Hypothalamic ObesityPhase 1/2NCT06239116
A Study of RM-718 in Healthy Subjects and Patients With MC4R Pathway Impairment
A Phase 1/2 study testing Part C: RM-718 and Part D: RM-718 in people with Hypothalamic Obesity and Prader-Willi Syndrome, run by Rhythm Pharmaceuticals, Inc. at 7 sites in United States.
Trial summary
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 Read more about this study
- Who is it for?
- Ages 12 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Hypothalamic Obesity, Prader-Willi Syndrome, PWS
- What is being tested?
- Part C: RM-718 and Part D: RM-718
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 111 participants
- Where
- 7 sitesUnited States
- Study dates
- 5 Mar 2024to 1 Nov 2028
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 65 years
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Who may be able to join
- Parts A and B:
- 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.
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- Part C:
- 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.
- Part D:
- 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.
Who cannot join
- Parts A and B
- 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.
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- 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.
- Part C
- 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 use of 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.
- Part D
- 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.
- 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 use of setmelanotide.
- Other protocol defined Inclusion/Exclusion criteria may apply.
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
United States7 sites
UAB Pediatric Endocrinology (Part C and Part D)
Birmingham, Alabama, 35233
Boston Children's Hospital (Part C only)
Boston, Massachusetts, 021115
Brigham and Women's Hospital (Part C and Part D)
Boston, Massachusetts, 02115
Ann and Robert H. Lurie Children's Hospital of Chicago (Part C and Part D)
Chicago, Illinois, 60611
Vanderbilt University Medical Center (Part C only)
Nashville, Tennessee, 37232
University of Utah Pediatric Endocrine Clinic (Part C and Part D)
Salt Lake City, Utah, 84112
Worldwide Clinical Trials (Part A and Part B)
San Antonio, Texas, 78217
About this study
Official title: A Study of RM-718 Weekly Formulation in Healthy Subjects With Obesity and in Patients With Obesity Due to MC4R Impairment
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
Read the detailed description
This is a first-in-human and first-in-patient, 4-part study that includes the evaluation of safety, tolerability, and PK of: single ascending doses (SAD) of RM-718 weekly (RM-718) in healthy subjects 18 to 55 years of age with obesity (Part A), multiple ascending doses (MAD) of RM-718 in healthy subjects 18 to 55 years of age with obesity (Part B), MAD of RM-718 in patients 12 to 65 years of age with HO (Part C), and MAD of RM-718 in patients with PWS (Part D). Cohorts in Parts A and B are double-blind, placebo-controlled, and randomized 2:1 (4 subjects receive RM-718, 2 subjects receive placebo). Part C evaluates open-label dose escalation in patients 12 to 65 years of age with HO. Part D evaluates open-label dose escalation in patients 12 to 65 years of age. Study participants will receive: 1 weekly dose of either RM-718 or placebo in Part A, 4 weekly doses of either RM-718 or placebo in Part B,16 weekly doses of open-label RM-718 in Part C, and 26 weekly doses of RM-718 in Part D. Study drug (RM-718 or placebo) doses are administered weekly via subcutaneous injection.
- Conditions
- Hypothalamic Obesity, Prader-Willi Syndrome, PWS
- Being tested
- Part A: RM-718 or placebo (matched to specific RM-718 dose cohort), Part B: RM-718 or placebo (matched to specific RM-718 dose cohort), Part C: RM-718, Part D: RM-718
- Keywords
- melanocortin 4 receptor (MC4R), MC4R agonism, obesity
- Medical subject headings
- Sexual Infantilism, Prader-Willi Syndrome, Obesity
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Part A: RM-718 or placebo (matched to specific RM-718 dose cohort)Drug
Single ascending dose of RM-718 or placebo (matched to specific RM-718 Part A dose cohort)
Part B: RM-718 or placebo (matched to specific RM-718 dose cohort)Drug
Multiple ascending doses of RM-718 or placebo (matched to specific RM-718 Part B dose cohorts)
Part C: RM-718Drug
Multiple ascending doses of RM-718
Part D: RM-718Drug
Multiple ascending doses of RM-718
| Study group | Role | What they receive |
|---|---|---|
| RM-718 (Cohort A1) | Experimental | Single dose of RM-718 (4) or placebo (2) Receives: Drug: Part A: RM-718 or placebo (matched to specific RM-718 dose cohort) |
| RM-718 (Cohort A2) | Experimental | Single dose of RM-718 (4) or placebo (2) Receives: Drug: Part A: RM-718 or placebo (matched to specific RM-718 dose cohort) |
| RM-718 (Cohort A3) | Experimental | Single dose of RM-718 (4) or placebo (2) Receives: Drug: Part A: RM-718 or placebo (matched to specific RM-718 dose cohort) |
| RM-718 (Cohort A4) | Experimental | Single dose of RM-718 (4) or placebo (2) Receives: Drug: Part A: RM-718 or placebo (matched to specific RM-718 dose cohort) |
| RM-718 (Cohort A5) | Experimental | Single dose of RM-718 (4) or placebo (2) Receives: Drug: Part A: RM-718 or placebo (matched to specific RM-718 dose cohort) |
| RM-718 (Cohort A6) | Experimental | Single dose of RM-718 (4) or placebo (2) Receives: Drug: Part A: RM-718 or placebo (matched to specific RM-718 dose cohort) |
| RM-718 (Cohort A7) | Experimental | Single dose of RM-718 (4) or placebo (2) Receives: Drug: Part A: RM-718 or placebo (matched to specific RM-718 dose cohort) |
| RM-718 (Cohort A8) | Experimental | Single dose of RM-718 (4) or placebo (2) Receives: Drug: Part A: RM-718 or placebo (matched to specific RM-718 dose cohort) |
| RM-718 (Cohort A9) | Experimental | Single dose of RM-718 (4) or placebo (2) Receives: Drug: Part A: RM-718 or placebo (matched to specific RM-718 dose cohort) |
| RM-718 (Cohort B1) | Experimental | Multiple ascending doses of RM-718 (4) or placebo (2) Receives: Drug: Part B: RM-718 or placebo (matched to specific RM-718 dose cohort) |
| RM-718 (Cohort B2) | Experimental | Multiple ascending doses of RM-718 (4) or placebo (2) Receives: Drug: Part B: RM-718 or placebo (matched to specific RM-718 dose cohort) |
| RM-718 (Cohort B3) | Experimental | Multiple ascending doses of RM-718 (4) or placebo (2) Receives: Drug: Part B: RM-718 or placebo (matched to specific RM-718 dose cohort) |
| RM-718 (Cohort B4) | Experimental | Multiple ascending doses of RM-718 (4) or placebo (2) Receives: Drug: Part B: RM-718 or placebo (matched to specific RM-718 dose cohort) |
| RM-718 (Cohort B5) | Experimental | Multiple ascending doses of RM-718 (4) or placebo (2) Receives: Drug: Part B: RM-718 or placebo (matched to specific RM-718 dose cohort) |
| RM-718 (Cohort B6) | Experimental | Multiple ascending doses of RM-718 (4) or placebo (2) Receives: Drug: Part B: RM-718 or placebo (matched to specific RM-718 dose cohort) |
| RM-718 (Cohort C1) | Experimental | Multiple ascending doses of RM-718 (30) Receives: Drug: Part C: RM-718 |
| RM-718 (Cohort D1) | Experimental | Multiple ascending doses of RM-718 (up to 30) Receives: Drug: Part D: RM-718 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Parts A, B, C, D: Safety and Tolerability Assessed by Number of Study Participants with Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Measured: From Day 1 through the Safety-Follow-up call (up to Day 43 for all Part A cohorts, up to Day 70 for all Part B cohorts, up to Day 140 for Part C cohort, up to Day 210 for Part D cohort)
Secondary outcomes (24)
AUCtau measurement of RM-718
Area under the concentration versus time curve during a dosing interval
Measured: up to 168 hours post-dose on Day 1 (Parts A, B and C) and 168 hours post-dose on Day 22 (Parts B and C).
Cmax measurement of RM-718
Maximum concentration measurement of RM-718 in plasma
Measured: up to 168 hours post-dose on Day 1 (Parts A, B and C) and 168 hours post-dose on Day 22 (Parts B and C).
Cmin measurement of RM-718
Minimum plasma concentration of RM-718 reached during dosing interval
Measured: up to 168 hours post-dose on Day 1 (Parts A, B and C) and 168 hours post-dose on Day 22 (Parts B and C).
Tmax measurement of RM-718
Time it takes for RM-718 to reach the maximum concentration (Cmax)
Measured: up to 168 hours post-dose on Day 1 (Parts A, B and C) and 168 hours post-dose on Day 22 (Parts B and C).
Tmin measurement of RM-718
Time at which the lowest concentration value of RM-718 is observed
Measured: up to 168 hours post-dose on Day 1 (Parts A, B and C) and 168 hours post-dose on Day 22 (Parts B and C).
Ctrough measurement of RM-718
Observed pre-dose plasma concentration of RM-718
Measured: up to 168 hours post-dose on Day 1 (Parts A, B and C) and 168 hours post-dose on Day 22 (Parts B and C).
Cavg measurement of RM-718
Average concentration of RM-718 during a dosing interval in steady state
Measured: up to 168 hours post-dose on Day 1 (Parts A, B and C) and 168 hours post-dose on Day 22 (Parts B and C).
t1/2 measurement of RM-718
Terminal elimination half-life of RM-718 in plasma
Measured: up to 168 hours post-dose on Day 1 (Parts A, B and C) and 168 hours post-dose on Day 22 (Parts B and C).
λz measurement of RM-718
Estimate of the terminal elimination rate constant of RM-718
Measured: up to 168 hours post-dose on Day 1 (Parts A, B and C) and 168 hours post-dose on Day 22 (Parts B and C).
CL/F measurement of RM-718
Clearance of RM-718 following extravascular administration
Measured: up to 168 hours post-dose on Day 1 (Parts A, B and C) and 168 hours post-dose on Day 22 (Parts B and C).
Vz/F measurement of RM-718
Volume of distribution of RM-718 following extravascular administration
Measured: up to 168 hours post-dose on Day 1 (Parts A, B and C) and 168 hours post-dose on Day 22 (Parts B and C).
Accumulation ratio of RM-718
Ratio of accumulation of RM-718 under steady state conditions
Measured: Week 1 to Week 4 (AUC on Week 4/AUC on Week 1) (Parts B, C)
Change from baseline in BMI (Part C only)
Measured: Baseline to Week 16
Mean change in weight (Part C only)
Measured: Baseline to Week 16
Mean change in waist circumference (Part C only)
Measured: Baseline to Week 16
Mean change in weekly average of the daily most hunger score in patients ≥12 years of age (Part C only)
Measured: Baseline to Week 16
Mean change in weekly average of the Symptoms of Hyperphagia composite score (Part C only)
Measured: Baseline to Week 16
Ctrough measurement of RM-718 (Part D)
Observed pre-dose plasma concentration of RM-718
Measured: up to 168 hours post-dose on Day 8 and up to 168 hours post-dose on Day 29
Change from baseline in BMI (Part D)
Measured: Baseline to Week 26
Mean change in weight (Part D)
Measured: Baseline to Week 26
Mean change in waist circumference (Part D)
Measured: Baseline to Week 26
Mean change in the weekly average of the Prader-Willi Syndrome Food Problem Diary (PWS-FPD) total score (Part D)
Measured: Baseline to Week 26
Mean change in the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) total score (Part D)
Measured: Baseline to Week 26
Change in total body mass (Part D)
Change in body mass as measured by dual-energy x-ray
Measured: Baseline to Week 26
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Feb 2024
- Study start5 Mar 2024
- Primary completion7 May 2027 (estimated)
- Study completion1 Nov 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hypothalamic ObesityStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 111 participants (actual)
Sponsor and investigators
- Lead sponsor
- Rhythm Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- David Meeker, Study chair, Rhythm Pharmaceuticals, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06239116
- Sponsor's protocol ID
- RM-718-001
- Status last verified
- Oct 2026
- First submitted
- 25 Jan 2024
- Last updated
- 9 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study of RM-718 Weekly Formulation in Healthy Subjects With Obesity and in Patients With Obesity Due to MC4R Impairment
- ClinicalTrials.gov ID
- NCT06239116
- Acronym / sponsor ID
- RM-718-001
- Conditions
- Hypothalamic Obesity, Prader-Willi Syndrome, PWS
- Keywords
- melanocortin 4 receptor (MC4R), MC4R agonism, obesity
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 111 participants (actual)
- Lead sponsor
- Rhythm Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 5 Mar 2024 (actual)
- Primary completion
- 7 May 2027 (estimated)
- Study completion
- 1 Nov 2028 (estimated)
- First posted
- 2 Feb 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Detailed description
This is a first-in-human and first-in-patient, 4-part study that includes the evaluation of safety, tolerability, and PK of: single ascending doses (SAD) of RM-718 weekly (RM-718) in healthy subjects 18 to 55 years of age with obesity (Part A), multiple ascending doses (MAD) of RM-718 in healthy subjects 18 to 55 years of age with obesity (Part B), MAD of RM-718 in patients 12 to 65 years of age with HO (Part C), and MAD of RM-718 in patients with PWS (Part D). Cohorts in Parts A and B are double-blind, placebo-controlled, and randomized 2:1 (4 subjects receive RM-718, 2 subjects receive placebo). Part C evaluates open-label dose escalation in patients 12 to 65 years of age with HO. Part D evaluates open-label dose escalation in patients 12 to 65 years of age. Study participants will receive: 1 weekly dose of either RM-718 or placebo in Part A, 4 weekly doses of either RM-718 or placebo in Part B,16 weekly doses of open-label RM-718 in Part C, and 26 weekly doses of RM-718 in Part D. Study drug (RM-718 or placebo) doses are administered weekly via subcutaneous injection.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 17
| Arm | Type | Receives |
|---|---|---|
| RM-718 (Cohort A1) | Experimental | Drug: Part A: RM-718 or placebo (matched to specific RM-718 dose cohort)Single dose of RM-718 (4) or placebo (2) |
| RM-718 (Cohort A2) | Experimental | Drug: Part A: RM-718 or placebo (matched to specific RM-718 dose cohort)Single dose of RM-718 (4) or placebo (2) |
| RM-718 (Cohort A3) | Experimental | Drug: Part A: RM-718 or placebo (matched to specific RM-718 dose cohort)Single dose of RM-718 (4) or placebo (2) |
| RM-718 (Cohort A4) | Experimental | Drug: Part A: RM-718 or placebo (matched to specific RM-718 dose cohort)Single dose of RM-718 (4) or placebo (2) |
| RM-718 (Cohort A5) | Experimental | Drug: Part A: RM-718 or placebo (matched to specific RM-718 dose cohort)Single dose of RM-718 (4) or placebo (2) |
| RM-718 (Cohort A6) | Experimental | Drug: Part A: RM-718 or placebo (matched to specific RM-718 dose cohort)Single dose of RM-718 (4) or placebo (2) |
| RM-718 (Cohort A7) | Experimental | Drug: Part A: RM-718 or placebo (matched to specific RM-718 dose cohort)Single dose of RM-718 (4) or placebo (2) |
| RM-718 (Cohort A8) | Experimental | Drug: Part A: RM-718 or placebo (matched to specific RM-718 dose cohort)Single dose of RM-718 (4) or placebo (2) |
| RM-718 (Cohort A9) | Experimental | Drug: Part A: RM-718 or placebo (matched to specific RM-718 dose cohort)Single dose of RM-718 (4) or placebo (2) |
| RM-718 (Cohort B1) | Experimental | Drug: Part B: RM-718 or placebo (matched to specific RM-718 dose cohort)Multiple ascending doses of RM-718 (4) or placebo (2) |
| RM-718 (Cohort B2) | Experimental | Drug: Part B: RM-718 or placebo (matched to specific RM-718 dose cohort)Multiple ascending doses of RM-718 (4) or placebo (2) |
| RM-718 (Cohort B3) | Experimental | Drug: Part B: RM-718 or placebo (matched to specific RM-718 dose cohort)Multiple ascending doses of RM-718 (4) or placebo (2) |
| RM-718 (Cohort B4) | Experimental | Drug: Part B: RM-718 or placebo (matched to specific RM-718 dose cohort)Multiple ascending doses of RM-718 (4) or placebo (2) |
| RM-718 (Cohort B5) | Experimental | Drug: Part B: RM-718 or placebo (matched to specific RM-718 dose cohort)Multiple ascending doses of RM-718 (4) or placebo (2) |
| RM-718 (Cohort B6) | Experimental | Drug: Part B: RM-718 or placebo (matched to specific RM-718 dose cohort)Multiple ascending doses of RM-718 (4) or placebo (2) |
| RM-718 (Cohort C1) | Experimental | Drug: Part C: RM-718Multiple ascending doses of RM-718 (30) |
| RM-718 (Cohort D1) | Experimental | Drug: Part D: RM-718Multiple ascending doses of RM-718 (up to 30) |
Who may qualify
- Ages
- 12 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (19 listed)
- 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.
Key exclusion criteria (29 listed)
- Parts A and B
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Part A: RM-718 or placebo (matched to specific RM-718 dose cohort)Single ascending dose of RM-718 or placebo (matched to specific RM-718 Part A dose cohort) | Drug | RM-718 (Cohort A1), RM-718 (Cohort A2), RM-718 (Cohort A3), RM-718 (Cohort A4), RM-718 (Cohort A5), RM-718 (Cohort A6), RM-718 (Cohort A7), RM-718 (Cohort A8), RM-718 (Cohort A9) |
| Part B: RM-718 or placebo (matched to specific RM-718 dose cohort)Multiple ascending doses of RM-718 or placebo (matched to specific RM-718 Part B dose cohorts) | Drug | RM-718 (Cohort B1), RM-718 (Cohort B2), RM-718 (Cohort B3), RM-718 (Cohort B4), RM-718 (Cohort B5), RM-718 (Cohort B6) |
| Part C: RM-718Multiple ascending doses of RM-718 | Drug | RM-718 (Cohort C1) |
| Part D: RM-718Multiple ascending doses of RM-718 | Drug | RM-718 (Cohort D1) |
Primary outcome
Parts A, B, C, D: Safety and Tolerability Assessed by Number of Study Participants with Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time frame: From Day 1 through the Safety-Follow-up call (up to Day 43 for all Part A cohorts, up to Day 70 for all Part B cohorts, up to Day 140 for Part C cohort, up to Day 210 for Part D cohort)
24 secondary outcomes
AUCtau measurement of RM-718
Time frame: up to 168 hours post-dose on Day 1 (Parts A, B and C) and 168 hours post-dose on Day 22 (Parts B and C).
Cmax measurement of RM-718
Time frame: up to 168 hours post-dose on Day 1 (Parts A, B and C) and 168 hours post-dose on Day 22 (Parts B and C).
Cmin measurement of RM-718
Time frame: up to 168 hours post-dose on Day 1 (Parts A, B and C) and 168 hours post-dose on Day 22 (Parts B and C).
Tmax measurement of RM-718
Time frame: up to 168 hours post-dose on Day 1 (Parts A, B and C) and 168 hours post-dose on Day 22 (Parts B and C).
Tmin measurement of RM-718
Time frame: up to 168 hours post-dose on Day 1 (Parts A, B and C) and 168 hours post-dose on Day 22 (Parts B and C).
Ctrough measurement of RM-718
Time frame: up to 168 hours post-dose on Day 1 (Parts A, B and C) and 168 hours post-dose on Day 22 (Parts B and C).
Cavg measurement of RM-718
Time frame: up to 168 hours post-dose on Day 1 (Parts A, B and C) and 168 hours post-dose on Day 22 (Parts B and C).
t1/2 measurement of RM-718
Time frame: up to 168 hours post-dose on Day 1 (Parts A, B and C) and 168 hours post-dose on Day 22 (Parts B and C).
λz measurement of RM-718
Time frame: up to 168 hours post-dose on Day 1 (Parts A, B and C) and 168 hours post-dose on Day 22 (Parts B and C).
CL/F measurement of RM-718
Time frame: up to 168 hours post-dose on Day 1 (Parts A, B and C) and 168 hours post-dose on Day 22 (Parts B and C).
Vz/F measurement of RM-718
Time frame: up to 168 hours post-dose on Day 1 (Parts A, B and C) and 168 hours post-dose on Day 22 (Parts B and C).
Accumulation ratio of RM-718
Time frame: Week 1 to Week 4 (AUC on Week 4/AUC on Week 1) (Parts B, C)
Change from baseline in BMI (Part C only)
Time frame: Baseline to Week 16
Mean change in weight (Part C only)
Time frame: Baseline to Week 16
Mean change in waist circumference (Part C only)
Time frame: Baseline to Week 16
Mean change in weekly average of the daily most hunger score in patients ≥12 years of age (Part C only)
Time frame: Baseline to Week 16
Mean change in weekly average of the Symptoms of Hyperphagia composite score (Part C only)
Time frame: Baseline to Week 16
Ctrough measurement of RM-718 (Part D)
Time frame: up to 168 hours post-dose on Day 8 and up to 168 hours post-dose on Day 29
Change from baseline in BMI (Part D)
Time frame: Baseline to Week 26
Mean change in weight (Part D)
Time frame: Baseline to Week 26
Mean change in waist circumference (Part D)
Time frame: Baseline to Week 26
Mean change in the weekly average of the Prader-Willi Syndrome Food Problem Diary (PWS-FPD) total score (Part D)
Time frame: Baseline to Week 26
Mean change in the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) total score (Part D)
Time frame: Baseline to Week 26
Change in total body mass (Part D)
Time frame: Baseline to Week 26
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Oct 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
David Meeker study chair
Rhythm Pharmaceuticals, Inc.
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Part C: RM-718 and Part D: RM-718 in people with Hypothalamic Obesity and Prader-Willi Syndrome, run by Rhythm Pharmaceuticals, Inc. at 7 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 19 inclusion and 29 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 9 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Parts A, B, C, D: Safety and Tolerability Assessed by Number of Study Participants with Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) (time frame: From Day 1 through the Safety-Follow-up call (up to Day 43 for all Part A cohorts, up to Day 70 for all Part B cohorts, up to Day 140 for Part C cohort, up to Day 210 for Part D cohort)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Oct 2026.