation is not indicated.
* Weight gain associated with the hypothalamic injury either before or following therapy (surgery and/or following chemotherapy or radiotherapy), and a BMI of ≥30 kg/m2 for patients ≥18 years of age or BMI ≥95th percentile for age and sex for patients 12 to \<18 years of age.
* Patients must use a highly effective form of contraception and follow the study contraception requirements.
* Ability to communicate with the Investigator, understand and comply with the requirements of the trial, and understand and sign the written informed consent and assent (for patients aged \<18 years), and informed consent for a parent or guardian of any patient \<18.
* Confirmed diagnosis of PWS as determined by the Investigator at the time of Screening.
* Age ≥12 to 65, inclusive, at the time of signing Informed Consent and/or Assent.
* BMI ≥30 kg/m2 for patients ≥18 years of age or BMI ≥95th Percentile for age and sex for patients \<18 years of age based on the US CDC criteria.
* Able to meet contraception requirements.

* Any clinically significant abnormalities on screening laboratories or physical examination as determined by the Investigator.
* Active or history of any significant medical condition such as and including renal, hepatic, pulmonary, gastrointestinal, cardiovascular, genitourinary, endocrine, immunologic, metabolic, neurologic or hematological disease.
* Obesity due to genetic, syndromic, or endocrine etiologies.
* History of renal transplant, end stage renal disease.
* Diagnosis of severe psychiatric disorders.
* Current, clinically significant pulmonary, cardiac, metabolic, or oncologic disease considered severe enough to interfere with the trial and/or confound the results.
* Cigarette smoking or dependence on caffeine, alcohol or drugs; unable or unwilling to abstain completely from caffeine, alcohol and related substances for 24 hours prior to and after study visits.
* History of recent surgery (within 60 days of Screening).
* Participation in any clinical trial with an investigational drug/device within 3 months or 5 half-lives, whichever is longer, prior to the first trial dose.
* Pregnant and/or breastfeeding or desiring to become pregnant during this trial.
* Diagnosis of Prader-Willi syndrome (PWS) or Rapid-onset obesity with hypoventilation, hypothalamic, autonomic dysregulation, neuroendocrine tumor syndrome (ROHHADNET).
* Weight loss \>2% in the previous 3 months for patients aged ≥18 years or \>2% reduction in BMI for patients aged 12 to \<18 years and/or anti-obesity medications for the treatment of obesity.
* Bariatric surgery or procedure within the last 2 years.
* Diagnosis of severe psychiatric disorders; any suicidal ideation, attempt or behavior.
* Current, clinically significant pulmonary, cardiac, metabolic, or oncologic disease considered severe enough to interfere with the trial and/or confound the results.
* History of renal transplant, end stage renal disease.
* Participation in any clinical trial with an investigational drug/device within 3 months or 5 half-lives, whichever is longer, prior to the first trial dose, or previous participation in a trial with setmelanotide.
* Pregnant and/or breastfeeding or desiring to become pregnant during this trial.
* Obesity attributable to other genetic or syndromic conditions (eg, PPL \[pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), leptin receptor (LEPR), collectively\], Bardet-Biedl syndrome \[BBS\]) prior to the hypothalamic injury.
* Weight loss \>2% in the previous 3 months for patients aged ≥18 years or \>2% reduction in BMI for patients aged 12 to \<18 years or therapies for the treatment of obesity or hyperphagia.
* Metabolic and bariatric surgery (MBS) or procedure within last 6 months.
* Diagnosis of severe psychiatric disorders; any suicidal ideation, attempt or behavior.
* Current, clinically significant pulmonary, cardiac, metabolic, or oncologic disease considered severe enough to interfere with the trial and/or confound the results.
* Pregnant and/or breastfeeding or desiring to become pregnant during this trial.

Contacts and Locations

Study Contact

Rhythm Clinical Trials

CONTACT

(857) 264-4280

clinicaltrials@rhythmtx.com

Physician Inquiry Clinical Trials

CONTACT

(857) 264-4280

clinicaltrials@rhythmtx.com

Principal Investigator

David Meeker

STUDY_CHAIR

Rhythm Pharmaceuticals, Inc.

Study Locations (Sites)

UAB Pediatric Endocrinology (Part C and Part D)

Birmingham, Alabama, 35233

United States

Ann and Robert H. Lurie Children's Hospital of Chicago (Part C and Part D)

Chicago, Illinois, 60611

United States

Boston Children's Hospital (Part C only)

Boston, Massachusetts, 021115

United States

Brigham and Women's Hospital (Part C and Part D)

Boston, Massachusetts, 02115

United States

Vanderbilt University Medical Center (Part C only)

Nashville, Tennessee, 37232

United States

Worldwide Clinical Trials (Part A and Part B)

San Antonio, Texas, 78217

United States

University of Utah Pediatric Endocrine Clinic (Part C and Part D)

Salt Lake City, Utah, 84112

United States

Collaborators and Investigators

Sponsor: Rhythm Pharmaceuticals, Inc.

David Meeker, STUDY_CHAIR, Rhythm Pharmaceuticals, Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-03-05

Study Completion Date2028-11-01

Study Record Updates

Study Start Date2024-03-05

Study Completion Date2028-11-01

Terms related to this study

Keywords Provided by Researchers

melanocortin 4 receptor (MC4R)
MC4R agonism
obesity

Additional Relevant MeSH Terms

Hypothalamic Obesity
Prader-Willi Syndrome
PWS