CompletedPhase 2

Phase 2 Trial to Evaluate Safety and Efficacy of Setmelanotide (RM-493) in Obese Participants With Prader-Willi Syndrome

United States40 participantsStarted 2015-03-19

Plain-language summary

The purpose of this study was to evaluate the effects of a once daily subcutaneous injectable formulation of setmelanotide in obese participants with Prader-Willi syndrome on tolerability, weight loss, and hyperphagia-related behavior. The study drug (setmelanotide and placebo) was administered in a blinded fashion.

Who can participate

Age range

16 Years – 65 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. PWS due to chromosome 15 micro-deletion, maternal uniparental disomy, or imprinting defect, confirmed by fluorescent in situ hybridization, chromosomal microarray, and/or methylation studies. Obese male or female participants weighing at least 50 kilograms (kg) with body mass index (BMI) ≥ 27 kilogram per square meter (kg/m²)
. Age 16-65 years
. If a participant has diagnosis of type 2 diabetes, following criteria must be met:
. hemoglobin A1C (HbA1c) \< 7.5% not being managed with insulin. Participants taking glucagon-like peptide-1 (GLP-1) analogues (exenatide or liraglutide) must have been on stable dose for greater than 3 months.
. Fasting plasma glucose \< 140 milligrams per deciliter (mg/dL)
. No history of ketoacidosis or hyperosmolar coma

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE) - Period 2

Timeframe: Days 15 to 41

Number of Participants Who Experienced a TEAE - Period 3

Timeframe: Days 42 to 55

Number of Participants Who Experienced a TEAE - Period 4

Timeframe: Days 56 to 69

Mean Body Weight - Period 2

Timeframe: Baseline (Day 15)

Percent Change From Baseline in Body Weight - Period 2

Timeframe: Baseline (Day 15) and Day 42

Overall Score of Prader-Willi Syndrome (PWS) Hyperphagia Questionnaire - Period 2

Timeframe: Baseline (Day 15)

Percent Change From Baseline in Overall Score of Prader-Willi Syndrome (PWS) Hyperphagia Questionnaire - Period 2

Timeframe: Baseline (Day 15) and Day 42

Trial details

NCT IDNCT02311673

SponsorRhythm Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2016-10-26

Results submitted2023-05-18

View on ClinicalTrials.gov More Prader-Willi Syndrome trials

Plain-language summary

Who can participate

Age range

16 Years – 65 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. PWS due to chromosome 15 micro-deletion, maternal uniparental disomy, or imprinting defect, confirmed by fluorescent in situ hybridization, chromosomal microarray, and/or methylation studies. Obese male or female participants weighing at least 50 kilograms (kg) with body mass index (BMI) ≥ 27 kilogram per square meter (kg/m²)
. Age 16-65 years
. If a participant has diagnosis of type 2 diabetes, following criteria must be met:
. hemoglobin A1C (HbA1c) \< 7.5% not being managed with insulin. Participants taking glucagon-like peptide-1 (GLP-1) analogues (exenatide or liraglutide) must have been on stable dose for greater than 3 months.
. Fasting plasma glucose \< 140 milligrams per deciliter (mg/dL)
. No history of ketoacidosis or hyperosmolar coma

What they're measuring

Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE) - Period 2

Timeframe: Days 15 to 41

Number of Participants Who Experienced a TEAE - Period 3

Timeframe: Days 42 to 55

Number of Participants Who Experienced a TEAE - Period 4

Timeframe: Days 56 to 69

Mean Body Weight - Period 2

Timeframe: Baseline (Day 15)

Percent Change From Baseline in Body Weight - Period 2

Timeframe: Baseline (Day 15) and Day 42

Overall Score of Prader-Willi Syndrome (PWS) Hyperphagia Questionnaire - Period 2

Timeframe: Baseline (Day 15)

Percent Change From Baseline in Overall Score of Prader-Willi Syndrome (PWS) Hyperphagia Questionnaire - Period 2

Timeframe: Baseline (Day 15) and Day 42

Phase 2 Trial to Evaluate Safety and Efficacy of Setmelanotide (RM-493) in Obese Participants With Prader-Willi Syndrome

Plain-language summary

Who can participate

Inclusion criteria

Questions worth asking your doctor

Questions for the trial coordinator

What they're measuring

Trial details

Phase 2 Trial to Evaluate Safety and Efficacy of Setmelanotide (RM-493) in Obese Participants With Prader-Willi Syndrome

Plain-language summary

Who can participate

Inclusion criteria

Questions worth asking your doctor

Questions for the trial coordinator

What they're measuring

Trial details

Exclusion criteria