Actively Recruiting

Phase 1
Phase 2
Age: 12Years - 65Years
All Genders
Healthy Volunteers
ID06239116

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

Led by Rhythm Pharmaceuticals, Inc. · Updated on 2025-12-22

150

Participants Needed

7

Research Sites

52 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

Researchers are studying RM-718 to assess its safety, tolerability, and how the body processes it in healthy individuals with obesity and in patients with impairments in the MC4R pathway. This study includes people aged 12 to 65 years and focuses on those with hypothalamic obesity and Prader-Willi Syndrome, conditions related to obesity caused by specific brain or genetic issues. The study has four parts: Part A tests single weekly doses of RM-718 or placebo in healthy obese adults aged 18 to 55; Part B tests multiple weekly doses in a similar group; Part C involves multiple weekly doses in patients aged 12 to 65 with hypothalamic obesity; and Part D gives multiple weekly doses to patients with Prader-Willi Syndrome aged 12 to 65. Doses are administered by weekly injections under the skin, and parts A and B are randomized, placebo-controlled, and double-blind, while parts C and D are open-label dose escalations. Participants will receive various weekly doses of RM-718 or placebo depending on their study part, with monitoring for side effects throughout. Researchers will assess safety by tracking adverse events during and after treatment, as well as measure how the drug behaves in the body over time. They will also monitor changes in body mass index, weight, waist size, and hunger symptoms in patients with hypothalamic obesity and Prader-Willi Syndrome. The study includes safety follow-up calls extending up to 210 days after dosing in some parts, with total participation lasting up to 26 weeks depending on the group.

CONDITIONS

Brief Title

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

Who Can Participate

Age: 12Years - 65Years
All Genders
Healthy Volunteers

Eligibility Criteria

Eligible

You may qualify if you...

  • For Parts A and B: Males and females aged 18 to 55 years in good health with a body mass index (BMI) of 30 kg/m2 or higher.
  • Healthy status with normal or clinically insignificant screening results.
  • Use of a highly effective form of contraception during the study.
  • Ability to communicate well, understand the trial, and provide informed consent in English.
  • For Part C: Males and females aged 12 to 65 years with hypothalamic obesity, documented by specific brain injury or treatment at least 6 months prior.
  • Weight gain associated with hypothalamic injury and BMI of 30 kg/m2 or higher for adults or above the 95th percentile for younger patients.
  • Use of a highly effective form of contraception during the study.
  • Ability to communicate and provide informed consent and assent as applicable.
  • For Part D: Patients aged 12 to 65 years with confirmed Prader-Willi Syndrome.
  • BMI of 30 kg/m2 or higher for adults or above the 95th percentile for younger patients.
  • Ability to meet contraception requirements.
Not Eligible

You will not qualify if you...

  • For Parts A and B: Significant abnormalities in labs or physical exams.
  • History or presence of serious medical conditions including kidney, liver, lung, heart, gastrointestinal, endocrine, immune, metabolic, neurological, or blood disorders.
  • Obesity caused by genetic, syndromic, or endocrine conditions.
  • History of renal transplant or end-stage kidney disease.
  • Severe psychiatric disorders.
  • Current severe pulmonary, cardiac, metabolic, or cancer diseases that might affect the trial.
  • Smoking or dependence on caffeine, alcohol, or drugs; inability to abstain 24 hours before and after visits.
  • Recent surgery within 60 days before screening.
  • Participation in another investigational drug or device trial within 3 months or 5 half-lives.
  • Pregnancy, breastfeeding, or desire to become pregnant during the trial.
  • For Part C: Diagnosis of Prader-Willi Syndrome or ROHHADNET.
  • Recent weight loss over 2% or use of anti-obesity medications.
  • Bariatric surgery within 2 years.
  • Severe psychiatric disorders or suicidal behavior.
  • History of renal transplant or end-stage kidney disease.
  • Prior participation in a trial with setmelanotide.
  • Obesity due to other genetic or syndromic causes before hypothalamic injury.
  • For Part D: Recent significant weight loss or therapies for obesity or hyperphagia.
  • Bariatric surgery within 6 months.
  • Severe psychiatric disorders or suicidal behavior.
  • Current severe pulmonary, cardiac, metabolic, or cancer diseases affecting the trial.
  • Pregnancy, breastfeeding, or desire to become pregnant during the trial.

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Treatment

Duration - Varies by part: single dose in Part A, 4 weeks in Part B, 16 weeks in Part C, and 26 weeks in Part D

Participants receive weekly subcutaneous injections of RM-718 or placebo depending on the study part and cohort. Part A involves a single dose; Part B involves 4 weekly doses; Part C involves 16 weekly doses; and Part D involves 26 weekly doses.

Weekly visits for dosing and assessments during treatment

Follow-up

Duration - Up to 43 days for Part A, up to 70 days for Part B, up to 140 days for Part C, and up to 210 days for Part D after treatment

Participants are monitored for safety and tolerability after completing treatment through follow-up calls and visits.

Follow-up visits or calls as scheduled after last dose

Trial Site Locations

Total: 7 locations

1

UAB Pediatric Endocrinology (Part C and Part D)

Birmingham, Alabama, United States, 35233

Actively Recruiting

2

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

Chicago, Illinois, United States, 60611

Actively Recruiting

3

Boston Children's Hospital (Part C only)

Boston, Massachusetts, United States, 021115

Actively Recruiting

4

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

Boston, Massachusetts, United States, 02115

Actively Recruiting

5

Vanderbilt University Medical Center (Part C only)

Nashville, Tennessee, United States, 37232

Actively Recruiting

6

Worldwide Clinical Trials (Part A and Part B)

San Antonio, Texas, United States, 78217

Completed

7

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

Salt Lake City, Utah, United States, 84112

Actively Recruiting

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Research Team

R

Rhythm Clinical Trials

P

Physician Inquiry Clinical Trials

How is the study designed?

Study Type

INTERVENTIONAL

Masking

TRIPLE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

17

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